abstract directory, wednesday may 4, 2016 · ivana trapani, alberto auricchio 29 11:30 am ......

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ASGCT 19TH ANNUAL MEETING MAY 4- 7 , 2016 WASHINGTO N , DC ABSTRACT DIRECTORY AND AUTHOR INDEX — 1 Oral Abstract Session 110 RNA Virus Vectors 10:30 AM - 12:00 PM Room: Roosevelt 1-2 1 10:30 AM A Direct Comparison of Foamy and Lentiviral Vector Genotoxicity in SCID-Repopulating Cells Shows Foamy Vectors Are Less Prone to Clonal Dominance Elizabeth M. Everson, Miles E. Olzsko, David J. Leap, Jonah D. Hocum, Grant D. Trobridge 2 10:45 AM Identification and Ranking of Different Chromatin Insulators to Block Vector-Driven Enhancer-Mediated Insertional Mutagenesis In Vivo Monica Volpin, Andrea Calabria, Daniela Cesana, Erika Tenderini, Fabrizio Benedicenti, Giulio Spinozzi, Jack Lenz, George Stamatoyannopoulos, Eugenio Montini 3 11:00 AM Development of a Cas9 Protein Delivery System with Lentiviral Vectors for RNA-Guided Genome Editing Naoya Uchida, Anna Shvygin, Luke Skala, Lydia Raines, Josiah Ballantine, John Tisdale 4 11:15 AM β-Deliverin: A Small Molecule for Improving Gene Transfer to Hematopoietic Stem Cells and Probing Mechanisms of Lentiviral Vector Restriction Nina Timberlake, Stosh Ozog, Saritha D’souza, Igor Slukvin, Sergio D. Catz, Scott Snyder, Bruce E. Torbett 5 11:30 AM Development of Enhancing Intraosseous Delivery Efficiency of LV-Factor VIII Variants in Platelets of Hemophilia A Mice Xuefeng Wang, Richard Y. Fu, Carol H. Miao 6 11:45 AM Retargeted Foamy Virus Vectors Integrate Less Frequently Near Proto-Oncogenes Jonah D. Hocum, Ian Linde, Dustin T. Rae, Casey P. Collins, Lindsay K. Matern, Grant D. Trobridge Oral Abstract Session 111 AAV Vector Biology 10:30 AM - 12:00 PM Room: Thurgood Marshall SW 7 10:30 AM High-Throughput Sequencing of AAV Proviral Libraries from the Human Population Reveals Novel Variants with Unprecedented Intra- and Inter-Tissue Diversity Guangchao Xu, Li Luo, Phillip W. L. Tai, Wanru Qin, Yuanyuan Xiao, Chunmei Wang, Qin Su, Hong Ma, Ran He, Yuquan Wei, Guangping Gao 8 10:45 AM Further Characterization of U2 snRNP Mediated Restriction of AAV Vector Transduction Claire A. Schreiber, Yoshihiro Izumiya, Aravind Asokan, Yasuhiro Ikeda 9 11:00 AM rAAV Designs Harboring DNA Secondary Structures with High Thermal Stabilities Produce Heterogenic Viral Genome Populations Jun Xie, Ran He, Qin Su, Phillip W. L. Tai, Hong Ma, Jia Li, Guangping Gao 10 11:15 AM High-Efficiency Transduction of Primary Human CD34 + Hematopoietic Stem/Progenitor Cells by AAV6 Serotype Vectors: Strategies for Overcoming Donor Variation and Implications in Genome Editing Zifei Yin, Kanit Bukhai, George Aslanidi, Chen Ling, Mengqun Tan, Mervin Yoder, Philippe Leboulch, Emmanuel Payen, Arun Srivastava 11 11:30 AM The Role of Glucocorticoid Receptor Signaling in Adeno- Associated Virus 2 Infection Chen Ling, Jun Li, Daniel Zhang, George Aslanidi, Changquan Ling, Arun Srivastava 12 11:45 AM Assembly-Activating Protein Is Not an Essential Requirement for Capsid Assembly of Adeno-Associated Virus Serotypes 4, 5, and 11 Lauriel F. Earley, John Powers, Kei Adachi, Nancy L. Meyer, Qing Xie, Michael S. Chapman, Hiroyuki Nakai Abstract Directory, Wednesday May 4, 2016

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Page 1: Abstract Directory, Wednesday May 4, 2016 · ivana Trapani, Alberto Auricchio 29 11:30 AM ... Giovanni Sitia, Angelo lombardo, luigi naldini 43 5:00 PM CRISPR/Cas9 and rAAV6-Mediated

ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 1

Oral Abstract Session 110RNA Virus Vectors10:30 AM - 12:00 PMroom: roosevelt 1-2

1 10:30 AMA Direct Comparison of Foamy and Lentiviral Vector Genotoxicity in SCID-Repopulating Cells Shows Foamy Vectors Are Less Prone to Clonal Dominanceelizabeth M. everson, Miles e. olzsko, David J. leap, Jonah D. hocum, Grant D. Trobridge

2 10:45 AMIdentification and Ranking of Different Chromatin Insulators to Block Vector-Driven Enhancer-Mediated Insertional Mutagenesis In VivoMonica Volpin, Andrea Calabria, Daniela Cesana, erika Tenderini, Fabrizio benedicenti, Giulio Spinozzi, Jack lenz, George Stamatoyannopoulos, eugenio Montini

3 11:00 AMDevelopment of a Cas9 Protein Delivery System with Lentiviral Vectors for RNA-Guided Genome Editingnaoya uchida, Anna Shvygin, luke Skala, lydia raines, Josiah ballantine, John Tisdale

4 11:15 AMβ-Deliverin: A Small Molecule for Improving Gene Transfer to Hematopoietic Stem Cells and Probing Mechanisms of Lentiviral Vector Restrictionnina Timberlake, Stosh ozog, Saritha D’souza, igor Slukvin, Sergio D. Catz, Scott Snyder, bruce e. Torbett

5 11:30 AMDevelopment of Enhancing Intraosseous Delivery Efficiency of LV-Factor VIII Variants in Platelets of Hemophilia A Micexuefeng Wang, richard y. Fu, Carol h. Miao

6 11:45 AMRetargeted Foamy Virus Vectors Integrate Less Frequently Near Proto-OncogenesJonah D. hocum, ian linde, Dustin T. rae, Casey P. Collins, lindsay K. Matern, Grant D. Trobridge

Oral Abstract Session 111AAV Vector Biology10:30 AM - 12:00 PMroom: Thurgood Marshall SW

7 10:30 AMHigh-Throughput Sequencing of AAV Proviral Libraries from the Human Population Reveals Novel Variants with Unprecedented Intra- and Inter-Tissue DiversityGuangchao xu, li luo, Phillip W. l. Tai, Wanru Qin, yuanyuan xiao, Chunmei Wang, Qin Su, hong Ma, ran he, yuquan Wei, Guangping Gao

8 10:45 AMFurther Characterization of U2 snRNP Mediated Restriction of AAV Vector TransductionClaire A. Schreiber, yoshihiro izumiya, Aravind Asokan, yasuhiro ikeda

9 11:00 AMrAAV Designs Harboring DNA Secondary Structures with High Thermal Stabilities Produce Heterogenic Viral Genome PopulationsJun xie, ran he, Qin Su, Phillip W. l. Tai, hong Ma, Jia li, Guangping Gao

10 11:15 AMHigh-Efficiency Transduction of Primary Human CD34+ Hematopoietic Stem/Progenitor Cells by AAV6 Serotype Vectors: Strategies for Overcoming Donor Variation and Implications in Genome EditingZifei yin, Kanit bukhai, George Aslanidi, Chen ling, Mengqun Tan, Mervin yoder, Philippe leboulch, emmanuel Payen, Arun Srivastava

11 11:30 AMThe Role of Glucocorticoid Receptor Signaling in Adeno-Associated Virus 2 InfectionChen ling, Jun li, Daniel Zhang, George Aslanidi, Changquan ling, Arun Srivastava

12 11:45 AMAssembly-Activating Protein Is Not an Essential Requirement for Capsid Assembly of Adeno-Associated Virus Serotypes 4, 5, and 11lauriel F. earley, John Powers, Kei Adachi, nancy l. Meyer, Qing xie, Michael S. Chapman, hiroyuki nakai

Abstract Directory, Wednesday May 4, 2016

Page 2: Abstract Directory, Wednesday May 4, 2016 · ivana Trapani, Alberto Auricchio 29 11:30 AM ... Giovanni Sitia, Angelo lombardo, luigi naldini 43 5:00 PM CRISPR/Cas9 and rAAV6-Mediated

ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

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Oral Abstract Session 112Adenoviruses10:30 AM - 12:00 PMroom: Washington 3

13 10:30 AMFunctional Role of Adenovirus Penton in Modulating In Vivo Properties of Liver-Targeted and Liver-Detargeted Adenovirus VariantsSvetlana Atasheva, Corey emerson, nelson C. Di Paolo, eric irons, neetu Gulati, Phoebe Stewart, Dmitry M. Shayakhmetov

14 10:45 AMEngineering and Characterization of a Cloned Adenoviral-Library to Explore Natural Virus Diversity Wenli Zhang, Jun Fu, Jing liu, Sebastian Janz, Maren Schiwon, eric ehrke-Schulz, Johannes Dörner, Philip boehme, Manish Solanki, Thorsten bergmann, hailong Wang, Andreas Petzold, roma yumul, Andre lieber, Francis Stewart, Anja ehrhardt

15 11:00 AMA Novel Fast Production Pipeline for High Capacity Adenoviral Vectors to Deliver All Components of CRISPR/Cas9 System for Somatic Gene Editing Using One Single Viral Vector with Multiple Guide RNAseric ehrke-Schulz, Theo leitner, Stephan Dávid, Jing liu, Maren Schiwon, Thorsten bergmann, Anja ehrhardt

16 11:15 AMSTAT2 Knockout Syrian Hamsters Support Enhanced Replication and Pathogenicity of Human Adenovirus Type 5, Revealing an Important Role of Type I Interferon Response in Viral ControlKaroly Toth, Sang r. lee, baoling ying, Jacqueline F. Spencer, Ann e. Tollefson, John e. Sagartz, il-Keun Kong, Zhongde Wang, William Wold

17 11:30 AMHuman Adenovirus D17 Has Tropism for Endothelium Cells and Can Use Both hCAR and CD46 as ReceptorsJing liu, Philip boehme, Jun Fu, Wenli Zhang, Manish Solanki, roma yumul, Achim Schmidtko, Francis Stewart, Andre lieber, Anja ehrhardt

18 11:45 AMDelivery of CRISPR/Cas9 or TALENs Against Hepatitis B Virus cccDNA by High-Capacity Adenoviral VectorsMaren Schiwon, Thorsten bergmann, eric ehrke-Schulz, Anja ehrhardt

Oral Abstract Session 113Cardiovascular and Pulmonary Diseases10:30 AM - 12:00 PMroom: Washington 5-6

19 10:30 AMUsing a Novel Combinatorial Non-Viral Vector and Small Molecules to Treat Familial Hypercholesterolaemia (FH)Alastair G. Kerr, lawrence Tam, Milena Cioroch, Ashley hale, Gillian Douglas, Joe Mason, Carole bataille, Graham Wynne, Angela russell, Keith Channon, richard Wade-Martins

20 10:45 AMA Novel Approach in the Treatment of Dystrophic CardiomyopathyMona el refaey, li xu, Jing xu, lixia Zhao, yandi Gao, renzhi han

21 11:00 AMPulmonary Macrophage Transplantation (PMT) Therapy of Hereditary Pulmonary Alveolar Proteinosis (hPAP) Is Effective with Mature Macrophages and Does Not Require Myeloid Precursor/Progenitor ExpansionTakuji Suzuki, Paritha Arumugam, Anthony Sallese, nico lachmann, Claudia Chalk, Diane black, brenna Carey, Thomas Moritz, bruce C. Trapnell

22 11:15 AMPost-Exposure Vaccination by Capsid-Modified AdC7 Vector Expressing Pseudomonas aeruginosa OprF in Chronic P. aeruginosa Lung InfectionAnurag Sharma, rika Gomi, Wenzhu Wu, biin Sung, Stefan Worgall

23 11:30 AMSiRNA and CRISPR/Cas9 Mediated Knockout of αENACAristides Tagalakis, Joost van haasteren, Ahmad Aldossary, luca rosa, Mustafa Munye, David McCarthy, Stephen hart

24 11:45 AMSustained Expression with Partial Correction of Neutrophil Defects 5 Years After Intramuscular rAAV1 Gene Therapy for Alpha-1 Antitrypsin DeficiencyChristian Mueller, Jeffrey D. Chulay, noel G. Mcelvaney, Gwladys Gernoux, emer P. reeves, Farshid n. rouhani, Margaret humphries, Alisha M. Gruntman, Martha Campbell-Thompson, James M. Wilson, Terence r. Flotte

Page 3: Abstract Directory, Wednesday May 4, 2016 · ivana Trapani, Alberto Auricchio 29 11:30 AM ... Giovanni Sitia, Angelo lombardo, luigi naldini 43 5:00 PM CRISPR/Cas9 and rAAV6-Mediated

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Oral Abstract Session 114Delivery Questions for Neural Applications10:30 AM - 12:00 PMroom: Washington 1-2

25 10:30 AMIntranasal Gene Delivery of AAV Iduronidase: An Effective and Non-Invasive Approach for Treatmentof CNS Disease in a Murine Model of MPS Type Ilalitha belur, Megan buckvold, Kelly M. Podetz-Pedersen, Maureen riedl, lucy Vulchanova, leah hanson, Carolyn Fairbanks, Karen Kozarsky, William h. Frey ii, Walter C. low, r. Scott Mcivor

26 10:45 AMProtein Delivery of an Artificial Transcription Factor Restores Widespread Ube3a Expression in an Angelman Syndrome Mouse Brainbarbara bailus, benjamin Pyles, Michelle McAlister, henriette o’Geen, Sarah lockwood, Alexa Adams, Jennifer Trang nguyen, Abigail yu, robert berman, David J. Segal

27 11:00 AMTherapeutic Efficacy of Intracranial and Intrathecal AAV2/9-PPT1 in Infantile Batten DiseaseCharles Shyng, hemanth r. nelvagal, Josh T. Dearborn, Jonathan D. Cooper, Mark S. Sands

28 11:15 AMMouse and Pig Photoreceptor Transduction Mediated by Triple AAV VectorsAndrea Maddalena, Patrizia Tornabene, Fabio Dell’Aquila, ivana Trapani, Alberto Auricchio

29 11:30 AMNovel Surgical Method for Intravitreal AAV Administration Overcomes Transduction Barriers in Non-Human PrimatesJason Comander, livia Carvalho, Sarah Wassmer, ru xiao, eva Plovie, Aliete langsdorf, lawrence lim, brian hafler, David M. Wu, Dean eliott, leo A. Kim, luk h. Vandenberghe

30 11:45 AMModulation of Intracellular Calcium Enhances AAV Transduction in the CNSGarrett berry, Giridhar Murlidharan, Aravind Asokan

Oral Abstract Session 115Pre-Clinical Development Activities for Gene Therapy Trials10:30 AM - 12:00 PMroom: Washington 4

31 10:30 AMAAV Gene Therapy for Choroideremia: Dose Determination Analysesirena ignatova, Paolo Falabella, Katherine A. high, linda b. Couto

32 10:45 AMTowards a Phase I Clinical Trial for CystinosisTatiana lobry, Jay Sharma, Sarah ur, Athena lau, Celine rocca, Donald b. Kohn, Denise Carbonaro, laura hernandez, Stephanie Cherqui

33 11:00 AMAdvancing a State of the Art Gene Therapy Called OXB-202 That Resists Corneal Rejection in High Risk Patientsnaghmeh Fouladi, Katie binley, laura McCloskey, Julie loader, Michelle Kelleher, Kyriacos Mitrophanous, Scott ellis

34 11:15 AMDevelopment and Validation of Identity and Homogeneity Assays for AAV PreparationsSimon Pacouret, Mathieu Mevel, Mohammed bouzelha, Anna Maurer, rajani Shelke, ru xiao, Magalie Penaud-budloo, heikki Turunen, Frederic broucque, Véronique blouin, Philippe Moullier, eduard Ayuso, luk h. Vandenberghe

35 11:30 AMEstablishment of the Dose-Response Relationship Needed for Human Translation of Pulmonary Macrophage Transplantation (PMT) Therapy of Hereditary Pulmonary Alveolar Proteinosis (hPAP)Paritha i. Arumugam, Takuji Suzuki, Anthony Sallese, nico lachmann, Claudia Chalk, Diane black, brenna Carey, Thomas Moritz, bruce C. Trapnell

36 11:45 AMTranslation of an Adenovirus-Based Cocaine Vaccine dAd5GNE to a Clinical TrialDavid F. havlicek, bishnu De, Jonathan rosenberg, odelya Pagovich, Dolan Sondhi, Stephen Kaminsky, ronald Crystal

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Oral Abstract Session 140Targeted Genome Editing: Gene Editing in Hematopoietic Cells3:30 PM - 5:30 PMroom: Thurgood Marshall ne

37 3:30 PMTowards Clinical Translation of Hematopoietic Stem Cell Gene Editing for the Correction of SCID-X1 MutationsPietro Genovese, Giulia Schiroli, Anthony Conway, Samuele Ferrari, Dmitry y. Guschin, Tiziano Di Tomaso, luisa Albano, Virna Marin, erica Giuliani, Philip D. Gregory, Michael C. holmes, bernhard Gentner, Chiara bovolenta, Gregory J. Cost, Angelo lombardo, luigi naldini

38 3:45 PMCCR5 Gene Edited Hematopoietic Stem Cells Engraft in Diverse Anatomical Locales and Undergo SHIV-Dependent Positive Selection in Nonhuman PrimatesChristopher W. Peterson, Jianbin Wang, Patricia Polacino, Shiu-lok hu, Michael C. holmes, hans-Peter Kiem

39 4:00 PMFACS-Based Enrichment of a Highly Purified HBB-Targeted Hematopoietic Stem and Progenitor Cell Population Using rAAV6 and CRISPR/Cas9Daniel P. Dever, rasmus o. bak, Joab Camarena, nivi Saxena, Andreas reinisch, Carmencita e. nicolas, Alec b. Wilkens, Ayal hendel, nobuko uchida, ravindra Majeti, Kenneth i. Weinberg, Matthew h. Porteus

40 4:15 PMNuclease-Targeted Gene-Editing of FOXP3 in Primary T Cells Creates a Stable and Functional Treg Phenotypenicholas W. hubbard, David hagin, Karen M. Sommer, yumei Song, iram Khan, Andrew M. Scharenberg, David J. rawlings, Troy r. Torgerson

41 4:30 PMTherapeutic Level CRISPR-Oligomer-Mediated Correction of X-CGD Patient Hematopoietic Stem Cells Using Non-Viral, cGMP Compliant, Scalable, and Closed Systemlinhong li, Suk see De ravin, Cornell Allen, uimook Choi, Sherry Koontz, narda Theobald, Janet lee, Angelia Viley, Pachai natarajan, hannah newCombe, Ashley McMichael, xiaolin Wu, harry l. Malech, Madhusudan V. Peshwa

42 4:45 PMCorrection of SCID-X1 by Targeted Genome Editing of Hematopoietic Stem/Progenitor Cells (HSPC) in the Mouse ModelGiulia Schiroli, Pietro Genovese, Valentina Capo, Maria C. Castiello, luisa Albano, Michael C. holmes, Anna Villa, Giovanni Sitia, Angelo lombardo, luigi naldini

43 5:00 PMCRISPR/Cas9 and rAAV6-Mediated Targeted Integration at the CCR5 Locus in Hematopoietic Stem and Progenitor Cellsrasmus o. bak, Daniel P. Dever, nivedita Saxena, Joab Camarena, Andreas reinisch, Alec b. Wilkens, Carmencita e. nicolas, Ayal hendel, ravindra Majeti, Kenneth i. Weinberg, Matthew h. Porteus

44 5:15 PMNovel Combination of megaTAL Nuclease-Driven Genome Engineering with a Drug Selection Cassette Increases Efficiency of HIV Gene Therapybiswajit Paul, Guillermo romano, Kevin haworth, Patrick younan, nicholas hubbard, Donald Gisch, iram Khan, Alexander Astrakhan, David J. rawlings, hans-Peter Kiem

Oral Abstract Session 141Diabetes, Metabolic and Genetic Diseases3:30 PM - 5:30 PMroom: Washington 3

45 3:30 PMRescue of the Functional Alterations of Motor Cortical Circuits in Arginase 1 Deficiency with AAV-Based Gene TherapyGloria Cantero, xiao-bo liu, Steven D. Cederbaum, Peyman Golshani, Gerald S. lipshutz

46 3:45 PMThe First Viable Mouse Model of cblC Type Combined Methylmalonic Acidemia and Homocysteinemia: AAV Gene Therapy Rescues Neonatal Lethality and Provides Insight into Disease-Associated Retinal DegenerationMadeline Arnold, Jennifer l. Sloan, nathan P. Achilly, Gene elliot, ighovie F. onojafe, brian P. brooks, Charles P. Venditti

47 4:00 PMGene Transfer-Mediated Diversion Towards Non-Toxic Metabolites for Therapy of Primary Hyperoxaluria Type 1nicola brunetti-Pierri, raffaele Castello, Patrizia Annunziata, roberta borzone

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48 4:15 PMTreatment of Methylmalonic Acidemia by Promoterless Gene-Targeting Using Adeno-Associated Viral (AAV) Mediated Homologous Recombinationrandy J. Chandler, Adi barzel, Mark A. Kay, Charles P. Venditti

49 4:30 PMMinimum Effective Dose of Liver-Targeted Gene Therapy for Pompe Disease Reduced 10-Fold from Prior EstimatesSang-oh han, benjamin Arnson, Korie busch, Giuseppe ronzitti, Songtao li, Federico Mingozzi, Dwight D. Koeberl

50 4:45 PMLong-Term Follow-Up of Diabetic Dogs Treated with Adeno-Associated Viral Vectors Encoding for Insulin and GlucokinaseVeronica Jimenez, Maria luisa Jaen, laia Vila, Jordi rodo, Miquel Garcia, ivet elias, luca Maggioni, xavier leon, Sergio Munoz, David Callejas, iris Grifoll, eduard Ayuso, Tura Ferre, Virginia haurigot, Fatima bosch

51 5:00 PMLV.InsB9-23/Anti-CD3 mAb Inhibits Recurrence of Autoimmunity in NOD Mice After Islet TransplantsFabio russo, Maria Grazia roncarolo, Andrea Annoni

52 5:15 PMTherapeutic Factor VIII Expression After AAV Delivery in Non-Human Primatesxavier M. Anguela, liron elkouby, raffaella Toso, Marti DiPietro, robert J. Davidson, Katherine A. high, Denise e. Sabatino

Oral Abstract Session 142Gene Therapy for CNS Diseases3:30 PM - 5:30 PMroom: Thurgood Marshall SW

53 3:30 PMA Neuro-Specific Gene Therapy Approach to Treat Cognitive Impairment in Down Syndrome by RNA InterferenceAndrea Contestabile, Martina Parrini, Shovan naskar, Micol Alberti, Anna rocchi, Marina nanni, Giuseppe ronzitti, Federico Mingozzi, laura Cancedda

54 3:45 PMPrevention of Sensory Ataxia in a Novel Mouse Model of Friedreich Ataxia Using Gene Therapy ApproachFrançoise Piguet, Charline de Montigny, nadège Vaucamps, laurence reutenauer, hélène Puccio

55 4:00 PMAquaporins and CSF Flux Are Critical Determinants of AAV Mediated CNS Gene TransferGiridhar Murlidharan, Aravind Asokan

56 4:15 PMIntracerebroventricular and Intravenous AAV Gene Therapy in Canine Globoid Cell LeukodystrophyAllison M. bradbury, Mohammad rafi, Gary P. Swain, Jessica h. bagel, Maria Prociuk, Patricia o’Donnell, David Peterson, Daniel S. ory, James M. Provenzale, David A. Wenger, Charles h. Vite

57 4:30 PMIntrathecal Administration of AAV/GALC Vectors in Juvenile Twitcher Mice Improves Survival and Is Enhanced by Bone Marrow TransplantSubha Karumuthil-Melethil, Mike Marshall, ernesto r. bongarzone, Steven J. Gray

58 4:45 PMPushing the Limits for Canavan Gene Therapy into Adulthood: Is There an Age Limit for Gene Therapy in CNS Disorders?Dominic J. Gessler, Danning li, hongxia xu, Qin Su, reuben Matalon, Guangping Gao

59 5:00 PMAPPsα Gene Therapy for Alzheimer’s Diseaseromain Fol, Susann ludewig, Tobias Abel, Sascha W. Weyer, Jan-Peter roederer, Florian brod, Mickael Audrain, Alexis-Pierre bemelmans, Christian J. buccholz, Martin Korte, ulrike C. Müller, nathalie Cartier-lacave

60 5:15 PMEngineering AAV Vector for the Delivery of Human BuChE to Protect Against Exposure to OrganophosphatesAngelica Medina-Jaszek, omua Ahokhai, Maryam Taheri, Muhammad Arif, Anna Tretiakova, James M. Wilson

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Oral Abstract Session 143Cancer-Oncolytic RNA Viruses3:30 PM - 5:30 PMroom: Washington 5-6

61 3:30 PMAscending Dose Trials of a Retroviral Replicating Vector (Toca 511) in Patients with Recurrent High-Grade Glioma: Clinical Update, Molecular Analyses, and Proposed Mechanism of ActionDouglas J. Jolly, Joan M. robbins, Derek ostertag, Carlos iban᷉ez, noriyuki Kasahara, harry Gruber, Steven n. Kalkanis, Michael Vogelbaum, Manish K. Aghi, Timothy Cloughesy, Alice Chu, Asha Das, Jamey Skillings

62 3:45 PMMiRNA Regulation of Oncolytic Measles Virus Infectivityhirosha Geekiyanage, evanthia Galanis

63 4:00 PMImmunogenicity of Self Tumor Associated Antigens Is Enhanced Through Protein TruncationTim Kottke, Kevin G. Shim, laura evgin, Vanesa Alonso-Camino, Shane Zaidi, rosa Diaz, Jose Pulido, Jill Thompson, Karishma rajani, Amanda huff, elizabeth ilett, hardev Pandha, Kevin harrington, Peter Selby, Alan Melcher, richard Vile

64 4:15 PMGeneration of Tumor Cells Resistant to Oncolysis Is Mediated Through Virus Induced APOBEC ExpressionKarishma rajani, Kevin G. Shim, nazanin yeganeh Kazemi, William Gendron, Tim Kottke, Amy Molan, Christopher Driscoll, reuben harris, richard Vile

66 4:30 PMRecombinant Newcastle Disease Virus as an Oncolytic Therapy for Ovarian and Prostate Cancerslisa A. Santry, Jacob P. van Vloten, Kathy Matuszewska, byram W. bridle, James J. Petrik, Sarah K. Wootton

67 4:45 PMRational Design of MicroRNA-Retargeted Coxsackievirus A21 Infectious RNA for Cancer Therapynoura elsedawy, Autumn ruiz, Stephen russell

68 5:00PMImmunovirotherapy: Enhancing Personalized T Cell Therapy with Oncolytic Measles Virus Vaccinationelena S. Czink, Tobias Speck, Johannes heidbuechel, Jessica Albert, birgit hoyler, Dirk Jaeger, Christof von Kalle, Guy ungerechts, Christine e. engeland

65 5:15 PMOncolytic Measles Virus Differentially Affects Mitochondrial Biogenesis in Transformed versus Non-Transformed BM-Derived MSCsSarah Aref, Aditi Dey, Katharine bailey, benjamin rosen, Anna Castleton, Adele K. Fielding

Oral Abstract Session 144Cancer-Targeted Gene and Cell Therapy3:30 PM - 5:30 PMroom: Washington 1-2

69 3:30 PMTNFR Costimulatory Domains Impair Expansion of CD5 CAR T Cells Due to Enhanced Fas-Mediated ApoptosisMaksim Mamonkin, Sandhya Sharma, Malini Mukherjee, Madhuwanti Srinivasan, Diogo Silva, Jordan S. orange, Malcolm K. brenner

70 3:45 PMKnocking Down the Circular RNA ciRS-E2 Blocks Cancer Cell Proliferation Demonstrating Circular RNAs as a New Therapeutic TargetAlessia b. baccarini, ravi Sachidanandam, brian brown

71 4:00 PMValidation of Manufacturing NKG2D Chimeric Antigen Receptor T Cells for a First-In-Human Clinical Trial in AML/MDS and Multiple Myelomaheather Daley, Sarah nikiforow, Joana Murad, helene negre, Jake reider, Charles Sentman, Sarah Snykers, rachel Allen, ilene Galinsky, nikhil Munshi, richard Stone, robert Soiffer, Jerome ritz, Suzanne baumeister

72 4:15 PMA Phase l Study of RB94 in Genitourinary Cancers Using a Tumor-Targeted Systemic Nanodelivery SystemArlene Siefker-radtke, xin-qiao Zhang, Charles C. Guo, Kathleen F. Pirollo, Chris P. leung, esther h. Chang, randall e. Millikan, William F. benedict

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73 4:30 PMEnhanced Engineering of Chimeric Antigen Receptor (CAR)-Modified T Cells Using Non-Viral Sleeping Beauty Transposition from Minicircle Vectorsrazieh Monjezi, Csaba Miskey, Tea Gogishvili, Martin Schleef, Marco Schmeer, Julia Wegner, hermann einsele, Zoltan ivics, Michael hudecek

74 4:45 PMThe Impact of Different Hinge and Transmembrane Components on the Function of a Novel Fully-Human Anti-CD19 Chimeric Antigen Receptorleah M. Alabanza, Melissa A. Pegues, Claudia Geldres, Victoria Shi, James n. Kochenderfer

75 5:00 PMDevelopment of an LMP-Specific T Cell Bank for Third Party Use as a Curative Strategy Post-Transplant Lymphoproliferative Disease After Solid Organ Transplantlauren Mclaughlin, lauren roesch, e. Kathy Mintz, Sarah McCormick, Cecilia barese, elizabeth Williams, neha Joshi, Fahmida hoq, Michael Keller, C. russell Cruz, Mitchell S. Cairo, Jessica hochberg, Patrick J. hanley, Catherine M. bollard

76 5:15 PMTransgenic Expression of IL15 Improves Antiglioma Activity of IL13Rα2-CAR T CellsGiedre Krenciute, irina balyasnikova, Gianpietro Dotti, Stephen Gottschalk

Oral Abstract Session 145Immunological Aspects of Gene Therapy I: AAV Vectors3:30 PM - 5:30 PMroom: Washington 4

77 3:30 PMAntigen-Specific Modulation of Capsid Immunogenicity with Tolerogenic Nanoparticles Results in Successful AAV Vector ReadministrationAmine Meliani, Florence boisgerault, Giuseppe ronzitti, Fanny Collaud, Christian leborgne, Takashi Kei Kishimoto, Federico Mingozzi

78 3:45 PMDistinct Roles of Dendritic Cell Subsets in Innate Immune Sensing and Cross-Presentation of AAV Capsid In VivoJamie l. Shirley, Geoffrey l. rogers, Monaro biswas, roland W. herzog

79 4:00 PMVector Dose Delineates Between Chronic, Non-Functional CD8+ T Cell Response and Tolerance to the Transgene Product Upon Liver Gene TransferSandeep Kumar, brad e. hoffman, roland W. herzog

80 4:15 PMDelayed Inflammatory Response to Intravitreal AAV Gene Transfer in Non-Human PrimatesSarah Wassmer, Jason Comander, livia Carvalho, ru xiao, eva Plovie, Aliete langsdorf, lawrence lim, brian hafler, David M. Wu, Dean eliott, leo A. Kim, luk h. Vandenberghe

81 4:30 PMDownregulation of Immune Responsive Chemokines Are Associated AAV8-Mediated Transgene Immune Toleranceyuanyuan xiao, Shaoyong li, Qin Su, li luo, Gaungchao xu, Wanru Qin, Chunmei Wang, yi lu, li yang, yuquan Wei, Guangping Gao

82 4:45 PMMapping the Humoral Immune Response to AAV by Molecular Docking and Cryo-Electron Microscopy for the Design of Next-Generation AAV VectorsApril r. Giles, lakshmanan Govindasamy, roberto Calcedo, Anna Tretiakova, Surina boyd, Qiuyue Qin, Mark Schneider, James M. Wilson

83 5:00 PMRelationship Between Immune Responses and Ocular Inflammation: What Is Learnt from Intravitreal Injection of rAAV2-2-ND4 (GS010) in Non-Human Primates and Leber Hereditary Optic Neuropathy (LHON) PatientsCéline bouquet, Anne Galy, Serge Fitoussi, Sandrine Meunier, roxane noel, Scott uretsky, Catherine Vignal, José A. sahel, Jean-Philippe Combal, nitza Thomasson

84 5:15 PMAlpha 1 Antitrypsin Protein & Gene Therapies for the Treatment of Lupus in Animal ModelsAhmed elshikha, yuanqing lu, Mong-Jen Chen, Mohammad Akbar, leilani Zeumer, ye yuan, laurence Morel, Sihong Song

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Poster Session I5:30 PM - 7:30 PMroom: exhibit hall C & b South

AAV Vectors I

85AAV9-Utrophin Prevents Myonecrosis in Dystrophic Mice and Dogs without Immunosuppressionyafeng Song, Alock Malik, Andrew Mead, Marilyn Mitchell, Mihail Petrov, xiangping lu, Daniel Vanbelzen, Maxwell Joffe, Margaret Choi, leonard Su, Joe Kornegay, Tejvir Tejvir Khurana, hansell Stedman

86AAV9 Mediated Correction of Iduronate-2-Sulfatase Deficiency in the Central Nervous System to Prevent the Onset of Neurologic Deficits in a Murine Model of Mucopolysaccharidosis Type II.Kanut laoharawee, Kelly M. Podetz-Petersen, Kelley Kitto, lucy Vulchanova, Carolyn Fairbanks, Karen Kozarsky, Walter C. low, r. Scott Mcivor

87Rationally Designed Inverted Terminal Repeats Enhance Transduction of Embryonic Stem Cellsnolan brown, Matthew hirsch

88Safety and Biodistribution Study of rAAV2tYF-PR1.7-hCNGB3 in Nonhuman PrimatesGuo-jie ye, ewa budzynski, Peter Sonnentag, T. Michael nork, Paul e. Miller, Alok K. Sharma, James n. Ver hoeve, leia M. Smith, Tara Arndt, roberto Calcedo, Chantelle Gaskin, Paulette M. robinson, David r. Knop, William W. hauswirth, Jeffrey D. Chulay

89Expanding the Toolkit of Protease-Activatable Viruses to Optimize Their Versatility and EfficiencyCaitlin Guenther, Michelle ho, Annicka evans, Paul Chariou, Momona yamagami, Michael lam, Junghae Suh

90Vector Biodistribution After Recombinant AAVrh10 Intrathecal Delivery in Non-Human PrimatesGwladys Gernoux, Florie borel, Guangping Gao, robert h. brown, Christian Mueller

91Development of Optimized [“Opt”] AAV Vectors by Combining Capsid-Modified NextGen and Genome-Modified GenX AAV Vectors for High-Efficiency Transduction at Further Reduced DosesChen ling, baozheng li, Wenqin Ma, George Aslanidi, Arun Srivastava

92Evaluation of Producer Cell Line Platform for Production of Oversized AAV-FVIII Vectorsbindu nambiar, Cathleen Sookdeo, Patricia berthelette, robert Jackson, brenda burnham, Shelley nass, David Souza, Catherine r. o’riordan, Karen Vincent, Seng h. Cheng, Donna Armentano, Sirkka Kyostio-Moore

93Development of AAVidua Vector for the Treatment of Cornea Associated Vision Loss in Bone Marrow Treated-Hurler PatientsMelisa Vance, llanga llanga, Joanne Kurtzberg, richard Jude Samulski, Matthew hirsch

94A Scalable Recombinant HSV-Based Manufacturing System for Production of Highly Potent Adeno-Associated Type 9 Vectorsnathalie Clement

95Incorporation of AAV Serotype 2 Viral Protein 2 into Other AAV Serotype CapsidsDaniel Zhang, Jun li, yinglu Feng, Adam Snowden, Georgy Aslanidi, nicholas Muzyczka, Arun Srivastava, Changquan ling, Chen ling

96 (Moved to Poster Session ii after Abstract 310)An Approach to Compare Multiple Adeno-Associated Virus (AAV) Capsids Side-by-Side in a Single Eye Following Intravitreal InjectionJohn M. Powers, Kei Adachi, benjamin Sivyer, William Taylor, Mark Pennesi, hiroyuki nakai

97Large Scale Purification of AAV with Continuous Flow Ultracentrifugationhaifeng Chen, Sandra Marino, Ching yi ho

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98Evaluation of Proteasome Inhibitors on AAV-Mediated Transduction Efficiency in Retinal Bipolar CellsShengjie Cui, Qi lu, Tushar Ganjawala, Gary Abrams, Zhuo-hua Pan

99Construction and Evaluation of Recombinant AAV Vectors for Central Nervous System Gene DeliveryDiego Sucunza, Jose Diego Pignataro, esperenza lopez-Franco, Alberto Jose rico, lucia Vanrell, iria Gonzalez, elvira roda, Africa Vales, Jose luis lanciego, Gloria Gonzalez-Aseguinolaza

100Evaluation of Ion-Exchange Membranes for the Purification of AAV8 from Culture Mediabryan A. Piras, David J. Mcnally, Timothy D. lockey, Amit C. nathwani, Michael M. Meagher

101Rational Design and Cloning of a Stable RPGR ORF15 cDNA Encoding the Full-Length Native RPGR ProteinJilin liu, Andrew espejo, Weihong Pan, Guojie ye

102Method Validation for Quantification of Recombinant Adeno-Associated Virus 5 in Mouse Genomic DNA Using Real-Time PCRhyun-Jung Kim, Sang-Jin Park, Sunyeong lee, ok-sun Kim, Su-yeon han, Kyoung-Sik Moon

Adenovirus Vectors and Other DNA Virus Vectors

103High-Capacity Adenoviral Vectors (HCAdV) Armed with a High Risk Human Papillomavirus (HPV) Oncogene Specific CRISPR/Cas9 Machinery Specifically Kill Cervical Cancer Cellseric ehrke-Schulz, Anja ehrhardt

104A Gene Deleted High Capacity Adenoviral Vector for Efficient Delivery of a Multiplex DMD Specific CRISPR/Cas9 Machineryeric ehrke-Schulz, rudolf A. Kley, Anja ehrhardt

105Enhanced Oncolytic Activity Mediated by a Novel Human Adenovirus Type 6 Based VectorJohannes Doerner, Wenli Zhang, Philip boehme, Anja ehrhardt

106A Sleeping Beauty Transposase System in the Context of HD-Ad5/35++ Vectors Achieves Stable In Vivo Transduction of Hematopoietic Stem Cells in Mouse ModelsMaximilian richter, Jing liu, Zsuzsanna izsvák, roberto Cattaneo, Wolfgang uckert, Philip ng, Anja ehrhardt, Thalia Papayannopoulou, André lieber

107Tracking Target Cell Fate After Oncolytic Herpes Simplex Virus InfectionAnna Grosskopf, Samuel D. rabkin, robert l. Martuza, hiroaki Wakimoto

108Pseudotyping Baculovirus Based Vectors for Enhanced In Vitro and In Vivo DeliveryJondavid de Jong, laura van lieshout, Jacob P. van Vloten, Sarah K. Wootton

109Therapeutic Efficacy and Safety Profile of EGFR-Targeted Oncolytic Ad Nanocomplex in Orthotopic Lung Tumor ModelA-rum yoon, Dayananda Kasala, yan li, Jinwoo hong, Wonsig lee, Soo-Jung Jung, Chae-ok yun

110Enhanced Intratumoral Accumulation of Nucleic Acid and Oncolytic Adenovirus by Complexation with Biocompatible and Bioreducible PolymerJung-Woo Choi, Jaesung Kim, yan li, Chae-ok yun, Sung Wan Kim

111HSV Vectors Retargeted for Infection of TrkA Receptor-Bearing, Pain-Sensing C-FibersMarco Marzulli, Justus b. Cohen, bonnie reinhart, William F. Goins, Joseph C. Glorioso

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112Potent Antifibrotic Effect on Human Dermal Fibroblasts and Keloid Spheroids by Adenovirus-Mediated Inhibition of Wnt and TGF-β Signalinghyo Min Ahn, Won Jai lee, youjin na, Jinwoo hong, Chae-ok yun

113Potent Therapeutic Efficacy of Neurotensin Receptor-Targeting and Extracellular Matrix-Degrading Oncolytic Adenovirus in an Orthotopic Pancreatic Tumor Modelyoujin na, Joung-Woo Choi, Dayananda Kasala, Jinwoo hong, eonju oh, yan li, Soo-Jung Jung, Sung Wan Kim, Chae-ok yun

114Replication-Defective HSV Vector Development for Targeted Gene Deliverybonnie reinhart, Daniela leronni, Justus b. Cohen, Joseph C. Glorioso

115Tumor Microenvironment-Targeting Hybrid Vector System Utilizing Oncolytic Adenovirus Complexed with pH-Sensitive and Bioreducible PolymerJung-Woo Choi, Dayananda Kasala, Chang yoon Moon, Soo-Jung Jung, Sung Wan Kim, Chae-ok yun

116Overcoming CAR-Dependency of Oncolytic Adenovirus via Adenoviral Fiber Modification with Vesicular Stomatitis Virus Glycoprotein Epitope for Enhanced Antitumor EfficacyA-rum yoon, Jinwoo hong, Chae-ok yun

117Development of Novel Cancer Therapy Combination of Ad-SOCS Gene Therapy and LAK Cell Immunotherapy for the Treatment of Prostate Cancerhiroki Saito, Koichi Kitagawa, Tetsuji naka, Satoshi Serada, Toshiro Shirakawa

Targeted Genome Editing I

118Characterization of Chromosomal Alterations Using a Zinc-Finger Nuclease Targeting the Beta-Globin Gene Locus in Hematopoietic Stem/Progenitor CellsJoseph long, Megan D. hoban, Caroline y. Kuo, beatriz Campo Fernandez, Aaron r. Cooper, Dianne lumaquin, roger P. hollis, Donald b. Kohn, Zulema romero

119Engineering a Self-Inactivating CRISPR System for AAV Vectorsbenjamin e. epstein, David V. Schaffer

120 (Moved to oral Session 411)Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9Kshitiz Singh, hanneke evens, Melvin rincón, nisha nair, Shilpita Sarcar, ermira Samara-Kuko, Marinee K. Chuah, Thierry VandenDriessche

121T Cell Receptor Modification by Highly Specific TALEN and CRISPR/Cas9Friederike Knipping, Karl Petri, eliana ruggiero, Mark osborn, Jakub Tolar, Manfred Schmidt, Christof von Kalle, richard Gabriel

122Seamless Correction by Donor DNA of a Class I CFTR Mutation Facilitated by a Double Nicking CRISPR/Cas9 in CF-iPSCsShingo Suzuki, Keisuke Chosa, Albert lee, Michael yezzi, hirofumi Kai, Dieter Gruenert

123Gene Editing as a Therapeutic Approach to Treat IPEX SyndromeMarianne Goodwin, Francesca Santoni de Sio, Daniel Dever, Matthew Porteus, Maria Grazia roncarolo, rosa bacchetta

124Therapeutic Correction of an LCA-Causing Splice Defect in the CEP290 Gene by CRISPR/Cas-Mediated Gene EditingMorgan l. Maeder, rina Mepani, Sebastian Gloskowski, Maxwell Skor, McKensie Collins, Shen Shen, Gregory Gotta, eugenio Marco, luis barrera, hari Jayaram, David bumcrot

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125Purging Latent HIV Infection by CRISPR-Mediated Viral ReactivationPrajit limsirichai, Thomas Gaj, David V. Schaffer

126Identification of Human Papillomavirus Entry Receptors with CRISPR-Cas9 Libraryxinping Fu, lihua Tao, Aiwu Jin, xiaoliu Zhang

127Lentiviral Protein Transduction for Tailored Genome Editing and Site-Directed Gene Insertionyujia Cai, rasmus o. bak, Anders laustsen, yan Zhou, Chenglong Sun, yonglun luo, Martin r. Jakobsen, Jacob G. Mikkelsen

128Targeting a High-Expression FVIII Transgene to Exogenous Locations in the Genome without Disrupting Endogenous Gene ExpressionJennifer M. Johnston, Matthew Porteus

129Does Transcription Influence AAV-Mediated Homologous Recombination?laura P. Spector, Mark A. Kay

130Purification of Large Scale mRNA Encoding ZFN Nucleases by dHPLC Technologyerica Giuliani, Claudia Piovan, Sergio bossi, Stefano Corna, Cinzia Scavullo, Monika Pema, Tiziano Di Tomaso, Pietro Genovese, Angelo lombardo, luigi naldini, Claudio bordignon, Gian Paolo rizzardi, Chiara bovolenta

131Chromatin-Dependent Loci Accessibility Affects CRISPR-Cas9 Targeting EfficiencyCiaran M. lee, Timothy h. Davis, harshavardhan Deshmukh, Gang bao

132Targeting the BTK Locus in Primary Human Hematopoietic Cells with TALENs and AAV Donor TemplateCourtnee Clough, yupeng Wang, iram F. Khan, Swati Singh, King hung, David J. rawlings

133Highly Efficient Homology-Driven Genome Editing in Human T Cells with Combined Zinc-Finger Nuclease mRNA and AAV6 Donor Delivery and Improved Efficiency Under Serum-Free ConditionsJianbin Wang, Joshua J. DeClercq, Samuel b. hayward, Patrick Wai-lun li, David A. Shivak, Gary lee, Michael C. holmes

134Gene Editing Approaches for Investigating Therapy-Resistance in Soft-Tissue SarcomaTim rath, Stefan Fröhling, Claudia Scholl, Priya Chudasama, hanno Glimm, Christof von Kalle, Ali nowrouzi, Manfred Schmidt

135Optimization of Dual-gRNA Lentiviral Vectors for Targeted Genomic DeletionsGiulia Pavani, Annalisa lattanzi, Fatima Amor, Chiara Antoniani, Fulvio Mavilio, Annarita Miccio, Mario Amendola

136Nuclease-Assisted Vector Integration (NAVI): A Robust Platform for Multiplexed Targeted Genome EngineeringAlexander brown, Wendy S. Woods, Pablo Perez-Pinera

137Analysis of Cas9-FokI and TALE-MutH Endonuclease Activity and Toxicity as Key Elements in the Development of a Gene Therapeutic Approach to Treat XLRPKnut Stieger, lennart Trimborn, Fei Song, birgit lorenz, Mert yanik

138Potential Therapeutic Treatment of Friedrich’s Ataxia Using Highly Specific Engineered Meganucleasesroshni Davey, Armin hekele, Jo Ann hux, Caitlin Turner, Wendy Sharer, Victor bartsevich, Ginger Tomberlin, Gene liau, Michael G. nicholson, Clayton W. beard, Jeff Smith, Derek Jantz

139Development of a Therapy for Duchenne Muscular Dystrophy Using Either TALEN of Cas9 ProteinsDaniel Agudelo, Joel rousseau, Jacques P. Tremblay

140Point Mutation Correction for ADA SCIDMiguel Calero-Garcia, Marlene Carmo, Adrian Thrasher, h bobby Gaspar

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141A Screening Platform for Generating Cas9 Mutants with Improved FunctionJeffrey M. Spencer, xiaoliu Zhang

142Functionalized Selenium Nanoparticles for mRNA DeliveryFiona C. Maiyo, Moganavelli Singh

Oligonucleotide Therapeutics I

143Durable Expression of TT-034 in Cynomolgus Monkey Hepatic and Cardiac Tissues without Long-Term Adverse Effects on Endogenous MicroRNA LevelsTin Mao, Shih-Chu Kao, David Suhy

144Development of Novel Dicer- and Ago2-Independent Small Hairpin RNAsShin-ichiro ohno, Karen itano, yuichirou harada, Koutaro Asada, Keiki oikawa, Mike Kashiwazako, Sudo Katsuko, Masakatsu Takanashi, norihiko ikeda, Masahiko Kuroda

145A Comparison of scAAV8-TT-034 Mediated Transduction and shRNA Expression in Human Liver Biopsy Samples versus a Chimeric Mouse Model with Humanized LiverShih-Chu Kao, Tin Mao, Terrie-Anne Cock, Peter roelvink, David Suhy

146Regulation of Inflammation in Cystic Fibrosis Lung Epithelial Cells by miR-155Motohiro Tsuchiya, Parameet Kumar, Sharmistha bhattacharyya, Swathi Kalurupalle, Shobha Vasudevan, Jennifer Martindale, Myriam Gorospe, roopa biswas

147 Abstract Withdrawn

148AptaMetrics: A Web-Based Aptamer Bioinformatics PlatformThomas bair, Julie S. Wertz, robert J. Miller, James Schappet, Paloma h. Giangrande, William h. Thiel

149In Vitro-Synthesized Sendai Virus RNA Fragments Induced Antitumor Immunity and Cancer Cell-Selective ApoptosisTomoyuki nishikawa, li-Wen liu, yasufumi Kaneda

150Further Development of an Allele-Specific Gene Silencing Strategy to Correct a Dominant-Negative Mutation Causing Collagen VI-Related Muscular DystrophyVéronique bolduc, Katherine Sizov, Apurva Sarathy, yaqun Zou, Carsten G. bönnemann

151 Abstract Withdrawn

152Tumor Inhibition by Using Chitosan:siRNA PDGFR-ß in Breast Cancer Model of RatJülide Akbuğa, Suna ozbas Turan, emine Salva, naziye ozkan, levent Kabasakal, Saadet Alan

153Third Generation Antisense Targeted to Double Homeobox Protein 4 (DUX4) Reduced DUX4 Expression and Improved Differentiation of FSHD MyoblastsSachchida nand Pandey, Kelly Murphy, hunain Khawaja, Mallikarjuna Putta, lakshmi bhagat, Sudhir Agrawal, yi-Wen Chen

154Applying Insights from Pri-miRNA Processing to shRNA Designxavier bofill-De ros, lisheng Dai, Shuo Gu

155Cancer-Specific Theranosis Using Quantum Dots and siRNA Co-Loaded in Micellar NanoparticlesSeong Jae Kang, hwa yeon Jeong, Min Woo Kim, in ho Jeong, Moon Jung Choi, young Myoung you, Chan Su im, yong Serk Park

156A Plasmid Manager, a Comprehensive Database for Archiving Plasmid DNA Sequenceemily J. heathward, Giun Sun, ron u. Domingo, Calvin A. Johnson, Christopher T. richie, brandon K. harvey

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Diabetes, Metabolic and Genetic Diseases I

157High-Field In Vivo Neuroimaging to Determine CNS Gene Therapy Outcome and Probe Disease PathomechanismDominic J. Gessler, Wei huang, Danning li, hongxia xu, Qin Su, Jean King, Constance Moore, Guangping Gao

158Cryptic ATG Removal from Synthetic Introns Increase the Therapeutic Efficacy of AAV Vector Mediated Gene TransferGiuseppe ronzitti, Fanny Collaud, Giulia bortolussi, Severine Charles, Patrice Vidal, Marcelo Simon Sola, Andres Muro, Federico Mingozzi

159Sustained Weight Loss and Altered Lipid Taste Sensitivity Induced by Modulation of Salivary PYY and Excendin-4 in MiceSean M. Crosson, Seth Currlin, Taimoor Khan, Cedric D. Dotson, Sergei Zolotukhin

160Successful Treatment of Neonatal Metachromatic Leukodystrophy Model Mice by Low Dose of Self-Complementary AAV Type9 Vector Expressing ASAnoriko Miyake, Koichi Miyake, Motoko yamamoto, Takashi Shimada, Takashi okada

161Novel Gene Transfer Strategy for Hemophilia A by Omental Implantaion of Autologous Endothelial Cell Sheethideto Matsui, Masahi noda, Midori Shima, rie utoh, Teruo okano, Mitsuhiko Sugimoto

162Functional Correction of Mucopolysaccharidosis I in Adult Mice by a Systemic rAAV9-IDUA Gene DeliveryAaron Meadows, ricardo Pineda, Marybeth Camboni, Kathryn Waligura, Darren Murrey, Douglas M. McCarty, haiyan Fu

163Hematopoietic Stem Cells Transplantation Can Normalize Thyroid Function in a Cystinosis Mouse Modelheloise P. Gaide Chevronnay, Virginie Janssens, Patrick Van Der Smissen, Celine J. rocca, xiao-hui liao, Samuel refetoff, Christophe e. Pierreux, Stephanie Cherqui, Pierre J. Courtoy

164Improvement of Gene Therapy for Wilson Diseaseoihana Murillo, Daniel Moreno, Cristina Gazquez, iñigo navarro-blasco, Jesus Prieto, ruben hernandez-Alcoceba, Gloria Gonzalez-Aseguinolaza

165Liver-Directed Gene Therapy for Murine Glycogen Storage Disease Type IBJoonhyun Kwon, Jun-ho Cho, young Mok lee, Goo-young Kim, Javier Anduaga, Janice Chou

166Lentiviral Hematopoietic Stem Cell Gene Therapy for Sjögren-Larsson Syndromeyoon-Sang Kim, irudayam Maria Johnson, Dana S’Aulis, William b. rizzo, Arthur W. nienhuis

167Genome Editing to Generate the First Mouse Model of Alpha-One Antitrypsin Deficiency, the Leading Cause of Genetic COPDFlorie borel, brynn Cardozo, Andrew Cox, Weiying li, Andrew hoffman, Mai elMallah, Christian Mueller

168Assaying Hepatic Correction Mediated by Varied AAV Vectors in a Knock-Out Transgenic Mouse Model of Methylmalonic Acidemia (MMA)brandon T. hubbard, randy J. Chandler, Charles P. Venditti

169Expression Studies of Ornithine Transcarbamylase in Liver Using Minicircle Vectors for Gene Therapy: Flag-Tagging of a Mitochondrial Enzyme and Stable Expression Under Control of an Endogenous Otc Promoter-Enhancerhiu Man Viecelli, Sereina Deplazes, Andrea Schlegel, Sharon Cunningham, ian Alexander, Johannes häberle, beat Thöny

170PhaseRx mRNA Technology Platform Uses SMARTT Polymer Technology® to Target and Deliver mRNA to the Liver and Treat a Urea Cycle Disorder in a Mouse Disease ModelMary Prieve, Allen li, Alex baturevych, eric bell, Teri blevins, Anna Galperin, Pierrot harvie, Jean-rene ella Menye, Sean Monahan, Amber Paschal, Maher Qabar, Debashish roy, Matt Waldheim, Mike houston

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171Quantification of Human FVIII and Estimation of Its Molecular Weight: Potential Applications in Gene Therapy for Hemophilia Arajeev Mahimkar, barrie Carter, Gordon Vehar

Cardiovascular and Pulmonary Diseases

172B-Type Natriuretic Peptide Gene Therapy as a Novel Early Treatment for Familial Hypertrophic CardiomyopathyCassandra A. bongers, Sara J. holditch, Claire A. Schreiber, John P. Konhilas, John C. burnett, yasuhiro ikeda

173Electroporation-Mediated FER Gene Delivery in the Resolution of Severe Gram Negative PneumoniaVladislav Dolgachev, rebecca Goldberg, Suresh Madathilparambil, Krishnan raghavendran, Mark r. hemmila, Mark Johnson, David Machado-Aranda

174Post-Deployment Modifications of Stent with Endothelial Cellsilia Fishbein, ivan S. Alferiev, richard F. Adamo, David T. Guerrero, yanqing Tang, Michael Chorny, robert J. levy

175 Abstract Withdrawn

176Novel Molecules That Enhance Adenovirus Transduction of the Airway Epithelium: Reinvigorating Adenovirus-Mediated Gene TherapyKatherine J. D. A. excoffon, Priyanka Sharma, James M. readler, Mahmoud S. Alghamri

177Cross-Talk Between Human Relaxin Gene Therapy and Infarct-Related Coronary Arteryyoung Sook lee, Joung-Woo Choi, Jung-eun oh, Sung Wan Kim, Chae-ok yun

178Effect of Heme Oxygenase-1 Overexpression on DNA Synthesis of Adult Cardiac Progenitor Cells Under HypoxiaDing li, landys Z. Guo, Qinghui ou, Wei xie, roberto bolli, Qianhong li

179Helper-Dependent Adenovirus-Mediated Gene Transfer of an LDL Receptor/Transferrin Chimeric Protein Reduces Aortic Atherosclerosis in LDL Receptor-Deficient Miceeleonora leggiero, Giuseppe labruna, laura iaffaldano, Martina esposito, Dalila Savoia, lucia Sacchetti, lucio Pastore

180The Role of the Pyruvate Dehydrogenase Kinase 4 (PDK4) Gene Mutation in the Development of Dilated Cardiomyopathy in Dobermanpinschers and Potential Application of Cardiac Gene Therapyluiz bolfer, Amara h. estrada, brandy Winter, Kathryn Taggart, Francisco lourenço, naohiro Terada, barry J. byrne, Thomas J. Conlon, Christina A. Pacak

181Optimization of In Vivo Delivery of Baculovirus Gene Therapy Vectors for the Treatment of Alpha-1 Antitrypsin Deficiencylaura van lieshout, Jondavid de Jong, Jacob van Vloten, Sarah K. Wootton

182Effects of Inflammation-Mediated Autophagy of Human Placental Mesenchymal Stem Cells of Fetal Origin on Their Immunomodulatory Properties and Therapeutic Potential in Bleomycin-Induced Pulmonary Fibrosis Modelsxiaoming liu, yongzhao Zhu, xueyun liang, Fei han, Jun Wei

183Elucidation of Alpha-1 Antitrypsin Levels in Human Infant Blood Samplesheather S. loring, Alisha M. Gruntman, Qiushi Tang, Christian Mueller, Terence r. Flotte

Gene Therapy for Neurosensory Diseases

184PGN-503, a Herpes Simplex Virus Based Vector Expressing Neurotrophin-3, Prevents and Reverses Neuropathy in a Mouse Model of Paclitaxel-Induced Peripheral NeuropathyJames r. Goss, David Krisky, Karen bouch, Mark o’Malley, Sarah Coghlan, James Wechuck

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185Safety Study by Validated Immunoassays in a Phase III Study of Subjects with Inherited Retinal Dystrophy Due to Mutations in the Gene Encoding Human Retinal Pigment Epithelium-Specific Protein 65 (RPE65) Injected with Adeno-Associated Viral VectorsDaniel J. hui, yifeng Chen, Thomas Antrilli, yun liu, romuald Corbau, Stephen r. russell, Albert M. Maguire, Jean bennett, Katherine A. high

186Translating an Optogenetic Gene Therapy Approach for Treatment of Neuropathic Pain in HumansChris Towne, Jesus Aguado, Annie Arguello, Claire Discenza, Tim Galfin, Stephan Gehrke, Sabeena Khan, Michael Kaplitt

187TFEB-Mediated Clearance of the Lipofuscin Fluorophore A2Eivana Trapani, elisabetta Toriello, renato Minopoli, Alberto Auricchio

188 (Moved to Poster Session ii after Abstract 376)AAV Mediated Gene Therapy to Modulate Neurotropic Factors in the Retina and in Neuronal Cells in Culturenitin Chitranshi, roshana Vander Wall, yogita Dheer, Stuart l. Graham, Vivek Gupta

189Therapeutic Gene Transfer as a Treatment Option for Age-Related Macular Degenerationerik Wielechowski, Angelica Medina-Jaszek, omua Ahonkhai, Muhammad Arif, Jean bennett, Albert Maguire, Anna Tretiakova, James M. Wilson

Cancer-Immunotherapy, Cancer Vaccines I

190 Abstract Withdrawn

191Development of an Effective Cancer Vaccine Platform Using Attenuated Salmonella to Deliver Recombinant Tumor-Associated Antigensxin xu, Michael S. Wood, Suhrab Kurbanov, linjie Guo, xiuhua Gao, James e. Galen, leonid S. Metelitsa

192Deep Phenotyping of Manufactured Enhanced-Affinity NY-ESO-1-Specific T Cells Reveals a Pattern of Effector and Memory Programming That Correlates with Clinical Outcome in Observed Cancer Indicationsluca Melchiori, Daniel e. lowther, Gwendolyn K. binder-Scholl, Daniel D. Williams, yoav Peretz, Marylene Fortin, Willian D. Tap, Aaron P. rapoport, Crystal l. Mackall, bent K. Jakobsen

193Efficacy and Safety of Immunotherapy with Chimeric Antigen Receptor Targeting WT1 and HLA-A24:02 pMHC Complexhiroaki ikeda, yasushi Akahori, yuki orito, yoshihiro Miyahara, yasunori Amaishi, Sachiko okamoto, Junichi Mineno, Kazutoh Takesako, hiroshi Fujiwara, Masaki yasukawa, hiroshi Shiku

194Adenoviruses Armed with TNFα and IL-2 Successfully Enable Adoptive T-Cell TherapyAino Kalervo, Mikko Siurala, riikka havunen, Suvi Parviainen, Simona bramante, Dipongor Saha, Siri Tähtinen, Markus Vähä-Koskela, Michael behr, Dirk nettelbeck, Anja ehrhardt, Akseli hemminki

195Allogeneic CD19-CART Therapy to the Rescue of a Pediatric Patient with Relapsed and Refractory Acute B Lymphoblastic Leukemialung-Ji Chang, Jian-Ping Zhang, yu-Chen liu, lujia Dong, Tong Wu, Dao-Pei lu

196Nordihydroguaiaretic Acid (NDGA) Improves Function of Antigen-Specific T-Cells Stimulated by Capsid-Optimized AAV6 VectorsDaniel Zhang, Chen ling, George Aslanidi

197Balancing Anti-Tumor Efficacy with Local Inflammatory Toxicity for the Treatment of Diffuse Intrinsic Pontine Glioma and Other Brain TumorsMatthew Schuelke, laura evgin, Tim Kottke, Jill Thompson, Christopher Driscoll, elizabeth ilett, Julia Cockle, Amulya nageswararao, richard bram, Alan Melcher, richard Vile

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198CD4+ IL13Rα2-Specific CAR T Cells Exhibit Potent Effector Function Against GlioblastomaDongrui Wang, renate Starr, brenda Aguilar, Alfonso brito, brenda Chang, Aniee Sarkissian, behnam badie, Michael e. barish, Stephen J. Forman, Christine e. brown

199Genetic Engineering of Hematopoietic Stem Cells to Generate Invariant Natural Killer T CellsDrake J. Smith

200Generation of CAR-T Cells Lacking T Cell Receptor and Human Leukocyte Antigen Using Engineered MeganucleasesChristina Pham, Aaron Martin, Jeyaraj Antony, Daniel Macleod, Audrey brown, Michael nicholson, Jo Ann hux, Caitlin Turner, Wendy Sharer, bruce McCreedy, Victor bartsevich, Ginger Tomberlin, Janel lape, Jeff Smith, Derek Jantz

201Modulation of the Antitumor Immune Response Using Semireplication-Competent VSV Armed with Immunostimulatory TransgenesJohanna reul, Christian J. buchholz, Alexander Muik

202 Abstract Withdrawn

203Shortened T Cell Culture with IL-7 and IL-15 Provides the Most Potent Chimeric Antigen Receptor (CAR)-Modified T Cells for Adoptive ImmunotherapySaba Ghassemi, Felipe bedoya, Selene nunez-Cruz, Carl June, Jos Melenhorst, Michael Milone

204HER2-Specific Chimeric Antigen Receptor T Cells for the Treatment of Breast-to-Brain MetastasisJohn P. Murad, Dileshni Tilakawardane, brenda Aguilar, Anthony Park, ethan Gerdts, Aniee Sarkissian, Alfonso brito, Josh neman, renate Starr, brenda Chang, Wen-Chung Chang, Sarah Wright, behnam badie, Stephen J. Forman, Christine e. brown, Saul J. Priceman

205Cytotoxic and Immunotherapeutic Effects of Toca 511 and 5-Fluorocytosine in an Intraperitoneal Model of Metastatic Colorectal CancerKader yagiz, Daniel Mendoza, Maria rodriguez-Aguirre, Fernando lopez espinoza, Carlos e. ibañez, harry e. Gruber, Douglas J. Jolly, Joan M. robbins

206Determining the Effect of Endogenous PD-1 Expression on the Co-Stimulatory Potential of the PD1:CD28 ChimeraMegan e. Keys, Jonathan Arroyo, bridgette Kielhack, Megan Prosser

Cancer-Targeted Gene and Cell Therapy I

207Variable Lymphocyte Receptors Enable Development of Chimeric Antigen Receptors for the Treatment of T-Cell MalignanciesSunil S. raikar, Daniel Tylawsky, robert Moot, lauren Fleischer, David McCarty, Christopher Doering, h. Trent Spencer

208Neural Stem Cell Mediated Oncolytic Virotherapy for Ovarian CancerAsma Abdul Majid, rachael Mooney, Megan Gilchrist, Maciej lesniak, Karen Aboody

209Stem Cell/Nanoparticle Constructs for Targeted Ovarian Cancer TherapyPengpeng Cao, revathiswari Tirughana, ugochi nwokafor, Soraya Aramburo, linda Flores, Jacob berlin, Karen S. Aboody

210High Expression of Second Generation CD19 CAR with a 4-1BB Costimulatory Domain from a Retroviral Vector Impairs CAR T Cell Expansion by Enhancing Fas-Mediated ApoptosisDiogo Silva, Malini Mukherjee, Madhuwanti Srinivasan, Jordan S. orange, Malcolm K. brenner, Maksim Mamonkin

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211 (Moved to Poster Session iii after Abstract 680)Combination of Forced Transduction of P53 and an Agent That Blocks MDM2-p53 Interactions Produces Synergistic Cytotoxicity on Mesothelioma Defective of the INK4A/ARF RegionMasatoshi Tagawa, Takao Morinaga, Zhihan li, Thao T. T. nguyen, boya Zhong, Shuji Kubo, ikuo Sekine, yuji Tada, Koichiro Tatsumi, hideaki Shimada, Kenzo hiroshima

212Lentiviral Insertional Mutagenesis Helps to Uncover the Mechanisms of Resistance to AZD9291 and CO-1686 in EGFR-Mutant Lung AdenocarcinomaValentina Pirazzoli, Giulio Spinozzi, erika Tenderini, Fabrizio benedicenti, Andrea Calabria, eugenio Montini

213Detection of Integration Sites of CD19 Chimeric Antigen Receptor Gene in a Non-Viral Gene Transfer SystemMotoharu hamada

214Specific Targeting of IL-13 Receptor alpha 2 Expressing Breast Cancer Cells by Paramyxovirus-Pseudotyped Lentiviral Vectors Displaying IL-13Anna r. Kwilas, Wu ou, Michael P. Marino, Takele Argaw, Akiko Suzuki, bharat Joshi, Syed r. husain, raj K. Puri, Jakob reiser

215The Use of miR-122 and Its Target Sequence in Adeno-Associated Virus-Mediated Cytotoxic Gene TherapyDaniel Zhang, Jun li, Zifei yin, Georgy Aslanidi, Arun Srivastava, Changquan ling, Chen ling

216Exploiting Redox-Based Vulnerabilities in Field Cancerization by Targeting Cancer Associated FibroblastsJeremy Soon Kiat Chan, Ming Jie Tan, nguan Soon Tan

217Combinatorial Anti-Angiogenic Gene Therapy in a Human Malignant Mesothelioma ModelShuji Kubo, yuki Mawatari-Furukawa, noriyuki Kasahara

218Monitoring ER Stress Activation of the ATF6 Pathway Using Nanoluciferaseemily S. Wires, Doug howard, Mark J. henderson, xiaokang yan, Kathleen A. Trychta, emily J. heathward, yajun Zhang, Molly lutrey, Christopher richie, brandon K. harvey

219Investigation of the Role of Decorin Gene (DCN) in Glioblastoma and Cancer Stem Cells (CSCs)Jun-Ming han, hsin-i Ma

Hematologic & Immunologic Diseases I

220AAV-Mediated miR122 Overexpression Rescues Hepatic Inflammatory Response Induced by Acute Iron-OverloadMin li, yuxiao Tang, lusha Wu, Fengfeng Mo, xin Wang, hongxia li, ruirui Qi, Arun Srivastava, Chen ling

221Staurosporine Increases Lentiviral Transduction of Human CD34+ CellsMelissa bonner, Gretchen lewis, lauryn Christiansen, Dakota Campbell, Christopher Tipper, Amanda hamel, Seema Shah, holly horton, olivier negre, Gabor Veres, Philip Gregory

222The LCR-Free γ-Globin Lentiviral Vector Combining Two HPFH Activating Elements Corrects Murine Thalassemic Phenotype In VivoPanayotis Antoniou, eleni P. Papanikolaou, Maria Georgomanoli, ekati Drakopoulou, nicholas P. Anagnou

223DURATION OF FACTOR IX EXPRESSION IN MACAQUES FOLLOWING AAV8-MEDIATED LIVER TRANSDUCTIONhiroaki Mizukami, Tsukasa ohmori, ryosuke uchibori, yasushi Saga, Masashi urabe, Akihiro Kume, yoichi Sakata, Keiya ozawa

224A Gene Therapy Clinical Study of a Patient with X-Linked Chronic Granulomatous DiseaseToshinao Kawai, Kohji okamura, Mari yagita, Fumihiro Goto, yumiko nakazawa, Toru uchiyama, Kazuhiko nakabayashi, hiroyuki nunoi, harry Malech, Masafumi onodera

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225Preserving CD4+ T-Cells Phenotype and Function Upon Ex Vivo Lentiviral Transductionisabelle hmitou, Amani ouedrani, lounes Djerroudi, Cécile roudaut, laure Caccavelli, Tayebeh S. Soheili, isabelle André-Schmutz, Marina Cavazzana, Fabien Touzot

226Lentiviral Vector Gene Therapy Protects XCGD Mice from Acute Staphylococcus aureus Pneumonia and Inflammatory ResponseGiada Farinelli, raisa Jofra hernandez, Alice rossi, Serena ranucci, Francesca Sanvito, Maddalena Migliavacca, Chiara brombin, Aleksandar Pramov, Clelia Di Serio, bernhard Gentner, Alessandra bragonzi, Alessandro Aiuti

227A Dose-Escalating Preclinical Study to Determine the Efficacy, MED, and Safety of a Clinical Candidate Vector in a Mouse Model of Hemophilia Blili Wang, John White, Zhenning he, Tamara Goode, Deirdre McMenamin, Christine Draper, Cassandra brown, edward A. Chroscinski, lisa Kattenhorn, Gwen Wilmes, reed Clark, Samuel Wadsworth, James M. Wilson

228Characterization of Hematopoietic Progenitors from Pyruvate Kinase Deficient (PKD) Patients and Transduction of PKD CD34+ Cells with a Therapeutic Lentiviral VectorSusana navarro, Maria Garcia-Gomez, Sergio lopez-Manzaneda, rebeca Sanchez-Dominguez, Maria Garcia-bravo, omaira Alberquilla, Maria M. Mañu, Manuela Muriel-ramos, Joan ll. Vives-Corrons, Julian Sevilla, Juan A. bueren, Jose C. Segovia

229PGE2 Increases Lentiviral Vector Transduction Efficiency of Human HSCGarrett heffner, Melissa bonner, Dakota Campbell, lauryn Christiansen, F. John Pierciey, Wen Zhang, Gretchen lewis, yegor Smurnyy, Amanda hamel, Seema Shah, holly horton, byoung ryu, Kendrick Goss, olivier negre, Gabor Veres, Christopher horvath, Mitchell Finer, Philip Gregory

230Designing High-Titer Lentiviral Vectors for Gene Therapy of Sickle-Cell DiseaseFabrizia urbinati, Valentina Poletti, beatriz Campo Fernandez, roger P. hollis, Colin Koziol, Michael l. Kaufman, Devin brown, Annarita Miccio, Michael Antoniou, Donald b. Kohn, Fulvio Mavilio

231Mixed Chimerism After Allogeneic Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: Preliminary Results on Peripheral Blood Sorted Subpopulations and Erythroid ProgenitorsAlessandra Magnani, laure Caccavelli, Mathieu Fusaro, Jeremy Magalon, elisa Magrin, emmanuelle Six, Fabien Touzot, Chloé Couzin, Jean Antoine ribeil, Cécile Arnaud, Corinne Pondarré, Annie Kamdem, Françoise bernaudin, Jean Paul Vernant, eliane Gluckman, Dominique bories, Marina Cavazzana

232Generalized Entropy Based Clonal Diversity Estimation of TCR RepertoireSaira Afzal, Shahzad Ahmad, richard Gabriel, eliana ruggiero, Christof von Kalle, Manfred Schmidt, raffaele Fronza

233The Efficacy of Megakaryocytic Progenitor Products Generated from Cord Blood in Acute Myeloid Leukemia Patients: A Before-After Phase 2 Trialxuetao Pei

Cell Therapies I

234Efficient Generation of Stable Genetically Modified Human iPSC-Derived Macrophages for Innovative Gene and Cell Therapeutic StrategiesMania Ackermann, uta Müller-Kuller, Sylvia Merkert, ulrich Martin, Manuel Grez, Axel Schambach, Thomas Moritz, nico lachmann

235Improved Ex Vivo Gene Therapy Using Highly Purified Hematopoietic Stem and Progenitor Cellserika Zonari, oriana Meo, Samantha Scaramuzza, eugenio Montini, Giuliana Ferrari, luigi naldini, bernhard Gentner

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236Role of P53 on T Lymphopoiesis from Human Embryonic Stem Cellshiroshi Kohara, Jiyuan liao, Shohei Miyamoto, yoko nagai, Tomotoshi Marumoto, Kenzaburo Tani

237 Abstract Withdrawn

238 Abstract Withdrawn

239Induction of Allograft Immune Tolerance with Bioengineered Thymus OrganoidsAsako Tajima, isha Pradhan, ipsita banerjee, William A. rudert, Massimo Trucco, yong Fan

240Lenti-BMP2/GFP Gene Therapy Enhance the Chondrogenic and Osteogenic Differentiation of Human Muscle Derived Stem Cells In Vitroxueqin Gao, ying Tang, Aiping lu, bing Wang, xiaohong bi, Guijin lv, Johnny huard

241An Efficient Construction of Lentiviral Vectors That Identify and Eliminate Tumorigenic Cells in Pluripotent Stem CellsKanako ide, Kaoru Mitsui, Ken-ichiro Kosai

242Genetically Engineered Mesenchymal Stem Cell Spheroids for Brain Tumor TherapySmruthi Suryaprakash, hon Fai Chan, Juli rodriguez, Shawn hingtgen, Kam W. leong

243The Use of Thymidine Kinase Mutants as a Safeguard Switch for IPS CellsMasashi urabe, ryosuke uchibori, Akihiro Kume, hiroaki Mizukami, Keiya ozawa

244Development and Qualification of Assays for Human Embryonic Stem Cell (hESC)-Derived Cardiomyocytes Intended for Clinical UseJennil Patel, Wei Dang, Aparna Krishnan, Derek Kong, David hsu, larry Couture

245MicroRNA Expression in Bone Marrow-Derived Human MSCsian h. bellayr, Jennifer G. Catalano, raj K. Puri

246Characterization of Peripheral Cardiac ProgenitorsJosé r. navarro-betancourt, Salomón hernández, Felipe Masso, luis A. lara-Martínez, Alejandra Contreras-ramos, Andrés r. Martinez-Calderón, Victor M. Arenas, José l. Aceves-Chimal

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Thursday, May 5, 2016Scientific Symposium 201Clinical Trials Spotlight Symposium8:00 AM - 10:00 AMroom: Thurgood Marshall ne

247 8:00 AMPhase I Study of Second Generation Chimeric Antigen Receptor-Engineered T Cells Targeting IL13Rα2 for the Treatment of GlioblastomaChristine e. brown, renate Starr, lihong Weng, Darya Alizadeh, Jamie r. Wagner, Julie Kilpatrick, Todd l. harshbarger, Massimo D’Apuzzo, Julie A. ressler, Michael C. Jensen, Jana Portnow, Michael e. barish, Stephen J. Forman, behnam badie

248 8:20 AMResults of a Phase 1 and 2a Trial on rAAV.sFlt-1 in Treatment of Wet Age-Related Macular DegenerationChooi-May lai, Aaron l. Magno, Martyn A. French, Samuel b. barone, Steven D. Schwartz, Mark S. blumenkranz, Mariapia A. Degli-esposti, ian J. Constable, elizabeth P. rakoczy

249 8:40 AMPreliminary Results of a Phase 1, Open-Label, Safety and Tolerability Study of a Single Intravitreal Injection of AAV2-sFLT01 in Patients with Neovascular Age-Related Macular DegenerationAbraham Scaria, Annaig lehalpere, Annie Purvis, Cheryl Delacono, Seng Cheng, Samuel Wadsworth, Peter Campochiaro, Jeff heier, ronald buggage

250 9:00 AMA Phase 2/3 Study of the Efficacy and Safety of Ex Vivo Gene Therapy with Lenti-DTM Lentiviral Vector for the Treatment of Cerebral AdrenoleukodystrophyFlorian S. eichler, Christine Duncan, hernan Amartino, razina Aziz-bose, André baruchel, Colleen Dansereau, Jean-hugues Dalle, h. bobby Gaspar, Paul Gissen, Donald b. Kohn, Jerome larghero, Troy C. lund, Weston P. Miller, Patricia l. Musolino, Tara o’Meara, Paul J. orchard, Asif M. Paker, Ami J. Shah, esther Shamir, Gerald V. raymond, raman Sankar, nicholas J. C. Smith, Adrian J. Thrasher, Patrick Aubourg, David A. Williams

Oral Abstract Session 240Evolving Better Parvoviral Vectors for Gene Therapy4:00 PM - 5:45 PMroom: Thurgood Marshall ne

251 4:00 PMDefining Conserved Structural Components of the AAV Capsid That Enable Tissue-Specific Transduction Following Systemic AdministrationJayme K. Warischalk, Samuel C. eaton, r. Jude Samulski

252 4:15 PMUsing Cre-Dependent In Vivo Selection to Identify AAV Variants That Enable Efficient and Widespread Gene Transfer to the Adult Central Nervous Systembenjamin e. Deverman, Piers l. Pravdo, bryan P. Simpson, Sripriya ravindra Kumar, yicheng luo, Ken y. Chan, Abhik banerjee, Wei-li Wu, bin yang, nina huber, Sergiu P. Pasca, Viviana Gradinaru

253 4:30 PMExpanded Packaging Capacity of AAV by Lumenal Charge AlterationMatthew Tiffany, Mark A. Kay

254 4:45 PMNew Chimeric Gene Therapy Vectors Based on Four Different Mammalian BocavirusesJulia Fakhiri, Marc Schneider, Shweta Kailasan, Michael Meister, Mavis A. McKenna, Ziying yan, Jianming Qiu, Dirk Grimm

255 5:00 PMA Method for Developing More Potent AAV Capsids by Prolonging the Vector Half-Lives in the Blood CirculationMiyuki Kimura, Kei Adachi, hiroyuki nakai

256 5:15 PMA Novel Rationally Designed AAV Capsid Yields a Potent Neurotropic Gene Therapy Vectorels henckaerts, Julie Tordo, nuria Palomar, Jahedur rahman, Patrick Aldrin-Kirk, Mark basche, Antonette bennett, Claire o’leary, robin Ali, Mavis Agbandje-McKenna, brian bigger, Ahad A. rahim, Tomas björklund, Simon Waddington, r. Michael linden

257 5:30 PMSelection of Next Generation AAV Gene Therapy Vectors for Specific and Precise Gene Deliveryrobert Muench, Mark Kay

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Oral Abstract Session 241Oligonucleotide Therapeutics I4:00 PM - 5:45 PMroom: Washington 3

258 4:00 PMPhase I/IIa Study of TT-034, a DNA-Directed RNA Interference (ddRNAi) Agent Delivered as a Single Administration for the Treatment of Subjects with Chronic Hepatitis C Virus (HCV)Keyur Patel, Georgina Kilfoil, David l. Wyles, Susanna naggie, eric lawitz, Stephen bradley, Per lindell, David Suhy

259 4:15 PMTargeted Gene Silencing by U1 Adaptor Oligonucleotides in Preclinical Models of Parkinson’s Disease and Huntington’s Diseaseeric richfield, Kavita Prasad, Mark brenneman, rafal Goraczniak, Samuel Gunderson

260 4:30 PMBB-HB-331, a DNA-Directed RNA Interference (ddRNAi) Agent Targeting Hepatitis B Virus (HBV), Can Effectively Suppress HBV In Vitro and In VivoTin Mao, Shih-Chu Kao, Terrie-Anne Cock, Kermit Zhang, Michael Graham, Peter roelvink, David Suhy

261 4:45 PMGain-of-Function Effect Augments Therapeutic Efficacy of CpG-STAT3 Anti-Sense Oligonucleotide Against Castration-Resistant Prostate CancersDayson Moreira, xingli Zhao, haejung Won, Priyanka Duttagupta, Summanta Pal, Marcin Kortylewski

262 5:00 PMTherapeutic Suppression of the KRAS-MYC Oncogenic Axis in Human Pancreatic Cancer Xenografts with U1 Adaptor Oligonucleotide / RGD Peptide ConjugatesDarren Carpizo, Ashley Tsang, xin yu, lan yi, Crissy Dudgeon, Kristen Donohue, Mark brenneman, rafal Goraczniak, Samuel Gunderson

263 5:15 PMNuclease-Activated Oligonucleotide Probes for the Rapid and Robust Detection of Breast Cancer Circulating Tumor Cells (CTCs)David D. Dickey, Sven Kruspe, Kevin T. urak, William h. Thiel, Karen C. Clark, elliot burghardt, Justin P. Dassie, Alexandra Thomas, James o. Mcnamara, Paloma h. Giangrande

264 5:30 PMIn Vivo DNA-Monoclonal Antibody (DMAb) Gene Delivery Protects Against Lethal Bacterial and Viral Challenges in MiceAmi Patel, Seleeke Flingai, Sarah T. C. elliott, Trevor Smith, Megan C. Wise, Stephanie ramos, Jian yan, Katherine Schultheis, Amelia A. Keaton, Daniel h. Park, niranjan Sardesai, Muthumani Karuppiah, David b. Weiner

Oral Abstract Session 242Gene Therapy for Neurosensory Diseases4:00 PM - 5:45 PMroom: Thurgood Marshall SW

265 4:00 PMBrain Pathways Enabling Vision in LCA Patients Before and After Gene TherapyManzar Ashtari, Philip Cook, hui Zhang, laura Cyckowski, elena nikonova, Gloria young, Kathleen Marshall, James Gee, David leopold, Chris baker, Albert Maguire, Jean bennett

266 4:15 PMIn Vivo Rod Photoreceptor Reprogramming Using AAV-Delivered CRISPR/Cas9 Rescues Retinal DegenerationWenhan yu, Suddhasil Mookherjee, Jung-Woong Kim, Suja hiriyanna, yasaman Ataeijannati, xun Sun, lijin Dong, Tiansen li, Anand Swaroop, Zhijian Wu

267 4:30 PMPhase I Gene Therapy Preliminary Clinical Results for Treatment of ND4 Leber Hereditary Optic Neuropathy with rAAV2-2-ND4Scott uretsky, Catherine Vignal, Samuel bidot, Serge Fitoussi, Anne Galy, Sandrine Meunier, roxane noel, Céline bouquet, nitza Thomasson, Jean-Philippe Combal, José A. Sahel

268 4:45 PMOptogenetic Engineering of Retinal Ganglion Cells with AAV2.7m8-ChrimsonR-tdTomato (GS030-DP) Is Well Tolerated and Induces Functional Responses to Light in Non-Human PrimatesAnne M. Douar, Celine bouquet, Didier Pruneau, Joel Chavas, Deniz Dalkara, Jens Duebel, ryad benosman, Guillaume Chenegros, Serge Picaud, José Sahel, nitza Thomasson

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269 5:00 PMNovel Synthetic AAV Efficiently Transduces Neurosensory Hair Cells in the Cochlealukas landegger, bifeng Pang, Sarah Wassmer, Sarah Gluck, Alice Galvin, Konstantina M. Stankovic, Jeff r. holt, luk h. Vandenberghe

270 5:15 PMOptogenetic Activation of Remnant Cone Cells Can Restore Ganglion Cell Light Responses in a Canine Model of Retinitis PigmentosaPavitra S. ramachandran, Sergei nikonov, Keirnan Willett, rachel huckfeldt, Arkady lyubarsky, Jessica Morgan, Tomas Aleman, Albert Maguire, Jean bennett

271 5:30 PMStrategies Based on NexGen AAV2 to Improve AKT3-Induced CNS Axon Regeneration In Vivoyang hu, linqing Miao, liu yang, haoliang huang, Feisi liang, Chen ling

Oral Abstract Session 243Cancer-Immunotherapy, Cancer Vaccines I4:00 PM - 5:45 PMroom: Washington 4

272 4:00 PMRapid Identification of Tumor- and Neoantigen-Reactive T-Cell Receptors for Personalized ImmunotherapyAnna Pasetto, Alena Gros, Paul F. robbins, Drew C. Deniger, Todd D. Prickett, Maria r. Parkhurst, rodrigo Matus-nicodemos, Daniel C. Douek, Jared Gartner, Katarzyna Trebska-McGowan, Jessica Crystal, Steven A. rosenberg

273 4:15 PMGenome Editing Using CRISPR-Cas9 to Increase the Therapeutic Index of Antigen-Specific Immunotherapy in Acute Myeloid LeukemiaMiriam y. Kim, Saad S. Kenderian, Daniel Schreeder, Michael Klichinsky, Miroslaw Kozlowski, olga Schestova, Marco ruella, Saar Gill

274 4:30 PMOne-Step Generation of Universal CAR T CellsJustin eyquem, Jorge Mansilla-Soto, Ashlesha odak, Michel Sadelain

275 4:45 PMOptimization of IL13Rα2-Specific CAR T Cells for Clinical Development Using Orthotopic Human Glioblastoma Models in NSG Micebrenda Aguilar, Aniee Sarkissian, Alfonso brito, renate Starr, brenda Chang, yubo Zhai, lihong Weng, Michael barish, behnam badie, Stephen Forman, Christine brown

276 5:00 PMLong-Term Relapse-Free Survival of Patients with Acute Myeloid Leukemia (AML) Receiving a Telomerase-Engineered Dendritic Cell ImmunotherapyJane S. lebkowski, hanna J. Khoury, robert h. Collins, William blum, Patrick Stiff, edward Wirth, Kevin nishimoto, John DiPersio

277 5:15 PMSmall Molecule-Regulated Antigen Recognition System for Inducible T Cell Targeting of Cancer CellsWai-hang leung, Michael Certo, holly horton, iulia Diaconu, Tracy Vandenberg, Jordan Jarjour, Alexander Astrakhan

278 5:30 PMNext-Generation Non-Viral Gene Transfer to Redirect T-Cell Specificityharjeet Singh, Mary helen huls, Matthew J. Figliola, ling Zhang, Tiejuan Mi, Sourindra Maiti, Gabrielle romain, Simon olivares, Shihuang Su, lenka V. hurton, navin Varadarajan, laurence J. n. Cooper, Partow Kebriaei

Oral Abstract Session 244Hematologic & Immunologic Diseases I4:00 PM - 5:45 PMroom: Washington 5-6

279 4:00 PMClinical Outcomes of Gene Therapy with BB305 Lentiviral Vector for Sickle Cell Disease and β-ThalassemiaMarina Cavazzana, Jean-Antoine ribeil, emmanuel Payen, Fabien Touzot, bénédicte neven, Francois lefrere, Felipe Suarez, elisa Magrin, yves beuzard, Stany Chretien, Philippe bourget, Fabrice Monpoux, Corinne Pondarré, Pablo bartolucci, Manfred Schmidt, Christof von Kalle, laura Sandler, Sandeep Soni, Michaela Semeraro, Wassim el nemer, olivier hermine, Mariane de Montalembert, Stéphane blanche, Salima hacein-bey-Abina, Philippe leboulch

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280 4:15 PMLentiviral-Mediated Gene Therapy Restores B Cell Homeostasis and Tolerance in Wiskott-Aldrich Syndrome PatientsMaria Carmina Castiello, Francesca Pala, henner Morbach, Jean-nicolas Schickel, Samantha Scaramuzza, nicolas Chamberlain, neil romberg, Francesca Ferrua, Fabio Candotti, Mirjam van der burg, luigi naldini, Alessandro Aiuti, eric Meffre, Marita bosticardo, Anna Villa

281 4:30 PMEnforced Expression of a Mutant HMGA2 Gene Leads to Competitive Expansion and High Level Marking of Long-Term Hematopoietic Stem Cells in Transplanted Nemestrina MacaquesMelissa A. bonner, Sheng Zhou, yong-Dong Wang, Zhijun Ma, lance Palmer, brian P. Sorrentino

282 4:45 PMCircumventing Hematopoietic Toxicities and Reversing Sickle Phenotype by Lineage-Specific BCL11A Knock Down and Γ-Globin InductionChristian brendel, Swaroopa Guda, raffaele renella, Daniel e. bauer, Matthew C. Canver, young-Jo Kim, Matthew M. heeney, Denise Klatt, Jonathan Fogel, Michael D. Milsom, Stuart h. orkin, richard i. Gregory, David A. Williams

283 5:00 PMIn Vivo Selection of MGMT(P140K) Gene Modified Hematopoietic Cells in the Nonhuman Primate Unmasks a Dormant Pool of Repopulating Clones with Progenitor Cell OntologyJennifer e. Adair, lauren Schefter, Daniel humphries, Kevin G. haworth, hans-Peter Kiem

284 5:15 PMLong-Term Therapeutic Immune Reconstitution in XSCID Canine Model via In Vivo Foamy Virus Delivery of Common Gamma ChainFrieda Chan, olivier humbert, Christopher burtner, Daniel humphrys, Jennifer Adair, Grant Trobridge, Patricia o’Donnell, nicholas hubbard, Troy Torgerson, Andrew Scharenberg, David rawlings, Peter J. Felsburg, hans-Peter Kiem

285 5:30 PMFoamy Viral Vector Expressing Human CD18 Results in High Levels of Transduction and Multilineage Engraftment with CD18+ LAD-1 Cells in NSG Micerichard h. Smith, Md nasimuzzaman, roop Dutta, Gulbu uzel, Thomas r. bauer, Steven M. holland, David W. russell, Dennis D. hickstein, Punam Malik, Johannes C. M. van der loo, Andre larochelle

Oral Abstract Session 245Vector and Cell Engineering/Manufacturing4:00 PM - 5:45 PMroom: Washington 1-2

286 4:00 PMGenome Editing of Inducible Cell Lines for Scalable Production of Improved Lentiviral Vectors for Human Gene TherapyAlessio Cantore, Michela Milani, Andrea Annoni, Johannes lengler, Sara bartolaccini, Tiziano Di Tomaso, Michael C. holmes, Friedrich Scheiflinger, Angelo lombardo, luigi naldini

287 4:15 PMProduction of KTE-C19 (Anti-CD19 CAR T Cells) for ZUMA-1: A Phase 1/2 Multi-Center Study Evaluating Safety and Efficacy in Subjects with Refractory Aggressive Non-Hodgkin Lymphoma (NHL)Marc better, Vijay Chiruvolu, James oliver, emily lowe, John M. rossi, Arianne Perez, lynn navale, Adrian bot

288 4:30 PMDevelopment of Cocal Glycoprotein Envelope Producer Cell Lines for Robust Lentiviral Gene Transfer into Hematopoietic Stem Cells and T Cellsolivier humbert, Donald Gisch, Martin Wohlfahrt, Phil Greenberg, Tom Schmitt, Grant Trobridge, hans-Peter Kiem

289 4:45 PMSequence Modified Antibiotic Resistance Genes Provide Sustained Plasmid Mediated Transgene Expression in MammalsJiamiao lu, Feijie Zhang, Andrew Fire, Mark Kay

290 5:00 PMA High-Throughput, Sensitive, Accurate and Reproducible Method for Quantifying Vector Genomes of Recombinant Adeno-Associated Viruses in Crude LysateJianzhong Ai, raed Al-Abbasee, Guangping Gao

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291 5:15 PMCD26 Inhibition Enhances Chimerism of Lentivirally Transduced Hematopoietic Stem Cells in a Non-Myeloablative SettingGaryfalia Karponi, Afroditi Georgakopoulou, eleni Sgouramalli, eirini rantoglou, loudovikos Soufleros, Achilles Anagnostopoulos, evangelia yannaki

292 5:30 PMTowards Large-Scale Manufacturing of Adeno-Associated Virus by Transient Transfection of HEK293 Suspension Cells in a Stirred Tank Bioreactor Using Serum-Free MediumParminder Chahal, Érica Alessandra Schulze, Alice bernier, Stephane lanthier, nathalie Coulombe, Amine Kamen, renald Gilbert

Poster Session II6:00 PM - 8:00 PMroom: exhibit hall C & b South

AAV Vectors II

293Discovery of an Essential Receptor for Adeno-Associated Virus InfectionSirika Pillay, nancy l. Meyer, Andreas S. Puschnik, omar Davulco, Jonathan Diep, yoshi ishikawa, lucas T. Jae, Jonathan e. Wosen, Claude M. nagamine, Michael S. Chapman, Jan e. Carette

294Characterizing AAV CaptureSelect Affinity Ligand InteractionsJames K. S. Smith, Vibhu banala, Mario Mietzsch, Paul Chipman, Mavis Agbandje-McKenna

295Intrathecal Delivery of rAAV9-ABCD1 by Osmotic Pump in a Mouse Model of Adrenomyeloneuropathyyi Gong, rene Kok, Dakai Mu, Guangping Gao, Florian eichler

296Longitudinal Surveillance of Non Human Primate Recipients of Intrauterine Gene Transfer for Coagulation Factor DeficienciesCitra Mattar, Arijit biswas, nuryanti Johana, yvonne y. W. Tan, Cecilia rosales, Jenny McKintosh, lay-Geok Tan, irene Gil-Farina, Mahesh Choolani, Manfred Schmidt, Amit nathwani, Jerry K. y. Chan

297The Influence of ELABELA AAV Gene Therapy on Cardiorenal Function in Rat Models of Salt-Induced Hypertension and Myocardial InfarctionClaire A. Schreiber, Sara holditch, Alex Generous, yasuhiro ikeda

298Characterization of a Novel Anti-Sense Promoter Element in Adeno-Associated Virus: Implications for Hepatocellular CarcinomaGrant logan, Christine M. Smyth, Claus V. hallwirth, neeta Khandekar, Samantha l. Ginn, Sophia laio, Allison P. Dane, ian e. Alexander

299Safety and Biodistribution Study of rAAV2tYF-PR1.7-hCNGB3 in CNGB3-Deficient MiceGuo-jie ye, ewa budzynski, Peter Sonnentag, T. Michael nork, Paul e. Miller, leslie McPherson, James n. Ver hoeve, leia M. Smith, Tara Arndt Arndt, Savitri Mandapati, Paulette M. robinson, roberto Calcedo, David r. Knop, William W. hauswirth, Jeffrey D. Chulay

300Changing the Route of Administration to Improve Liver Transduction by Recombinant AAV-Based Vectorsnerea Zabaleta, David Salas, Anna Majowicz, rubén hernandez-Alcoceba, Valerie Sier-Ferreira, Jesús Prieto, ignacio bilbao, Gloria González-Aseguinolaza

301AAV Capsid Engineering to Improve Transduction in Retina and BrainJennifer Sullivan, lisa Stanek, Michael lukason, elizabeth barry, Shayla russell, James Morris, bryan Mastis, Anna Alves, Jie bu, lamya S. Shihabuddin, Seng Cheng, Abraham Scaria

302Molecular AAV Capsid Evolution Is Not Primarily Restricted by Inadvertent Shuffling of the Assembly-Activating Protein AAPStefanie Grosse, Magalie Penaud-budloo, eduard Ayuso, Dirk Grimm

303Deletion of Pdx1 in Mouse Pancreatic Ducts by CRISPR-Cas9 Mediated Gene EditingAiping lu, George K. Gittes, Johnny huard, Ping Guo

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304Particle Titer Determination and Characterization of rAAV Molecules Using Nanoparticle Tracking AnalysisSamantha F. Povlich, Jonathan G. Mehtala, Gwendolyn M. Wilmes, Christopher J. Morrison, Samuel C. Wadsworth, K. reed Clark

305Generation of Light-Producing, Somatic-Transgenic Mice Using Lentivirus and Adeno-Associated Virus Vectorsrajvinder Karda, Dany P. Perocheau, Suzanne M. K. buckley, Juliette M. K. M. Delhove, Michael hughes, Ahad A. rahim, Mark r. Johnson, Joanne ng, natalie Suff, Tristan r. Mckay, Simon n. Waddington

306Production of Safe and Efficient AAV Vectors Using Minicircle DNAMarco Schmeer, Tatjana buchholz, Martin Schleef, Maria Schnödt, Anja Grünert, ram Shankar, Thomas Fuchsluger, hildegard büning

307Hitting the Kidney from All Angles: Using Direct Intrarenal Pelvis Injection Combined with Serotype and Promoter Targeting to Achieve Renal AAV Gene DeliveryClaire A. Schreiber, Sara holditch, yasuhiro ikeda

308Ultracentrifugation-Free Chromatography-Mediated Large-Scale Purification of Recombinant Adeno-Associated Virus Serotype 1 (rAAV1) and rAAV9 from the Serum-Free Culture SupernatantTaro Tomono, yukihiko hirai, hironori okada, Kumi Adachi, Akiko ishii, Takashi Shimada, Masafumi onodera, Akira Tamaoka, Takashi okada

309Optimization of Production of AAVrh.10 Viral Vectorshyunmi lee, lucy Quach, Fan Fan, Sara Cram, Dolan Sondhi, ronald Crystal, Stephen Kaminsky

310Analysis of AAV5 Biodistribution and Viral Shedding in the Presence or Absence of Neutralizing AntibodiesDavid Salas, Anna Majowicz, nerea Zabaleta, estefania rodríguez-Garcia, harald Petry, Valerie Ferreira, Gloria Gonzalez Aseguinolaza

96 (Moved from Poster Session i)An Approach to Compare Multiple Adeno-Associated Virus (AAV) Capsids Side-by-Side in a Single Eye Following Intravitreal InjectionJohn M. Powers, Kei Adachi, benjamin Sivyer, William Taylor, Mark Pennesi, hiroyuki nakai

Targeted Genome Editing II

311In Vivo Zinc Finger Nuclease-Mediated Targeted Integration of a Glucose-6-Phosphatase Transgene Enhances Biochemical Correction in Mice with Glycogen Storage Disease Type IADustin J. landau, elizabeth D. brooks, Pablo Pablo Perez-Pinera, hiruni Amarasekara, Adam Mefferd, Songtao li, Andrew bird, Charles A. Gersbach, Dwight D. Koeberl

312Gene Correction of LGMD2A Patient-Specific iPS Cells for Targeted Autologous Cell TherapySridhar Selvaraj, Antonio Filareto, James Kiley, Daniel Voytas, Michael Kyba, rita Perlingeiro

313Gesicle Mediated Delivery of a Cas9-sgRNA Protein ComplexThomas Quinn, Montse Morell, Tatiana Garachtchenko, lily lee, Mei Fong, Michael haugwitz, Andrew Farmer

314Mathematical Modeling of Erythrocyte Chimerism Informs Clinical Strategies for Sickle Cell DiseasePhilipp M. Altrock, Christian brendel, raffaele renella, Stuart h. orkin, David A. Williams, Franziska Michor

315Highly Efficient Targeted Addition of Large XIST Transgenes in a Population of Somatic CellsJun Jiang, Jeanne lawrence

316Successful Editing of the CD40LG Locus in Human Hematopoietic Stem CellsGabrielle Curinga, Sarah leach, Swati Singh, nick hubbard, David hagin, Karen Sommer, iram Khan, yumei Song, Troy Torgerson, Andy Scharenberg, David J. rawlings

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317Screening S. Aureus CRISPR-Cas9 Paired-Guide RNAs for Efficient Targeted Deletion in Duchenne Muscular DystrophyJosh Tycko, nick huston, Jacqueline n. robinson-hamm, Chris Wilson, Charles A. Gersbach, Patrick D. hsu, David bumcrot

318Targeted Genome Editing of Human Induced Pluripotent Stem Cells Using CRISPR/CAS9 to Generate a Knock-in Type II Collagen Reporter for the Purification of Chondrogenic CellsShaunak S. Adkar, Vincent P. Willard, Jonathan M. brunger, Kenneth T. Shiao, Charles A. Gersbach, Farshid Guilak

319Rationally Designed AAV Inverted Terminal Repeats Enhance Gene TargetingMatthew hirsch, Jerry Wu

320Transcriptional Silencing via Synthetic DNA Binding Protein Lacking Canonical Repressor Domains as a Potent Tool to Generate TherapeuticsSalvatore botta, elena Marrocco, nicola de Prisco, Fabiola Curion, Mario renda, Martina Sofia, Mariangela lupo, Maria laura bacci, Carlo Gesualdo, Settimio rossi, Francesca Simonelli, enrico Maria Surace

321Deletion of Mutated GAA Repeats from the Intron 1 of the Frataxin Gene Using the CRISPR System Restores the Protein Expression in a Friedreich Ataxia ModelDominique l. ouellet, Joel rousseau, Khadija Cherif, Catherine Gérard, renald Gilbert, Alain Garnier, bruno Gaillet, Christian beauséjour, Jacques P. Tremblay

322Genome Editing for Nucleotide Repeat Disorders: Towards a New Therapeutic Approach for Myotonic Dystrophy Type 1Mirella lo Scrudato, Samia Martin, Geneviève Gourdon, Denis Furling, Ana buj-bello

323Efficient Generation of CART Cells by Homology Directed Transgene Integration into the TCR-Alpha Locusbaeckseung lee, hang leung, Jordan Jarjour, Alexander Astrakhan

324Successful Repeated Delivery of Helper-Dependent Adenoviral Vector Toachieve Efficient Long-Term Human CFTR Expression in Mouse Airwaythrough Transient Immunosuppressionhuibi Cao, Jim hu

325Targeted Gene Addition Strategies for the Treatment of X-Linked Lymphoproliferative Diseasebenjamin C. houghton, Claudio Mussolino, Toni Cathomen, h bobby Gaspar, Claire booth

326Improving CRISPR-Cas9 Precision Through Directly or Indirectly Tethered DNA-Binding DomainsScot Wolfe, Mehmet F. bolukbasi, Ankit Gupta, lihua J. Zhu

327Genetic Editing for Huntington’s Diseasenicole Deglon, Cécile Meunier, Virginie Zimmer, Guillaume Perriard, Mathieu Canales, Gabriel Vachey, lucas hergott, Tim beltraminelli, Thomas Dequesne, Catherine Pythoud, Maria rey, renaud Du Pasquier, luc Pellerin, Anselme Perrier

328Proof-of-Principle Study Shows Efficient Skipping of Exon 2 Using Antisense Morpholino OligomersTabatha r. Simmons, nicolas Wein, Felecia Gumienny, hannah huang, Kevin M. Flanigan

329Using CRISPR/Cas9 as a Therapeutic Approach for Leber Congenital Amaurosis 10 (LCA10)Guoxiang ruan, elizabeth barry, Michael lukason, Seng Cheng, Abraham Scaria

330Using CRISPR/Cas9 to Edit Hematopoietic Stem and Progenitor Cellseduardo Seclen, hsu-yu Chen, George n. llewellyn, Su J. yang, Morgan Chateau, Cathy x. Wang, Paula M. Cannon

331CRISPR-Cas9 Can Inhibit HIV-1 Replication but Also Facilitates Virus Escapeben berkhout, Gang Wang, na Zhao, Atze T. Das

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332Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cellsr. Geoffrey Sargent, Shingo Suzuki, luke Gruenert, Fei xie, lin ye, yuet W. Kan, Dieter C. Gruenert

333Intracellular Ankyrin Specific to p24 and zfp Specific to 2-LTR Are Alternatives to CCR5 Knockdown for Stem Cell Gene TherapyChatchai Tayapiwatsna

334Double Expression Cassettes Engineering at the Two Alleles with Same Genome Site of Human Cell Using CRISPR/Cas9 Vector SystemZhimin Wang

Non-Viral Gene Transfer and Therapy I

335High Content Analysis Platform for Optimization of Lipid Mediated CRISPR-Cas9 Delivery Strategies in Human Cellsbenjamin Steyer, Jared Carlson-Stevermer, nicolas Angenent-Mari, Andrew Khalil, Ty harkness, Krishanu Saha

336Delivery Highways: Tunneling Nanotubes Facilitate Transfer of Therapeutic Molecules for Gene Therapy Treatment of CystinosisSpencer Goodman, Swati naphade, Jay Sharma, héloïse P. Gaide Chevronnay, Pierre J. Courtoy, Stephanie Cherqui

337Transient Ultrasound-Mediated Microbubble-Assisted Modulation of Blood-Brain Interface in Adult Common Marmoset to Improve rAAV-Mediated Brain Transductionhironori okada, hidetoshi ishibashi, Chiaki Masuda, hiromi hayashita-Kinoh, Tomoko Chiyo, yuko nitahara-Kasahara, yoko endo-Takahashi, Koichi Kato, yoichi negishi, Shin’ichi Takeda, Takashi okada

338Improving Virus-Free Modification of Endothelial Cells - Magnetically Targeted Manipulationnatalia Voronina, heiko lemcke, Frank Wiekhorst, Jens Kühn, robert David, Gustav Steinhoff

339Development of Multifunctional Nanoparticles for Targeted Gene Deliveryrajendra n. Mitra, Min Zheng, Zongchao han

340Cell Penetrating Peptide Can Improve the Efficacy of Mitochondrial TransferDaisuke Kami, Tomoya Kitani, yuki Takaoka, yu yoshimoto, yasuhiko Tabata, Satoshi Gojo

341Nucleic Acid Delivery Using Vesicular Stomatitis Virus-Based VesiclesMathias Mangion, Alexandre Audy, igor Slivac, rénald Gilbert, bruno Gaillet

342Water Soluble and Bio-Friendly Nanoceria Reduces Laser-Induced CNV Mouse Model of Wet AMDrajendra n. Mitra, Min Zheng, Zongchao han

343Non-Viral Transfection of Primary Human Fibroblasts in Suspension Microcarrier CultureCharlie y. hsu, Derrick e. rancourt

344En Route to Non-Viral Genetic Engineering: Kinetics of DNA Uptake and Transgene Expression Following Repeated Transfection with Multiple Episomal Plasmids in Human Primary FibroblastCharlie y. hsu, Derrick e. rancourt

345Transient Removal of CD46 Sensitizes Breast Tumor Cells to Complement Attack Combination with CurcuminPei-rong Wang

600 (Moved from Poster Session iii)Development and Characterisation of Polymeric Nanocapsules Containing Macromoleculeomar S. Abu Abed, Amal A. elkordy, Ching Chaw, lee Williams, Marc Carlile

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Diabetes, Metabolic and Genetic Diseases II

346AAV9 Delivery into Cerebrospinal Fluid Corrects CNS Disease in a Murine Model of Mucopolysaccharidosis Type IIChristian hinderer, nathan Katz, Jean-Pierre louboutin, Peter bell, hongwei yu, Mohamad naval, Karen Kozarsky, Timothy o’brien, Tamara Goode, James M. Wilson

347CRISPR/Cas9-Based Gene Correction of Arginase-Deficient Human Induced Pluripotent Stem Cells to Recover Enzyme Functionbrian Truong, Patrick C. lee, Agustin Vega-Crespo, William b. Gilmore, Kip hermann, Stephanie Kingman, Jonathan K. Tang, Katherine M. Chang, James A. byrne, Gerald S. lipshutz

348Correction of CNS and Somatic Pathology by Intra-Cerebrospinal Fluid Gene Therapy for Mucopolysaccharidosis Type IIVirginia A. haurigot, Sandra Motas, Sara Marcó, Albert ribera, Carles roca, xavier Sánchez, Miquel García, Maria Molas, Victor Sánchez, xavier león, Jesús ruberte, Fatima bosch

349The Cure of Canavan Disease: Is It a Scientific Fiction or Clinical Reality?Dominic J. Gessler, Danning li, hongxia xu, Qin Su, Guangping Gao

350Kidney-Directed Gene Therapy for Murine Glycogen Storage Disease Type IAyoungmok lee, Seongho bae, Celine J. rocca, Stephanie Cherqui, David A. Weinstein, Janice y. Chou

351Efficacious Non-Oligodendrocyte Gene Therapy Suggests a New Dogma About CNS Compartmentalization of NAA Metabolism and Supports a Metabolic Sink TheoryDominic J. Gessler, Danning li, hongxia xu, Qin Su, reuben Matalon, Guangping Gao

352Functional Benefits of Systemic rAAV9-hIDS Gene Delivery in MPS II Mouse ModelDouglas M. McCarty, Kimberly Zaraspe, Aaron S. Meadows, naoka Murakami, ricardo Pineda, Marybeth Camboni, Joseph Muenzer, haiyan Fu

3531-13C-Propionate Oxidation as a Measure of Methylmalonyl-CoA Mutase (MUT) Activity in Methylmalonic AcidemiaJack Gagné, irini Manoli, elizabeth harrington, Sarah Smyth, Jacob D. hattenbach, Jennifer l. Sloan, Kong y. Chen, Charles P. Venditti

354Genetically Modified Human Mesenchymal Stromal Cell (MSC) Delivery Improved Glucose Tolerance in Diet Induced Obese (DIO) Mouse ModelsCleyton Domingues, nabanita Kundu, neeki Ahmadi, Sabyasachi Sen

355Minicircles Show Improved Hepatic Expression of Their Transgene from a Natural Endogenous Promoter and Are Lost Upon Partial Hepatectomy Due to the Episomal Nature of the Vectorhiu-Man Viecelli, Andrea Schlegel, Tanja Scherer, Gabirella Allegri, Martin Schleef, Cary o harding, Johannes häberle, beat Thöny

356High-Resolution X-Ray Fluorescence Microscopy (XFM) Indicates Enhanced Brain Copper Delivery in AAV9-Treated Menkes Disease MiceMarie reine haddad, Martina ralle, David J. Vine, Patricia M. Zerfas, Stephen G. Kaler

357A Mouse Model of Cobalamin A (cblA) Class Isolated Methymalonic Academy (MMA) Provides Unique Platform for Testing Gene and Cell TherapiesMadeline epping, irini Manoli, Cindy Wang, Pat Zerfas, Victoria hoffmann, Gene elliott, lingli li, Chuck Venditti

358AAV-MitoTimer to Asses Mitochondrial Turnoverbennett Gosiker, Skylar rizzo, Amy Donate, Christina Pacak

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359Targeted Genome Editing Using TALENs to Correct a Mouse Model of Methylmalonic Acidemia (MMA)Jessica l. Schneller, randy J. Chandler, Charles P. Venditti

360Genotype/Phenotype Correlation of Cellular Function and AAV-Mediated Gene Delivery to Treat Xeroderma Pigmentosum - Cockayne Syndrome (XP - CS)Skylar rizzo, Amy l. Donate, Michael Jones, Kelsey Manko, Katherine e. Santostefano, nao Terada, Fowzan S. Alkuraya, Peter b. Kang, Christina A. Pacak

Gene Therapy for CNS Diseases

361IV Gene Therapy Corrects Feline GM1 Gangliosidosis Long Termheather edwards, Ashley randle, Miguel Sena-esteves, Douglas Martin

362Translatable Gene Therapy for Infantile Neuronal LipofuscinosisAlejandra J. rozenberg, Sahana nagabhushan Kalburgi, Steven J. Gray

363Improvement of Sandhoff Phenotype Following Intravenous Injection of Adeno-Associated Viral Vector Expressing a Hexosaminidase Isoenzyme in Adult Sandhoff Mice: Preclinical Safety and Efficacy StudyKarlaina J. l. osmon, evan Woodley, Patrick Thompson, Subha Karumuthil-Melethil, Steven Gray, Jagdeep Walia

364Neurological Correction of Mucopolysaccharidosis IIIB Mice by Haematopoietic Stem Cell Gene TherapyStuart M. ellison, rebecca holley, Daniel Fil, John Mc Dermott, nisha Senthivel, Alex langford-Smith, Fiona Wilkinson, Simon Jones, rob Wynn, Shih-hsin Kan, Patti Dickson, brian bigger

365Long-Term Toxicology Evaluation of AAVrh.10hARSA Administration to the CNS of Nonhuman Primates to Treat Metachromatic LeukodystrophyDolan Sondhi, Jonathan b. rosenberg, bishnu P. De, Sebastien Monette, Alvin Chen, Thomas Flagiello, elisa Caffrey, Stephen M. Kaminsky, Patrick Aubourg, ronald G. Crystal

366Hitting Two Birds with One Stone: How Efficacious Pre-Clinical Gene Therapy Cures Canavan Disease and Sheds Light onto the PathomechanismDominic J. Gessler, Danning li, hongxia xu, Qin Su, reuben Matalon, Guangping Gao

367A Dopamine Gene Therapy for Advanced PD: 4 Years Phase I/II Clinical UpdateStéphane Palfi, Jean Marc Gurruchaga, Scott Gs ralph, Colin Watts, Philip buttery, helene lepetit, James Miskin, Gaetane Gouello, Sonia lavisse, Gilles Fenelon, Claire Thiriez, Pierre brugières, roger barker, Philippe hantraye, Kyriacos Mitrophanous

368Advances in Fragile X Gene Therapy Using Adeno-Associated Viral Vectors Coding for the Fragile X Mental Retardation ProteinJason Arsenault, yosuke niibori, David r. hampson, Shervin Gholizadeh

369Therapeutic Effectiveness of AAV-Mediated Iduronidase Delivery to the CNS Following Intravenous Administration in a Murine Model of Mucopolysaccharidosis Type Ilalitha belur, Thuy An Tran, Kelly M. Pedersen, Maureen riedl, lucy Vulchanova, Karen Kozarsky, Walter C. low, r. Scott Mcivor

370Reconstruction of the Nigrostriatal Pathway in Parkinsonian MacaquesDiego Pignataro, Diego Sucunza, Alberto J. rico, iria G. Dopeso-reyes, elvira roda, Africa Vales, Gloria Gonzalez-Aseguinolaza, nick Kholodilov, robert e. burke, José l. lanciego

371AAV9-Mediated Over-Expression of Hippocampal Neuritin Prevents Extremely Low-Frequency Electromagnetic Field Exposure-Induced Impairment of Recognition Memoryyanai Mei, Qianru Zhao, JunMei lu, Jinjing yao, Zhengyu Zhang, Chen ling

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372U1 snRNA-Mediated Correction of a Splicing Error of the Dopa Decarboxylase Geneni-Chung lee, yu-May lee, yin-hsiu Chien, barry byrne, Wuh-liang hwu

373Optimization of AAV-Gene Therapy for GM1-Gangliosidosislorelei Stoica, Sophia Todeasa, Jaclyn lata, Douglas Martin, Miguel Sena-esteves

374Developing Bone Marrow Transplant and Lentiviral Vectors to Treat Friedreich AtaxiaSze hwee ong, Dong C. Zhang, Chou h. Sim, Matthew burton, Dean Phelan, Sarah e. M. Stephenson, Gabrielle r. Wilson, Donald F. newgreen, Anthony J. hannan, Paul J. lockhart, Martin b. Delatycki, Marguerite V. evans-Galea

375TDP-43 Interacts with Cannabinoid Type 1 Receptor Modulating Its Signaling PropertiesCatalin Filipeanu, Matthew r. Deshotels, Peter Weed, Anastasia r. robinson, blessing ongulade, Peter J. Winsauer, Jessie J. Guidry

376Overexpression of Pitx3 in the SNpc and Protection from MPTP ToxicityJoanne Clark-Matott, Allison K. Graebner, Caroline e. bass, David K. Simon

188 (Moved from Poster Session i)AAV Mediated Gene Therapy to Modulate Neurotropic Factors in the Retina and in Neuronal Cells in Culturenitin Chitranshi, roshana Vander Wall, yogita Dheer, Stuart l. Graham, Vivek Gupta

Musculo-Skeletal Diseases I

377AAV-Mediated Transfer of FKRP Shows Therapeutic Efficacy in a Murine Model of Limb-Girdle Muscular Dystrophy Type 2i, but Requires Tight Control of Gene Expressionevelyne Gicquel, natacha Maizonnier, Steven J. Foltz, William Martin, nathalie bourg, Fedor Svinartchouk, Karine Charton, Aaron beedle, isabelle richard

378Improved Transduction of Canine X-Linked Muscular Dystrophy with rAAV9-Microdystrophin by Introducing Immune Tolerancehiromi hayashita-Kinoh, hironori okada, yuko n. Kasahara, Tomoko Chiyo, Kiwamu imagawa, Katsuhiko Tachibana, Shin’ichi Takeda, Takashi okada

379MicroRNA-29 and Micro-Dystrophin Combinatorial Therapy Suppresses Fibrosis and Restores Function to mdx/utrn+/- MiceKristin heller, eric Meadows, Sarah lewis, Josh Mendell, Jerry Mendell, louise rodino-Klapac

380Therapeutic Capacity of AAV-Delivered Muscle-Specific-Expressed Micro-Utrophin (ΔR4-R21/ΔCT) in mdx4cv Skeletal MusclesGlen b. banks, Quynh nguyen, Stephen D. hauschka, Jeffrey S. Chamberlain, Guy l. odom

381Prohypertrophic Agents Enhance the Response to Gene Therapy in Pompe DiseaseSang-oh han, Songtao li, Dwight D. Koeberl

382Correction of Autophagic Dysregulation in Pompe Disease Following Gene TherapyAngela l. McCall, Sylvia G. Stankov, Darin J. Falk, barry J. byrne

383Clinico-Pathological Correlation in the Earliest Stages of Muscular Dystrophy Suggests Sensitive Physiological Parameters as Novel Primary Endpoints for Systemic Gene Therapybenjamin W. Kozyak, Andy Mead, yafeng Song, leon Morales, Joe Kornegay, hansell h. Stedman

384 Abstract Withdrawn

385Inhibiting the Myostatin Signaling Pathway using CRISPR/Cas9-Based RepressorsPratiksha i. Thakore, nishkala K. Shivakumar, Charles A. Gersbach

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386Toxicology for DUX4-Targeted MicroRNAslindsay M. Wallace, Jacqueline S. Domire, Danielle A. Griffin, louise r. rodino-Klapac, Scott Q. harper

625 (Moved from Poster Session iii)RNAi Therapy for Dominant Limb Girdle Muscular Dystrophy Type 1Alindsay M. Wallace, Carlee r. Giesige, Danielle A. Griffin, louise r. rodino-Klapaca, Scott Q. harper

387Transfer of Hyaluronic Acid Synthase-2 Gene into Canine Joints Using Adeno-Associated Viral VectorsMonica D. Figueiredo, Patricia berthelette, Cathy Sookdeo, Donna Armentano, Sirkka Kyostio-Moore

388Hypersensitivity of MYH16 Fibers (M Fibers) to Myodegeneration in Canine Muscular Dystrophyyafeng Song, Andy Mead, Mihail Petrov, Phoebe ingraham, leon Morales, Shira rosenblum, Joe Kornegay, hansell h. Stedman

389Muscular Dystrophy Genes and the Early Metazoan Transition from Dynein- to Myosin-Powered LocomotionAndy Mead, Alock Malik, ranjith Krishnankutty, hansell h. Stedman

390Impact of a Treatment with Antioxidant on Gene Transfer Efficiency After Recombinant Adeno-Associated Vector Injection in a Mouse Model of Duchenne Muscular DystrophyJean-baptiste Dupont, benoît Tournaire, romain Durand, béatrice Marolleau, emilie bertil, Christophe Georger, emilie lecomte, benjamin Cogné, bernard Gjata, laetitia van Wittenberghe, Alban Vignaud, richard o. Snyder, Philippe Moullier, Adrien léger

622 (Moved from Poster Session iii)Systemic β-Sarcoglycan Gene Therapy for Treatment of Cardiac and Skeletal Muscle Deficits in LGMD2Eeric Pozsgai, Danielle Griffin, Kristin heller, Jerry Mendell, louise rodino-Klapac

Cancer-Immunotherapy, Cancer Vaccines II

391Mutated Tumor Neoantigens Are Recognized by Tumor Infiltrating Lymphocytes from Metastatic Ovarian CancerDrew C. Deniger, Anna Pasetto, Todd D. Prickett, Jared J. Gartner, Mini bharathan, eric Tran, Paul F. robbins, Steven A. rosenberg

392Super-Resolution (STED) Imaging Reveals Simultaneous Co-Docking of Tandem Chimeric Antigen Receptors to Two Target Antigens Enhancing T Cell Functionality and Mitigating Antigen EscapeMalini Mukherjee, Shoba navai, Daniel landi, Kristen Fousek, Antonella Pignata, Kevin bielamowicz, Tiara byrd, Maksim Mamonkin, Meenakshi hegde, Malcolm K. brenner, Jordan S. orange, nabil Ahmed

393Engineering Armored T Cell Receptor-Mimic (TCRm) Chimeric Antigen Receptor (CAR) T Cells Specific for the Intracellular Protein Wilms Tumor 1 (WT1) for Treatment of Hematologic and Solid MalignanciesSarwish rafiq, Terence Purdon, Tao Dao, Chen liu, David Scheinberg, renier J. brentjens

394Generation of a Novel Allogeneic CAR T Cell Platform Utilizing an Engineered Meganuclease and AAV Donor Template to Achieve Efficient Disruption of T Cell Receptor Expression and Simultaneous Homology-Directed Insertion of a CD19 CARDaniel T. Macleod, Jeyaraj Antony, Keith J. Wetzel, rachel Moser, Audrey e. brown, Jo Ann hux, Caitlin A. Turner, Armin hekele, Janel lape, Clayton W. beard, bruce McCreedy, Victor V. bartsevich, Michael G. nicholson, Jeff Smith, Matthew hirsch, Derek Jantz

395Development of the Novel Oral Tumor Vaccine Using Bifidobacterium longum Displaying Wilms’ Tumor 1 ProteinKoichi Kitagawa, Tsugumi oda, Ayame Araki, reina Gonoi, hiroki Saito, yoshiko hashii, Takane Katayama, Keiichi ozono, Toshiro Shirakawa

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396Development of GD2-Specific Immunoliposomes for Immunotherapy of NeuroblastomaWei huang, Daofeng liu, linjie Guo, ekaterina Marinova, Andras heczey, leonid Metelitsa

397Allogenic CAR T-Cells Targeting CD123 Effectively Eliminate Myeloid Leukemia Cellsroman Galetto, Céline lebuhotel, Agnès Gouble, Alexander bank, Gail J. roboz, Monica l. Guzman, Julianne Smith

398Site-Specific Introduction of Chimeric Antigen Receptors to Primary Human T CellsMalika hale, Alexandra Grier, yuchi honaker, Guillermo S. romano ibarra, Karen Sommer, iram F. Khan, nicholas hasle, Jaya Sahni, baeckseung lee, Jordan Jarjour, Alexander Astrakhan, Andrew M. Scharenberg, David J. rawlings

399Evaluation of CD123 Targeting CART Cells in Non-Human Primateslinda Dong, Saar Gill, Vijay bhoj, olga Shestova, lori Martin, Daher ibrahim Aibo, xiaorui yao, leeAnne Talbot, elizabeth Skuba, Celeste richardson, regina young, Michael C. Milone, Steve busch, Jennifer Marlowe, Timothy Maclachlan

400CD269 (BCMA)-Specific CAR-Expressing T Cells Dramatically Eradicate Myeloma Cells from Bone Marrow of an Orthotopic Multiple Myeloma Mouse Modelryosuke uchibori, Takeshi Teruya, Ken ohmine, Masashi urabe, yasushi Saga, hiroaki Mizukami, Junichi Mineno, Kazuto Takesako, Keiya ozawa

401In Vivo Expression of Plasmid Encoded IgG for PD-1 or LAG3 by Synthetic DNA as a New Tool for Cancer ImmunotherapyKar Muthumani, C. Chung, S. Agarwal, r. Plyler, S. Kudchodkar, S. Flingai, K. e. ugen, J. J. Kim, n. Sardesai, David b. Weiner

402Bioengineering of Peripheral Blood Derived Gamma Delta T Cells in a Serum-Free Expansion MediumKathryn Sutton, Anindya Dasgupta, McCarty David, Christopher Doering, h. Trent Spencer

403Toca Retroviral Replicating Vector in Tumor and Blood from Clinical Trial Subjects with Recurrent High Grade GliomaDaniel hogan

404Development of Combinational Cellular Immunotherapy to Treat Pediatric Acute Myeloid Leukemia Patientsniek P. van Til, lea Knežević, Maud Plantinga, Colin de haar, rob C. hoeben, Manuel A. Gonçalves, Stefan nierkens, Jaap Jan boelens

405IL-2 and TNF-a Coding Adenoviruses Enable Adoptive T Cell Therapy in Metastatic, Solid Cancer by Systemically Activating Tumor-Reactive TILsSiri Tähtinen, Carolin blattner, Markus Vähä-Koskela, Dipongkor Saha, Mikko Siurala, riikka havunen, Suvi Parviainen, Jochen utikal, Viktor umansky, Akseli hemminki

406Development and Optimization of PSCA-Specific CAR T Cells for the Treatment of Bone Metastatic Prostate CancerDileshni Tilakawardane, ethan A. Gerdts, Anthony K. Park, Wen-Chung Chang, Sarah Wright, Christine brown, Stephen J. Forman, Saul J. Priceman

407A Novel Rabbit Antibody-Derived, Anti-CD123 LV/CAR Construct for AML Immunotherapyrobyn A. A. oldham, elliot M. berinstein, Jeffrey A. Medin

408Oncolytic Vaccines in Combination with PD-L1 Blockade for the Treatment of MelanomaCristian Capasso, Davide Cardella, Martin Muller, Mariangela Garofalo, lukasz Kuryk, Karita Peltonen, Vincenzo Cerullo

Cancer-Oncolytic Viruses I

409Novel Recombinant Coxsackievirus B3 Infection Elicits Robust Oncolytic Activity Against Human Non-Small Lung Cancer and Triple-Negative Breast CancerMiyako Sagara, yuto Takishima, Shohei Miyamoto, hiroyuki Shimizu, yoichi nakanishi, Kenzaburo Tani

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410Oncolytic Adenoviruses Armed with TNFa and IL-2 Induce Antitumor Immune Responses and Protection from Tumor Rechallengeriikka havunen, Mikko Siurala, Suvi Parviainen, Michael behr, Dirk nettelbeck, Anja ehrhardt, Akseli hemminki

411Predicting Tumor Response to Oncolytic Virotherapy Using Dual Isotope SPECT/CT Imaging with NIS Reporter Gene and DuramycinKah-Whye Peng, lianwen Zhang, lukkana Suksanpaisan, Stephen J. russell, Ming Zhao

412The Efficacy and Bio-Distribution of Oncolytic HSV-1 (G47Δ) in Mouse Orthotopic Esophageal Cancer ModelsShoh yajima, yasushi ino, hiroshi Fukuhara, Miwako iwai, yasuyuki Seto, Tomoki Todo

413Effect of Adenoviral Death Protein on NIS-Based Iodine Therapy and Imaging for Pancreatic Cancerlisa Koodie, eriko Kawakami, ben eidenschink, Jordan Sell, Chris larocca, Kari Jacobsen, Jessica ryvlin, ezequiel Tolosa, John C. Morris, Martin Fernandez-Zapico, Julia Davydova

414A Novel System to Systematically Analyze Diverse Types of Conditionally Replicating Adenoviruses Expressing Immunostimulatory Genes in Syngenic Hamster Modelsnobuhiro ijichi, hirotaka Kawakami, yuki obama, Ken-ichiro Kosai

415The Potency of a Histone Deacetylase Inhibitor and Reolysin in Head and Neck Squamous Cell CarcinomaMatthew old, Cristina Jamie-ramirez, Jun-Ge yu, bhavna Kumar, Pawan Kumar, Quintin Pan, John lee, Theodoros Teknos, Flavia Pichiorri, balveen Kaur

416Enhanced Oncolytic Effect of Tumor Necrosis Factor Alpha Armed Engineered Oncolytic Measles Virus Therapy for Ovarian Carcinoma Cell LineAhmed M. Al-Shammari, Patrycja J. lech, Stephen J. russell

417Intratumoral Delivery of Oncolytic Adenovirus Expressing Decorin Inhibits Growth and Metastases of 4T1 Breast Tumors in Syngeneic Immune Competent BALB/c Mice ModelWeidong xu, yuefeng yang, Thomas neill, hua Wang, Chi-hsiung Wang, Chae-ok yun, Theresa Guise, Charles b. brendler, renato V. iozzo, lisheng Wang, Prem Seth

418Peripheral Blood Leucocytes, Neutralizing Antibodies and Tumor Burden as Predictive and Prognostic Factors in Patients Treated with Oncolytic Adenoviral ImmunotherapySuvi Parviainen, Kristian Taipale, ilkka liikanen, Anniina Koski, Anna Kanerva, Minna oksanen, Akseli hemminki

419Efficacy of Rambo for Sarcoma TherapyManinder Khosla

420Improvement of Antitumor Activity in Intraperitoneal Ovarian Cancer Model by a Noble Cloned Carrier Cell Infected with Oncolytic AdenovirusKatsuyuki hamada, Kazuko Takagi, Takashi Sugiyama

421Evaluating the Efficacy of Combination Cancer Treatment: Oncolytic Adenovirus and 5FU ChemotherapyJordan Sell, Amanda oliviera, eric Jensen, edward Greeno, Masato yamamoto, Julia Davydova

422Harnessing Immunogenic Cell Death to Potentiate the Oncolytic Effect of ORFV Infection in Murine Cancer CellsJacob P. van Vloten, lisa A. Santry, Jondavid de Jong, byram W. bridle, Sarah K. Wootton

423Clinical Translation of Immunovirotherapy: Measles Virus and Anti-PD1Christine e. engeland, rūta Veinalde, Christof von Kalle, Dirk Jaeger, Guy ungerechts

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Immunological Aspects of Gene Therapy I

424AAV Re-Administration Following Pediatric Application: Effect of Age at First AdministrationKimberly l. Pietersz, Sander J. van Deventer, harald Petry, Valerie Ferreira

425Production of Gene Corrected T Cell Precursors for Therapy of SCID X1Chantal lagresle-Peyrou, Cécile roudaut, Aurélie Gabrion, laure Caccavelli, hanem Sadek, Myriam Chouteau, Christian reimann, Salima hacein-bey-Abina, Marina Cavazzana, isabelle André-Schmutz

426Multi-Pathogen-Specific T Cells: A Single T-Cell Product Simultaneously Targeting Multiple Pathogens for Adoptive ImmunotherapyAnastasia Papadopoulou, Panayotis Kaloyannidis, Maria Alvanou, Stergios Papadimitriou, ioannis batsis, ioanna Sakellari, Achilles Anagnostopoulos, evangelia yannaki

427In Vivo Inhibition of HIV-1 in NSG Mice After Transduction of Primary Human T Cells with CXCR4 Conjugated to an HR2 PeptideGeorge J. leslie, Max richardson, Jianbin Wang, Philip Gregory, James riley, Michael holmes, James hoxie

428Generation of DNA Plasmid-Encoded Neutralizing Monoclonal Antibodies In VivoSeleeke Flingai, emily Plummer, Ami Patel, Janess Mendoza, yang Wang, Keith riemann, Mark Klempner, Kate broderick, niranjan Sardesai, Kar Muthumani, David Weiner

429Antibody Delivery by Genetically Engineered Human Hematopoietic Stem Cells to Fight HIVAnne-Sophie Kuhlmann, Kevin haworth, Morgan Giese, hans-Peter Kiem

430Antibodies to Recombinant Human α-L-Iduronidase Enhance Uptake into Macrophages in Murine Mucopolysaccharidosis Type IValentina Sanghez, Shih-hsin Kan, Steven Q le, Don Clarke, Kristen Vondrak, Moin u Vera, Michelina iacovino, Mark S Sands, Patricia i Dickson

431Lung Antibody Factory for Passive Immunisation Against InfluenzaTiong Kit Tan, Pramila rijal, lee A. Davies, ian A. Pringle, Alain r. Townsend, Stephen C. hyde, Deborah r. Gill

432Valproic Acid Treatment Enhances Hematopoietic Stem and Progenitor Cell Multipotency Ex Vivo for Enhanced Long-Term Engraftment of Gene-Modified CellsAnthony Conway, Alisa boyko, Jeremy hardin, Michael C. holmes, Gregory J. Cost

433DNA Monoclonal Antibodies Target Influenza Virus In VivoSarah T. C. elliott, Ami Patel, Megan C. Wise, Seleeke Flingai, Amelia Keaton, Jian yan, Kate e. broderick, Trevor Smith, niranjan y. Sardesai, Karrupiah Muthumani, David b. Weiner

434Adoptive Treg Cell Therapy Using Factor VIII-Specific CAR Regulatory T Cells Regulates Anti-Factor VIII Immune Responses in Hemophilia A Micerichard y. Fu, Meghan J. lyle, xuefeng Wang, Carol h. Miao

435Skin Delivery of a RSV Vaccine with Surface Electroporation Provides Full Protection from Lower Respiratory Disease in the Cotton RatTrevor Smith, Katherine Schultheis, Jay McCoy, niranjan Sardesai, Kate broderick

436Developing a Synthetic DNA Vaccine for an Emerging Pathogen - Middle East Respiratory Syndromeemma l. reuschel, Karrupiah Muthumani, Kim Kraynyak, Colleen Tingey, Sagar b. Kudchodkar, Amir S. Khan, niranjan y. Sardesai, J. Joseph Kim, Joel Maslow, young K. Park, Gary Kobinger, Darryl Falzarano, heinrich Feldman, David b. Weiner

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437A Light-Producing Model of Infection-Related Preterm Birthnatalie Suff, rajvinder Karda, Dany Perocheau, Juliette M. Delhove, Tristan r. McKay, Mark Tangney, Suzanne M. K. buckley, Donald Peebles, Simon n. Waddington

438Human, Pig and Mouse IFITMs Partially Restrict Pseudotyped Lentiviral VectorsAndrew l. hornick, ni li, Mayumi oakland, Paul b. McCray, Patrick l. Sinn

439Prolonged Expression of Secreted Enzymes in Dogs After Liver Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Mucopolysaccharidoses Types I and VIIelena l. Aronovich, Kendra A. hyland, bryan C. hall, Jason b. bell, erik r. olson, Myra rusten, David W. hunter, Chester b. Whitley, n. Matthew ellinwood, r. Scott Mcivor, Perry b. hackett

440Extreme Polyvalency Induces Potent Cross-Clade Cellular and Humoral Responses in Rabbits and Non-Human PrimatesMegan Wise, Kar Muthumani, Janess Mendoza, Jian yan, Anna Slager, Kim Kraynyak, David Montefiori, Celia labranche, Kate broderick, Matthew Morrow, niranjan Sardesai, David Weiner

441IL2 Plasmid Treated Hemophilia A Mice Show an Increase in Regulatory T Cell Population and Initial Tolerance to FVIIIMeghan J. lyle, richard y. Fu, Carol h. Miao

Cell Therapies II

442Developing Polymeric Bio-Scaffolds That Increase the Efficacy of Stem Cell-Mediated Therapy for Brain TumorsJuli bago, onyi okolie, Mahsa Mohiti-Asli, elizabeth loboa, Shawn hingtgen

443Neural Stem Cell-Mediated Enzyme/Prodrug Therapy for MedulloblastomaMargarita Gutova, lusine Tsaturyan, Michael e. barish, Karen S. Aboody, robert Wechsler-reya

444Large Scale Culture and Differentiation of Induced Pluripotent Stem Cells for Neutrophil Replacement Therapieslisa Trump, ramesh nayak, Schroll robin, Jose A. Cancelas, Carolyn lutzko

445cGMP Compliant Production for Human Embryonic Stem Cell Derived Retinal Pigment Epithelial Cells on a Synthetic Substrate for the Treatment of Non-Neovascular Age-Related Macular Degeneration for Phase I Clinical StudyP. M. lopez, T. Aguilar, rl Wu, A. Krishnan, D. Kong, J. Patel, h. Javier, W. Dang, D. Wong, D. Zhu, J. Dodd, y. Shad, M. humayun, D. r. hinton, D. o. Clegg, l. V. Johnson, C. M. lin, D. hsu, l. Couture

446Gender Differences in the Osteogenic & Chondrogenic Potential of Human Muscle-Derived Stem Cells In VitroAlex C. Scibetta, Andrea b. liebowitz, elizabeth r. Morris, Aiping lu, xueqin Gao, Johnny huard

447Notch Activation Enchances Vascular Lineage Commitment of Cardiac Stem CellsKonstantin Dergilev, Zoya Tsokolaeva, Marya boldyreva, irina beloglazova, ekaterina Zubkova, yelena Parfyonova

448Therapeutic Angiogenesis by Subcutaneous Cell Sheet Delivery Is Superior to Cell Injection: A Study of ADSC Efficacy in a Model of Hind Limb IschemiaPavel i. Makarevich, Maria A. boldyreva, Anastasia yu. efimenko, evgeny V. Gluhanyuk, Konstantin V. Dergilev, Julia o. Gallinger, yu-Chen hu, yelena V. Parfyonova

449IGF-1 Treatment Enhances the Myogenic Potential of Human Skeletal Muscle-Derived Stem Cellselizabeth Morris, Alex Scibetta, Aiping lu, xueqin Gao, Johnny huard

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450 Abstract Withdrawn

451Robust Manufacture of CAR-T CellsPradip bajgain, John Wilson, Dan Welch, helen e. heslop, Cliona M. rooney, Malcolm K. brenner, Ann M. leen, Juan F. Vera

452 Abstract Withdrawn

453Tumorigenic Potential in Clones Derived from Rat Bone Marrow CellsV. Arenas-luna, l. loredo-Mendoza, J. navarro-betancourt, C. bazán-A, P. García-V, l. lara-M, i. Gutiérrez-V, e. rodríguez-rivera, M. lópez-Martínez, A. Páez, F. Masso, S. hernández

454The Surface Marker Expression Changes in Monolayer Cultured Human Chondrocytes by TGF-β1hyun Joo yoon

Vector and Cell Engineering/Manufacturing I

455Automated Lentiviral Transduction of T Cells with CARS Using the CliniMACS Prodigylauren nickolay, ulrike Mock, brian Phillip, Gordon Weng Kit Cheung, hong Zhan, Karl Peggs, ian Johnston, Andrew Kaiser, Martin Pule, Adrian Thrasher, Waseem Qasim

456Transgene Bioengineering Through Ancestral Protein ReconstructionPhilip M. Zakas, harrison C. brown, Kristopher A. Knight, ernest T. Parker, h. Trent Spencer, eric A. Gaucher, Christopher b. Doering

457A Characterization of Transgene-Specific “Proteomes” in Lentiviral Vector Clinical Production Lots Identifies Differences between Vectors with High and Low Infectious TitersScott Witting, lilith reeves, Frank Witzmann

458Development of a Stable Producer Cell Line for Scalable Lentiviral Vector Production for Gene Therapy of HemoglobinopathieSarah Slauson, Amelia Thomas, byoung ryu, Melissa bonner, Gretchen lewis, Gabor Veres, Janet Chung, Geoffrey b. Parsons

459Evaluation of Miltenyi ExpAct and TransAct CD3/28 Beads for CAR-T Cell Manufacturingxiuyan Wang, Jinrong Qu, Jolanta Stefanski, oriana borquez-ojeda, Anniesha hack, Qing he, Teresa Wasielewska, Fang Du, Michel Sadelain, isabelle rivière

460Purification of AAV8 Using a Prototype Affinity Resin Compatible with cGMP ManufacturingColleen M. Dupuis, Amy A. Medeiros, Samuel C. Wadsworth, K. reed Clark, Christopher J. Morrison

461The Testing Strategy to Determine the Pharmacological Activity of Gene Therapy Drug Product (rAd-IFN) to Treat Intravesical Non-Muscle Invasive Bladder CancerMinna M. hassinen, Jenni Mykkänen, hanna lesch, Sara Paulo, Taina Koponen, Anna-Kaisa lehtivarjo, Steve Parrish, robert Shaw, nigel r. Parker, Seppo ylä-herttuala

462Serum Free Clinical Grade Large Scale Lentiviral Production System for Gene Therapy Applicationxin yu, Mu li, xavier de Mollerat du Jeu

463Suspension-Adapted HEK 293 Cells in Orbital Shaken Bioreactors for the Production of Adeno-Associated Virus VectorsDaniel r. blessing, bernard Schneider, Florian M. Wurm, nicole Déglon

464RCL-Pooling Assay: A Simplified Method for the Detection of Replication Competent Lentiviruses in Vector Batches Using Sequential Pooling.Corre Guillaume, Michel Dessainte, Jean-brice Marteau, bruno Dalle, David Fenard, Anne Galy

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465An Antibiotic-Free Strategy for Miniplasmid Productionram Shankar, Maurice Telaar, Karl Friehs, Martin Schleef, erwin Flaschel

466Manufacturing Solutions for Robust Cell Therapy Expansion and HarvestJulie Murrell, Samantha luther, Tristan lawson, Anjali Verma, Susan rigby, Sandhya Punreddy, Manjula Aysola, Tiffany hood, Daniel Kehoe, Aletta Schnitzler, Martha rook

Pharmacology/Toxicology Studies or Assay Development

467Assay Conditions Significantly Alter AAV Neutralizing Antibody Determinationheikki T. Turunen, eric Zinn, luk h. Vandenberghe

468GLP-Compliant Non-Clinical Safety and Biodistribution of a Recombinant AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VIrita Ferla, Jean-brice Marteau, Severine Pouillot, Karen Kozarsky, James brown, Stefania Galimberti, Maria Grazia Valsecchi, Vincent Zuliani, Alberto Auricchio

469Improved Protocol and Use of Mitochondrial DNA as Reference for qPCR Quantification of Integrase-Defective Lentiviral Vectors (IDLVs)bala Sai Sundarasetty, laura Gerasch, renata Stripecke

470New Insights into rAAV Integration Mechanisms by Targeted Enrichment SequencingStefan Wilkening, Saira Afzal, elena Senís, raffaele Fronza, Christof von Kalle, Dirk Grimm, Manfred Schmidt

471In Vivo Potency Assay for AAV-Based Gene Therapy Vectorsbishnu P. De, Alvin Chen, Jonathan b. rosenberg, Maria Chiuchiolo, benjamin Van de Graaf, odelya e. Pagovich, Dolan Sondhi, Carlo russo, Stephen M. Kaminsky, ronald G. Crystal

472Evaluation of Re-Administration of a Recombinant Adeno-Associated Vector Expressing Acid-Alpha-Glucosidase (rAAV9-DES-hGAA) in Pompe Disease: Preclinical to Clinical PlanningManuela Corti, brian Cleaver, nathalie Clement, Thomas Conlon, Kaitlyn Faris, Gensheng Wang, Janet benson, Alice Tarantal, Dave Fuller, roland herzog, barry J. byrne

473Qualification of a p24 ELISA Assay for Quantitation of Total Lentiviral Vector Concentrationelisabeth boucher, Pranali buch, Melissa buttimer, Josh Sorafine, Suzanna Melotti, Craig Fairchild, Mercedes Segura, James Warren

474Consequences of Infusion Time on Efficiency of Intravenous Delivery of Vector Genomes to the LiverJonathan b. rosenberg, bishnu P. De, lucy Quach, Carol russo, Stephen M. Kaminsky, Dolan Sondhi, ronald G. Crystal

475Effects of HSV-Tk Mediated Suicide Gene Therapy on Normal Brain CellsJubayer Al hossain, lars romø ystaas, Jelena Mrdalj, Kristjan Vaelk, Kristoffer riecken, boris Fehse, rolf bjerkvig, Janne Grønli, hrvoje Miletic

476No GVHD, but Human Inflammatory “Cytokine Storm” and Mouse Macrophage Activation Upon Accelerated Development of Human CD4+ Effector Memory T Cells in Long-Term Humanized Micebala Sai Sundarasetty, Andreas Schneider, Katherina Sewald, Susanne rittinghausen, Armin braun, Constanca Figueiredo, Arnold Ganser, renata Stripecke

477ANGPTL4 Limits the Extent of Colonic InflammationTerri Phua, Dickson Shao liang Chee, nguan Soon Tan

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Friday, May 6, 2016Presidential Symposium 320Presidential Symposium1:15 PM - 3:30 PMroom: Marriott Salons 1-3

478 2:30 PMAn Essential and Ubiquitous Protein Receptor for AAV; Glycans as Attachment Receptorsnancy l. Meyer, Sirika Pillay, Qing xie, omar Davulcu, Andreas Puschnik, Jonathan Diep, yoshi ishikawa, lucas Jae, Jonathan Wosen, Claude nagamine, Alex noble, Scott Stagg, Jan e. Carette, Michael S. Chapman

479 2:50 PMAn Optimized DNA Vaccine Formulation Protects Against Lethal Ebola Makona Virus Challenge in Non-Human Primates and Elicits Robust Immune ResponsesAmi Patel, emma l. reuschel, Kimberly A. Kraynyak, Trina racine, Daniel h. Park, Amelia A. Keaton, Muthumani Karuppiah, Devon Shedlock, Jian yan, Amir Khan, Kevin Tierney, Gary P. Kobinger, niranjan Sardesai, David b. Weiner

480 3:10 PMGene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional OutcomesJerry r. Mendell, Samiah Al-Zaidy, richard Shell, W Dave Arnold, louise rodino-Klapac, John T. Kissel, Thomas W. Prior, Carlos Miranda, linda lowes, lindsay Alfano, Katherine berry, Christopher Petek, Kathleen Church, lyndsey braun, Sarah Corcoran, Kathrin Meyer, Shibi likhite, Arthur hM burghes, Kevin D. Foust, brian K. Kaspar

Oral Abstract Session 330Targeted Genome Editing: In Vivo Genome Editing4:00 PM - 6:00 PMroom: Thurgood Marshall ne

481 4:00 PMCRISPR/Cas9-Mediated In Vivo Genome Editing to Correct the OTC spfash Mutation in Newborn Micelili Wang, yang yang, Peter bell, Deirdre McMenamin, Zhenning he, John White, hongwei yu, Chenyu xu, hiroki Morizono, Kiran Musunuru, Mark l. batshaw, James M. Wilson

482 4:15 PMLocal and Systemic Gene Editing in a Mouse Model of Duchenne Muscular DystrophyChristopher nelson, Matthew Gemberling, Chady h. hakim, David G. ousterout, Pratiksha i. Thakore, ruth M. Castellanos, Sarina Madhavan, xiufang Pan, F. Ann ran, Winston yan, Aravind Asokan, Feng Zhang, Dongsheng Duan, Charles A. Gersbach

483 4:30 PMIn Vivo DMD Gene Editing in Muscles and Muscle Stem Cells of Dystrophic MiceMohammadsharif Tabebordbar, Kexian Zhu, Jason Cheng, Jeffrey Widrick, Winston yan, ru xiao, luk Vandenberghe, Feng Zhang, Amy Wagers

484 4:45 PMIn Vivo Zinc-Finger Nuclease Mediated Iduronate-2-Sulfatase (IDS) Target Gene Insertion and Correction of Metabolic Disease in a Mouse Model of Mucopolysaccharidosis Type II (MPS II).Kanut laoharawee, russell DeKelver, Kelly M. Podetz-Petersen, Susan Tom, robert radeke, Michelle rohde, Amy Manning-bog, Scott Sproul, li ou, Kathleen Meyer, Michael C. holmes, Chester b. Whitley, Thomas Wechsler, r. Scott Mcivor

485 5:00 PMZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type Ili ou, russell DeKelver, Susan Tom, robert radeke, Michelle rohde, Amy Manning-bog, Scott Sproul, Michael J. Przybilla, brenda l. Koniar, Kelly Podetz-Pedersen, Kanut laoharawee, renee D. Cooksley, Michael C. holmes, Thomas Wechsler, r. Scott Mcivor, Chester b. Whitley

486 Withdrawn

733 5:15 PM (Moved from oral Session 411)Somatically Repairing Compound Heterozygous Recessive Mutations by Chromosomal Cut-and-Paste for In Vivo Gene TherapyDan Wang, haiwei Mou, Karen Tran, Jia li, blake y. Wang, Dominic J. Gessler, Phillip Wl Tai, Qin Su, Wen xue, Guangping Gao

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487 5:30 PMTherapeutic Genome Editing by Combined Viral and Non-Viral Delivery of CRISPR System Components to the Mouse Liverhao yin

488 5:45 PMIn Vitro and In Vivo Genome Editing of the RHO Gene to Downregulate Dominant MutationsMaria C. latella, Maria T. Di Salvo, Fabienne Cocchiarella, Daniela benati, Valeria Marigo, Alessandra recchia

Oral Abstract Session 331Chemical and Physical Methods for Delivery of Gene Therapeutics4:00 PM - 6:00 PMroom: Delaware Suite

489 4:00 PMTumor-Targeted Hursirna-Nanoparticle Delivery Inhibits Lung Tumor Growth In Vitro and In Vivoranganayaki Muralidharan, narsireddy Amreddy, Anish babu, Akhil Srivastava, Janani Panneerselvam, Allshine Chen, yan D. Zhao, lichao Zhao, uday b. Kompella, Anupama Munshi, rajagopal ramesh

490 4:15 PMElectroporation-Mediated Gene Transfer of Caveolin-1 Protects from Bleomycin-Induced Pulmonary Fibrosis Through Regulating Activation of Inflammasomexin lin, Michael barravecchia, David A Dean

491 4:30 PMEnhanced Delivery of Plasmid DNA to the Skin Using Gene Electrotransferrichard heller, Amy Donate, Siqi Guo, Chelsea edelblute, Anna bulysheva

492 4:45 PMManipulation of Ultrasound Conditions for Effective Gene Delivery in a Porcine ModelJames harrang, Shuxian Song, bryn M. Smith, Kyle Morrison, Carol h. Miao

493 5:00 PMNonviral Gene Transfer by Sequence-Defined Proton-Sponges with Combined Nucleic Acid Binding and Endosomal Buffering: Balancing Basicitiesulrich lächelt, Ana Krhac levacic, Dominik Wendel, ernst Wagner

494 5:15 PMDevelopment of a pH Sensor to Probe Endosomal Buffering of Polymeric Nanoparticles Effective for Gene DeliveryDavid r. Wilson, Denis routkevitch, Karl J. Wahlin, Don J. Zack, Alfredo Quinones-hinojosa, Jordan J. Green

495 5:30 PMActivation of DNA Pattern Recognition Receptors After Plasmid Electrotransfer in Melanoma Cells and Tumorsloree heller, Masa bosnjak, Katarina Znidar, Maja Cemazar

496 5:45 PMElucidating Design Rules Governing Extracellular Vesicle-Mediated Therapeutic Protein DeliveryMichelle e. hung, Joshua n. leonard

Oral Abstract Session 332Musculo-Skeletal Diseases4:00 PM - 6:00 PMroom: Maryland Suite

497 4:00 PMFollistatin Gene Therapy Improves Six Minute Walk Distance in Sporadic Inclusion Body Myositis (sIBM)Jerry r. Mendell, Zarife Sahenk, Mark hogan, Samiah Al-Zaidy, Kevin Flanigan, louise r. rodino-Klapac, Markus McColly, Kathleen Church, Sarah lewis, linda lowes, lindsay Alfano, Katherine berry, natalie Miller, igor Dvorchik, Melissa Moore-Clingenpeel, brian K. Kaspar

498 4:15 PMProlonged Benefit from Systemic rAAV8 in a Canine Model of Myotubular MyopathyMatthew elverman, David Mack, Melissa Goddard, Jessica Snyder, robert Grange, Karine Poulard, Virginie latournerie, Philippe Veron, laurine buscara, Christine le bec, Samia Martin, Fulvio Mavilio, Federico Mingozzi, hui Meng, Michael lawlor, Alan beggs, Anna buj-bello, Martin Childers

499 4:30 PMIntravenous Delivery of a Novel Micro-Dystrophin Vector Prevented Muscle Deterioration in Young Adult Canine Duchenne Muscular Dystrophy DogsChady hakim, xiufang Pan, Kasun Kodippili, Thais blessa, hsiao T. yang, Gary yao, Stacey leach, Craig emter, yongping yue, Keqing Zhang, Sean x. Duan, nalinda Wasala, Gregory Jenkins, Charles r. legg, Joel S. Schneider, Jeffrey S. Chamberlain, Dongsheng Duan

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500 4:45 PMGene Therapy Rescues Disease Phenotype in the Oculopharyngeal Muscular Dystrophy Mouse ModelGeorge Dickson, Alberto Malerba, Pierre Klein, Susan Jarmin, houria bachtarzi, Arnaud Ferry, Gillian butler-browne, David Suhy, Michael Graham, Capucine Trollet

501 5:00 PMIn Vivo Gene Editing for Duchenne Muscular Dystrophyniclas e. bengtsson, John K. hall, Guy l. odom, James Allen, Joel r. Chamberlain, Jeffrey S. Chamberlain

502 5:15 PMBiomaterial-Mediated Lentiviral Delivery of Anti-Inflammatory Genes in Cartilage-Derived Matrix HemispheresKatherine A. Glass, Christopher r. rowland, Adarsh r. ettyreddy, Jonathan M. brunger, nguyen P. T. huynh, Charles A. Gersbach, Farshid Guilak

503 5:30 PMAdeno-Associated Virus Vector (AAV) Microdystrophin Gene Therapy Prolongs Survival and Restores Muscle Function in the Canine Model of Duchenne Muscular Dystrophy (DMD)Caroline le Guiner, laurent Servais, Marie Montus, Thibaut larcher, bodvael Fraysse, Sophie Moullec, Taeyoung Koo, Alberto Malerba, Christine le bec, Matthias hebben, Carole Masurier, Federico Mingozzi, oumeya Adjali, Pierre Carlier, Jean-yves hogrel, bernard Gjata, yan Cherel, Takis Athanasopoulos, Fulvio Mavilio, Thomas Voit, Philippe Moullier, George Dickson, and the “AFM-sponsored Duchenne Consortium”

504 5:45 PMRestoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular DystrophyJacqueline n. robinson-hamm, Christopher e. nelson, ruth M. Castellanos rivera, Annemieke Aartsma-rus, Aravind Asokan, Charles A. Gersbach

Oral Abstract Session 333Cancer-Immunotherapy, Cancer Vaccines II4:00 PM - 6:00 PMroom: Thurgood Marshall SW

505 4:00 PMVGX-3100 Drives Regression of HPV16/18 CIN2/3 and Robust Cellular Immune Responses in Blood and Cervical Tissue in a Blinded, Randomized, Placebo-Controlled Phase 2B StudyMatthew Morrow, Connie Trimble, xuefei Shen, Michael Dallas, David Weiner, Jean boyer, Jian yan, Kimberly Kraynyak, Albert Sylester, Mary Giffear, Kathleen Marcozzi-Pierce, Divya Shah, Kate broderick, Amir Khan, Jessica lee, laurent humeau, niranjan Sardesai, Mark bagarazzi

506 4:15 PMIn Vivo Transduction of T Cells: The Future of Immunotherapy?inan edes, irene Schneider, Qi Zhou, Veit r. buchholz, Christoph Kemna, Dirk h. busch, Christian J. buchholz, Wolfgang uckert

507 4:30 PMChondroitin Sulfate Proteoglycan 4 (CSPG4)-Redirected T Cells Eliminate Glioblastoma-Derived NeurospheresSerena Pellegatta, barbara Savoldo, Chuang Su, Soldano Ferrone, Gaetano Finocchiaro, Gianpietro Dotti

508 4:45 PMIL-33/ST2 Triggering of IL-9-Secreting T Cells: From Proteomics to TherapeuticsAbdulraouf ramadan, Jilu Zhang, Mohammad Abu Zaid, lauren Taylor, heather A. o’leary, reuben Kapur, helmut hanenberg, hal e. broxmeyer, Mark h. Kaplan, Sophie Paczesny

509 5:00 PMRegulated Expression of IL-12 as Gene Therapy Concomitant with Blockade of PD-1 for Treatment of GliomaJohn A. barrett, hongliang Cai, John Miao, Margaret Murray, Paul Gonzales, Suma Krishnan, Francois lebel, laurence J. n. Cooper

510 5:15 PMTreating Epithelial and Metastatic Malignancies with Glycoform-Specific Chimeric Antigen ReceptorsAvery D. Posey, Alina boestaneau, laura A. Johnson, Carl h. June

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511 5:30 PMEngineered Donor Marrow Macrophages Phagocytose Cancer Cells and Aggressively Shrink Solid Tumor Xenografts Compared to Tumor Associated MacrophagesCory Alvey, Kyle Spinler, Manu Tewari, Dennis Discher

512 5:45 PMThe Cytokine Release Syndrome Crucially Contributes to the Anti-Leukemic Effects of CD44v6 CAR-T CellsMargherita norelli, Monica Casucci, barbara Camisa, laura Falcone, Aurore Saudemont, luigi naldini, Fabio Ciceri, Claudio bordignon, Chiara bonini, Attilio bondanza

Oral Abstract Session 334Cancer-Oncolytic DNA Viruses4:00 PM - 6:00 PMroom: Virginia Suite

513 4:00 PMArmed-Ad Gene Therapy Expressing PD-L1 Minibody Enhances the Anti-Tumor Effect of Adoptively Transferred Chimeric Antigen Receptor T-Cells for Solid Tumor TreatmentAmanda rosewell Shaw, Kiyonori Tanoue, norihiro Watanabe, Caroline Porter, Malcolm brenner, Masataka Suzuki

514 4:15 PMClinical Trials of Oncolytic Adenovirus-Mediated Gene TherapySvend o. Freytag, benjamin Movsas, hans Stricker

515 4:30 PMOncolytic Adenovirus Armed with Cytokines Enhances CAR-T Cell Efficacy in Pancreatic Tumor ModelKeisuke Watanabe, John Scholler, Sonia Guedan, Shannon e. McGettigan, yanping luo, Siri Tähtinen, Suvi Parviainen, riikka havunen, Mikko Siurala, Akseli hemminki, Carl h. June

516 4:45 PMA Novel Oncolytic Adenovirus Expressing Tumor Microenvironment Stimulators to Evoke and Facilitate Anti-Tumor Immune ResponsesJessica Wenthe, emma eriksson, ioanna Milenova, rafael Moreno, ramon Alemany, Angelica loskog

517 5:00 PMAnti-Tumor Immunity Is Elicited by a Conditionally Replicative Adenovirus in Canine Osteosarcomabruce F. Smith, Payal Agarwal, elizabeth Gammon, David T. Curiel, igor Dmitriev, elena Kashentseva

518 5:15 PMTumor Associated Macrophages Mitigate Oncolytic Herpes Simplex Virus Efficacy in Part Through TGF-β Signalingnicholas Denton, Chun-yu Chen, brian hutzen, Jennifer leddon, Pin-yi Wang, Thomas Scott, Mark Currier, Tim eubank, Timothy P. Cripe

519 5:30 PMIntravenous Application of CXCR4 Targeted Conditionally Replicative Adenovirus with Fiber and Hexon Modifications to Pancreatic CancerJing li huang, Christopher larocca, Kari Jacobsen, Sergey Kaliberov, David T. Curiel, Julia Davydova, Masato yamamoto

520 5:45 PMArming a Tumor Targeted Oncolytic HSV Vector with MMP9 for Enhanced Distribution and Killing ActivityPaola Sette, Aofei li, Marco Marzulli, Daniela leronni, Mingdi Zhang, William Goins, balveen Kaur, Chelsea bolyard, nduka Amankulor, Joseph Glorioso, Paola Grandi

Oral Abstract Session 335Cell Therapies4:00 PM - 6:00 PMroom: Washington 1-2

521 4:00 PMPlatelets Transfusion New Role as Brain Therapeutics for Acute Neuronopathic Gaucher DiseaseMei Dai, xiaohong Wang, benjamin liou, Venette inskeep, Gregory Grabowski, ying Sun, roscoe brady, Dao Pan

522 4:15 PMMurine iPSC-Derived Macrophages Improve the In Vivo Disease Phenotype of Pulmonary Alveolar Proteinosis Due to Csf2rb DeficiencyAdele Mucci, Jessica Kunkiel, Mania Ackermann, Takuji Suzuki, Christine happle, Sebastian brennig, elena lopez-rodriguez, Jens P. bankstahl, Mark P. Kühnel, Miriam hetzel, Axel Schambach, Gesine hansen, bruce Trapnell, nico lachmann, Thomas Moritz

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523 4:30 PMHuman Umbilical Vein Endothelial Cell, HUVEC, Co-Culture Promotes Robust Expansion and Maintains Phenotypic Integrity of Rhesus Hematopoietic Stem and Progenitor Cells, HSPC, Prior to Autologous TransplantationSandhya r. Panch, idalia yabe, Diego espinoza, Chuanfeng Wu, So gun hong, Stephanie Sellers, Allen Krouse, Aylin bonifacino, robert Donahue, Cynthia Dunbar

524 4:45 PMInjectable BMP-2 Gene-Activated Scaffold for the Repair of Mouse Cranial Bone Defecthang lin, ying Tang, Kai Sun, Jingwen xue, Weifeng yin, bing Wang, rocky Tuan

525 5:00 PMDirected Molecular Evolution of Transcription Factors in Mammalian Cells for Enhanced Directed Cell DifferentiationAmi M. Kabadi, Tyler S. Klann, Malathi Chellappan, Jennifer Kwon, Timothy e. reddy, Charles A. Gersbach

526 5:15 PMSpecific Companion Cells Enhance the Engraftment of Ex-Vivo Expanded HSCs (CD34+/CD38-/CD90+)nikoleta Psatha, Thalia Papayannopoulou

527 5:30 PMImprovement of Pre-Clinical Non-Human Primate Model for Pluripotent Stem Cell Based Therapies by Introducing Marker Genes in Safe Harbor Locusravi Chandra yada, So Gun hong, Jizhong Zou, Kyujoo Choi, Arnaud Carpentier, T. Jake liang, randall Merling, Colin Sweeney, harry Malech, Moonjung Jung, Marcus Corat, youngshun lin, ilker Tunc, xujing Wang, Maryknoll Palisoc, Stefania Pittaluga, Thomas Winkler, Cynthia Dunbar

528 5:45 PMTowards Personalized Cell Therapy for Cancer: Tumor-Homing Human Induced Neural Stem CellsJuli bago, onyi okolie, raluca Dumitru, Matt ewend, Shawn hingtgen

Poster Session III 6:00 PM - 8:00 PMroom: exhibit hall C & b South

RNA Virus Vectors

529Lentiviral Vectors with a Reduced Splicing Interference Potential Have a Significantly Improved Safety Profile In VivoDaniela Cesana, Pierangela Gallina, laura rudilosso, Andrea Calabria, Giulio Spinozzi, ivan Merelli, luciano Milanesi, eugenio Montini

530Lentiviral Vector Particles Pseudotyped with Wild-Type Baboon Endogenous Retrovirus (BAEV) Glycoprotein Outperform VSV-G Particles in Transducing Human CD34+ Cells Isolated from Cytokine-Mobilized Peripheral Blood or Bone MarrowPhillip hargrove, yoon-Sang Kim, Matthew Wielgosz, Arthur W. nienhuis

531Computational Pipeline for the Identification of Integration Sites and Novel Method for the Quantification of Clone Sizes in Clonal Tracking Studieslorena leonardelli, Danilo Pellin, Serena Scala, Francesca Dionisio, luca basso ricci, Davide Cittaro, Clelia Di Serio, Alessandro Aiuti, luca biasco

532Stable Transcriptional Repression, Gene Excision, and Parastism of HIV by Conditionally Replicating Lentiviral Vectorsuriel A. lopez-lemus, Amanda Ackley, Sheena Saayman, heather embree, boro Dropulic, ramesh Akkina, Paige Charlins, Marc S. Weinberg2, Kevin V. Morris

533Genomic Excision of PiggyBac Transposon Cassettes by Lentiviral Protein Transduction of GagPol-Fused, Excision-Only PiggyBac TransposaseKristian A. Skipper, Sofie Andersen, rasmus o. bak, yujia Cai, Jacob G. Mikkelsen

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534Preparation for a First-in-Man Lentivirus Trial in Cystic Fibrosis Patientsuta Griesenbach, eric W. F. W. Alton, Jeffery M. beekman, Christopher A. boyd, Jane C. Davies, lee A. Davies, Johanna F. Dekkers, Deborah r. Gill, Mamoru hasegawa, Tracy e. higgins, Takashi hironaka, Makoto inoue, Stephen C. hyde, Alastair J. innes, ian A. Pringle, Kamila M. Pytel, Stephanie G. Sumner-Jones, Shu Tsugumine, Marguerite W. Wasowicz

536The Viral Transduction Enhancer Vectofusin-1 Is a Nanofibrillar Peptide Capable of Increasing the Contact between Viral Vectors and Target CellsSaliha Majdoul, louic Vermeer, loic hamon, Justine Wolf, Ababacar K. Seye, nathalie holic, David Pastre, Daniel Stockholm, burkhard bechinger, Anne Galy, David Fenard

537New Graph-Based Algorithm for Comprehensive Identification and Tracking Retroviral Integration SitesAndrea Calabria, Stefano beretta, ivan Merelli, Giulio Spinozzi, Stefano brasca, Fabrizio benedicenti, erika Tenderini, Alessandra biffi, eugenio Montini

538Transient Non-Viral RNA Delivery Mediated by a Lentiviral ParticlePascale bouillé, régis Gayon, Alexandra iché, Christine Duthoit, lucille lamouroux, Jean-Christophe Pagès

AAV Vectors III

539Screening for Recombinant Adeno-Associated Viral Vectors That Selectively Transduce Hepatitis B Virus Infected CellsGustavo de Alencastro, Katja Pekrun, Mark Kay

540Identification of a 16 Amino Acid-Long Motif in the N-Terminal Half of the AAV9 Capsid Essential for Persistence in the Blood for an Extended Period of up to 3 DaysKei Adachi, hiroyuki nakai

541Use of Endogenous Viral Elements to Deduce the Sequence of an Ancient Adeno-Associated Virus That Circulated Amongst Australian Marsupials ~30 Million Years AgoClaus V. hallwirth, richard h. Smith, Michael Westerman, nicola A. hetherington, yu-Shan Tseng, Sylvain Cecchini, Tamas Virag, Mona-larissa Ziegler, igor b. rogozin, eugene V. Koonin, Mavis Agbandje-McKenna, ian e. Alexander, robert M. Kotin

542Novel Barcode-Based In Vivo Screening Method for Generating De Novo AAV Serotypes for CNS-Directed Gene TherapyMarcus Davidsson, Gang Wang, Patrick Aldrin-Kirk, Morgan hartnor, Tomas björklund

543In Vivo Beta-Cell-Targeted Gene Editing by AAV VectorsSalma G. Morsy, Jason Tonne, yaxi Zhu, Paul belmonte, yasuhiro ikeda

544 Abstract Withdrawn

545Syngeneic AAV Pseudo-Vectors Potentiates Full Vector TransductionQizhao Wang, biao Dong, Katie A. Pokiniewski, Jenni Firrman, Zhongren Wu, Mario P.s. Chin, yong Diao, ruian xu, Weidong xiao

546Development of MFP-Inducible System for AAV5 Gene Therapy of Chronic Diseases in the LiverJolanda liefhebber, raygene Martier, richard van logtenstein, harald Petry, Pavlina Konstantinova

547AAV Preparations Contain Contamination from DNA Sequences in Production Plasmids Directly Outside of the ITRsMark A. brimble, Junfang Zhou, Christopher Morton, Michael Meagher, Amit C. nathwani, John T. Gray, Andrew M. Davidoff

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548Recombinant AAV3 Serotype Vectors Generated by Using AAV3 ITRs and AAV3 Rep Proteins Efficiently Transduce Human Liver Cancer Cell Lines In Vitro and Human Liver Tumors in a Murine Xenograft Model In VivoChen ling, Zifei yin, Jun li, Daniel Zhang, George Aslanidi, Arun Srivastava

549Fine Tuning of Transduction Efficiency of rAAV Vectors via Modulation of Capsid Compositionoleksandr Kondratov, Damien Marsic, Mario Mietzsch, regine heilbronn, Sergei Zolotukhin

550Developing an Optimal Insulin Augmentation Therapy to Improve AAV Gene Transfer to Skeletal Muscle and Liver in MiceSean Carrig, Mitchell J. Wopat, Ashley T. Martino

551Enhancement of Gene Therapy Treatment for Sandhoff Disease Through Complimentary Drug Therapyevan J. r. Woodley, Karlaina osmon, Patrick Thompson, Subha Karumuthil-Melethil, Steven Gray, Jagdeep Walia

552AAV9-Utrophin Restores Muscle Function in Dystrophic MiceShira T. rosenblum

553Comparision of AAV Serotypes for Gene Delivery to iPSC Derived RPEVidyullatha Vasireddy, Thu T. Duong, Jeannette l. bennicelli, Jean bennett

554Serotype Comparison of AAV Transduction in Adipose-Derived Stem CellsPriyanka Sharma, Sunishka M. Wimalawansa, Gregory C. Gould, r. Michael Johnson, Katherine J. D. A. excoffon

555Development of a Post-Exposure Treatment for Ebola Virus Infections Based on AAV Vectors and Zmapp Antibody CocktailMarc-Andre robert, Amine Kamen, Gary Kobinger, rénald Gilbert, bruno Gaillet

556Comparative In Vitro Transduction Efficiency of AAV Vector Serotypes 1-9 in Different Cellular ModelsThu T. Duong, Vidyullatha Vasireddy, Carrie e. Merkel, Jeannette l. bennicelli, Jean bennett

Targeted Genome Editing III

557Targeted CYBB Minigene Insertion into the CYBB Locus for Correction of X-CGD iPSCs Requires Intronic Elements for ExpressionColin l. Sweeney, uimook Choi, Jizhong Zou, randall K. Merling, Suk See De ravin, harry l. Malech

558 Abstract Withdrawn

559Induction of Fetal Hemoglobin in Adult Erythroblasts by Genome Editing of the Beta-Globin LocusChiara Antoniani, Annalisa lattanzi, Giulia Pavani, Vasco Meneghini, elisa Magrin, leslie Weber, Matthew Porteus, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio

560Generation of Functional Regulatory T Cells by FOXP3 Gene Transfer into CD4 T Cell from IPEX Patientsemmanuelle Six, nelly Sigrist, David Zemmour, Marie-Andrée bessard, Fabienne Mazerolles, Samia Martin, Fulvio Mavilio, Frederic rieux-laucat, Christophe benoist, isabelle André-Schmutz, Marina Cavazzana

561CRISPR-Induced Deletion (CinDel) Method Allows Permanent and Efficient Restoration of the DMD Gene Reading Frame in Duchenne Patient Myoblasts and Preserves Truncated Dystrophin StructureJean-Paul iyombe-engembe, Dominique l. ouellet, xavier barbeau, Joël rousseau, Pierre Chapdelaine, Patrick lagüe, Jacques P. Tremblay

562Fetal Hemoglobin Expression Can Be Increased by Targeted Disruption of the Gamma Hemoglobin Promoter in Human Peripheral Blood Stem CellsChristopher T. lux, Queenie Vong, Julia yang, brandon K. hadland, Andy Scharenberg

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563Analysis of DNA Repair Pathway Choice Upon Induction of Double-Strand Breaks by Engineered Nucleaser. ileng Kumaran, Jingjing li, William S. Dynan, Matthew h. Porteus, David l. Spector

564The Cytotoxic Effect of RNA-Guided Endonuclease Cas9 on Human Hematopoietic Stem and Progenitor Cells (HSPCs)Kyung-rok yu, Marcus A. F. Corat, Jean-yves Metais, Cynthia e. Dunbar

565Human Dystrophin Expression in Rag/Mdx Mice Muscles Following the Graft Off Genetically Corrected Dystrophic hiPSCsWilliam-Édouard Gravel, Dominique l.oulette, Chantale Maltais, Catherine Gérard, li hongmei l., Akitsu hotta, Jacques-P. Tremblay

566Targeted Lentiviral Vector Integration Mediated by the AAV Rep78 ProteinPingjuan li, Jizhong Zou, Michael T. Morreale, Michael P. Marino, Jakob reiser

567CRISPR-Cas9 Mediated Gene Editing in a Monogenic Form of Alzheimer’s Diseasebence Gyorgy, Martin ingelsson, Camilla loov, Shuko Takeda, lars lannfelt, bradley T. hyman, xandra o. breakefield

568 Abstract Withdrawn

569Precision Editing of the WAS Locus via Homologous Recombination in Primary Human Hematopoietic Cells Mediated by Either TALEN or CRISPR/Cas Nucleasesiram F. Khan, yupeng Wang, Courtnee Clough, Swati Singh, King hung, David J. rawlings

570Transient Reconstitution of Cas9 Protein for Safer Gene Editing In Vivo - A Ferry Tale Starring rAAV CapsidDan Wang, Karen Tran, blake y. Wang, Guangchao xu, Mengxin li, hong Ma, ran he, Qin Su, Phillip D. Zamore, Guangping Gao

571Safe Harbor Targeting of IL2RG Expression Cassettes for Gene Therapy of X-Linked Severe Combined Immunodeficiency (SCID-X1)Sheng Zhou, Zhijun Ma, Taihe lu, Mark A. brimble, yong-Dong Wang, Andrew M. Davidoff, brian P. Sorrentino

572Successful Generation of CAR+PD-1- Primary T Cells Using Cas9-Mediated Genome EditingGrant Welstead, Justin Fang, Chris nye, Frank buquicchio, McKensie Collins, Will Selleck, hari Jayaram, ruth Salmon, Alex baturevych, Sherri Mudri, Jon Jones, David bumcrot, blythe D. Sather

573Vector Free Genome Editing of Immune Cells for Cell TherapyTia DiTommaso, Jonathan Gilbert, howard bernstein, Armon Sharei

574Lentivirus Pre-Packed with Cas9 Protein for Safer Gene EditingManjunath Swamy, Jang-Gi Choi, ying Dang, Sojan Abraham, hongming Ma, hua Guo, yujia Cai, Jacob Giehm Mikkelsen, haoquan Wu, Premlata Shankar

575High Content Analysis of CRISPR-Cas9 Gene-Edited Human Embryonic Stem CellsJared Carlson-Stevermer, Madelyn Goedland, benjamin Steyer, Arezoo Movaghar, Meng lou, lucille Kohlenberg, ryan Prestil, Krishanu Saha

576DNA Ends Matter: The Impact of Using CRISPR/Cas9 Variants on DNA Repair Pathway Choices and Editing Profiles at the HBB LocusTanushree Phadke, Anne bothmer, Christina lee, hayat Abdulkerim, luis barrera, S. Moss, hari Jayaram, Cecilia Cotta-ramusino

577Empower Multiplex CRISPR-Mediated Gene Manipulation with Self-Cleaving Ribozymes and tRNAli xu, Mona el refaey, lixia Zhao, Jing xu, yandi Gao, renzhi han

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578 Abstract Withdrawn

579Meganucleases as an Efficient Tool for Genome EditingVictor V. bartsevich, John Morris, Ginger Tomberlin, Caitlin Turner, Wendy Sharer, Michael G. nicholson, Jeff Smith, nag Kollu, rachel James, robin Armstrong, Matthew hirsch, Derek Jantz

Oligonucleotide Therapeutics II

580Prevention of Airway Inflammation by Simultaneous Inhibition of NFkB and STST6 Using Chimeric Decoy Oligonucleotides in a Mouse Model of AsthmaTetsuo Miyake, Takashi Miyake, hizuki hamada, ryuichi Morishita

581Correcting Exon Skipping Splicing Defects in BTK RNA by Using Bifunctional OligonucleotidesC. i. edvard Smith, burcu bestas, Janne J. Turunen, Jesper Wengel

582 Abstract Withdrawn

583Translating RNAi Therapy for Spinocerebellar Ataxia 1 to the ClinicMegan S. Keiser

584Myoblast Fusion Mediates Morpholino Entry and Exon Skipping In Dystrophic MuscleJames S. novak, Marie nearing, Marshall W. hogarth, Maria C. Vila, Jessica F. boehler, eric P. hoffman, Kanneboyina nagaraju, Sebahattin Cirak, Terence A. Partridge

585Long Non-Coding RNA NEAT1 Functions as a ceRNA to Regulate E2F3 Expression by Sponging miR-377-3p in Non-Small Cell Lung CancerChengcao Sun

586Dimerization Inhibition of HIV-1 RNA by 2’-Deoxy-2’-fluoro-beta-D-arabinose Nucleic Acid (2’-FANA) Modified Antisense Oligonucleotides Results in Potential Inhibition of Viral ExpressionMayumi Takahashi, haitang li, Veenu Aishwarya, Masad J. Damha, John J. rossi

587Genetic Delivery of a miRNA Cluster with Polycistronic siRNAs Reduces Expression of Epidermal Growth Receptor in Human Glioblastoma CellsMartin J. hicks, imari Patel

588MYCN Silencing Using RNA Interference Causes Apoptosis and Differentiation in MYCN Amplified Neuroblastoma Cell Linesruhina Maeshima, Aristides D. Tagalakis, Andrew W. Stoker, Stephen l. hart

589Short Hairpin RNAs Based on miR-451 Show Potent Knockdown without Passenger Strand Activityerik Kaadt, lars Aagaard

590Anti-EGFR Aptamer-Conjugated Lipoplexses, a Novel Gene Delivery Strategy for Triple Negative Breast Cancer TherapyMin Woo Kim, hwa yeon Jeong, Seong Jae Kang, in ho Jeong, Moon Jung Choi, young Myoung you, Chan Su im, yong Serk Park

591The OTTC Plasmid Manager: A Comprehensive Database for Archiving Plasmid DNA Sequenceemily J. heathward, Giun Sun, ron u. Domingo, Calvin A. Thomas, Christopher T. richie, brandon harvey

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592High Quality Grade Plasmid DNA for Indirect Clinical ApplicationsMarco Schmeer, ram Shankar, Tatjana buchholz, Martin Schleef

Non-Viral Gene Transfer and Therapy II

593Anti-Angiogenic Therapy for Pancreatic Cancer by Systemic Delivery of Messenger RNA Using Polyplex Nano MicelleSatoshi uchida, hiroaki Kinoh, Takehiko ishii, Akitsugu Matsui, Theofilus A. Tockary, Kaori M. Takeda, hirokuni uchida, Kensuke osada, Keiji itaka, Kazunori Kataoka

594Exosome-Associated AAV Enhances Retinal Transduction Following Intravitreal Injectionbence György, Sarah Wassmer, livia Carvalho, Casey Maguire, luk h. Vandenberghe

595Enhanced Liver Transduction and Efficient Protection from Pre-Existing Neutralizing Antibodies with Exosome-Associated AAV8 VectorsAmine Meliani, Zachary Fitzpatrick, Florence boisgerault, Giuseppe ronzitti, Fanny Collaud, Severine Charles, Casey A Maguire, Federico Mingozzi

596Effect of Chemokine/Cytokine Delivered by Nanoparticles on Tumor Migration of Neuroblastoma Targeting Chimeric Antigen Receptor T CellsMai T. huynh

597Development of Non-Viral Gene Delivery Systems with Antibacterial Effects Deliverable via Aerosol for Lungs Gene TherapyAngélique Mottais, Tony le Gall, Mathieu berchel, yann Sibiril, Paul-Alain Jaffrès, Tristan Montier

598TARGTCNS for the Treatment of Central Nervous System Disordersnir Shapir, reem Miari, Cathryn A. Sundback, irina Pomerantseva, Joshua P. Aronson, husam A. Katnani, olive Mwizerwa, herman Tse, haim Goltsman, yaron Fuerst, yonit Moraly h, Shany blum, Sagi harnof, Garry A. neil

599Microfluidic Based Synthesis of Folate-Targeted Monomolecular siRNA-Lipid Particlesrafał Krzysztoń, bässem Salem, Gerlinde Schwake, Joachim o. rädler

600 (Moved to Poster Session ii after Abstract 345)Development and Characterisation of Polymeric Nanocapsules Containing Macromoleculeomar S. Abu Abed, Amal A. elkordy, Ching Chaw, lee Williams, Marc Carlile

601An Optimized Approach to Non-Viral Transfection of Brain Tumor Initiating Cells Derived from Embryonal Tumor with Multilayered Rosettes Using Cationic ReagentsCharlie y. hsu, Myra J. Chen, Derrick e. rancourt, Jennifer A. Chan

602Stable In Vitro Reprogramming of Lentiviral Vectors for Target Delivery of Genetic CargoZhilei C. Chen, nagarjun Kasaraneni, Ana M. Chamoun-emanuelli

603Feldan Shuttle - Advanced Protein Delivery Technology for Cell TherapyThomas Del’Guidice, Jean-Pascal lepetit-Stoffaes, Joannie roberge, louis-Jean bordeleau, Mario harvey, Alain Garnier, David Guay

Delivery Questions for Neural Applications

604Comparison of Intra-Cisterna Magna and Lumbar Puncture Intrathecal Delivery of scAAV9 GeneTherapy for Giant Axonal Neuropathyrachel M. bailey, Alejandra rozenberg, Caleigh Toppins, Steven J. Gray

605Cerebrospinal Fluid for Delivery of AAV Gene Therapy in GM1 GangliosidosisAmanda l. Gross, heather Gray-edwards, bryan Murdock, Amanda Taylor, brandon brunson, Ashley n. randle, lorelei Stocia, Sophia Todessa, Jaclyn lata, Miguel Sena-esteves, Douglas r. Martin

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606Use of a Bicistronic Vector to Silence SOD1 in Motoneurons and Astrocytes for the Treatment of Familial Amyotrophic Lateral SclerosisJulianne bobela-Aebischer, Cylia rochat, nathalie bernard-Marissal, Patrick Aebischer, bernard l. Schneider

607Organotypic Culture of Adult Porcine Retina as an In Vitro Screening Model for AAV-Mediated Gene Therapy in OphthalmologyDiana Cepeda, Tawny neal, rangoli Aeran, Mehdi Gasmi, Annahita Keravala

608In Silico Reconstructed Ancestral Adeno-Associated Viruses Transduce Mouse Anterior Segmentru xiao, li Wang, livia Carvalho, Sarah Wassmer, luk h. Vandenberghe

609Transduction of the Central Nervous System with the LTR1 Lentiviral Backbonerajvinder Karda, John r. Counsell, Conrad A. Vink, Dany P. Perocheau, Suzanne M. K. buckley, Joanne ng, Simon n. Waddington, Steven J. howe

610An Inducible AAV Vector Mediating GDNF Signal Transduction at Clinically-Acceptable Sub-Antimicrobial Doxycycline Dosesliliane Tenenbaum, Abdelwahed Chtarto, Marie humbert-Claude, olivier bockstael, Atze T. Das, Sébastien boutry, ludivine breger, bep Claver, robert n. Müller, olivier De Witte, Marc levivier, Cecilia lundberg, ben berkhout

611Promoter Evaluation of AAV Gene Therapy in the Central Nervous System for Feline Niemann-Pick Type C Diseasebrittney l. Gurda, Gary Swain, Jessica h. bagel, Maria Prociuk, Steven J. Gray, Caitlin Fitzgerald, Patricia o’Donnell, Charles h. Vite

612Systemic AAV Injection in Guinea Pigs and Non-Human Primates Targets the Enteric Nervous SystemSara e. Gombash, Christopher J. Cowley, Julie A. Fitzgerald, Kathryn hook, emily Armstrong, brian K. Kaspar, Andrew J. Fischer, levi Todd, eric C. bielefeld, Kevin D. Foust

613A Comparison of CNS Transduction After Systemic versus Cranial Delivery of an AAV2/9 CamKII Promoter-eGFP Vector in MiceAlana l. Gibson, randy J. Chandler, ian M. Williams, Arturo A. incao, Forbes D. Porter, William J. Pavan, Charles P. Venditti

614Microfluidic Manufacture of RNA-Lipid Nanoparticles Leads to Highly Efficient Delivery of Potent Nucleic Acid Therapeutics for Controlling Gene ExpressionGrace Tharmarajah, eric ouellet, oscar Seira, Jie liu, Anitha Thomas, Timothy leaver, Andre Wild, yuping li, yu Tian Wang, Wolfram Tetzlaff, Carl hansen, Pieter Cullis, James r. Taylor, euan ramsay

615Choroid Plexus-Targeted Viral Gene Therapy for Lysosomal Storage Diseaseseun-young Choi, Shih-hsin Kan, S. Q. le, Patricia i. Dickson, Stephen G. Kaler

616Tailored Expression of a Transgene to Specific Cell Types in the Central Nervous System After Peripheral Injection of AAV9eloise hudry, Jonathan Dashkoff, Paul lerner, Shuko Takeda, nhi Truong, Casey Maguire, bradley T. hyman

617Identification of Novel AAV Capsid Variants with Enhanced Tropism for the Canine Outer Retina Following Intravitreal Deliveryleah C. byrne, Meike Visel, Valerie Dufour, Felipe Pompeo Marinho, Gustavo D. Aguirre, William A. beltran, John G. Flannery, David V. Schaffer

618Directional Transduction of AAV-5 Vectors in the NHP Brainbas blits, lluis Samaranch, John bringas, Waldy San Sebastian, Krystoff bankiewicz, harald Petry

619AAV9 Transduction Is Similar in Adult and Aged Mouse Brains Following Intraparenchymal InjectionSara e. Gombash, Julie A. Fitzgerald, Christopher J. Cowley, Mitchell G. neides, emily Armstrong, Diana M. norden, Jonathan P. Godbout, Kevin D. Foust

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620Use of Transgenic Mice to Quantify Transduction Efficiency and Specificity of Novel AAV Vectors in RetinaMiranda l. Scalabrino, K. Tyler McCullough, Shreyasi Choudhury, James J. Peterson, Seok-hong Min, Sanford l. boye, Shannon e. boye

621Widespread Gene Delivery to the Nonhuman Primate Brain for the Treatment of Huntington’s DiseasePiotr hadaczek, lisa Stanek, Agnieszka Ciesielska, Philip Pivirotto1, Catherine o’riordan, bryan Mastis, Waldy San Sebastian, John Forsayeth, Seng h. Cheng, Krystof S. bankiewicz, lamya S. Shihabuddin

Musculo-Skeletal Diseases II

622 (Moved to Poster Session ii after Abstract 390)Systemic β-Sarcoglycan Gene Therapy for Treatment of Cardiac and Skeletal Muscle Deficits in LGMD2Eeric Pozsgai, Danielle Griffin, Kristin heller, Jerry Mendell, louise rodino-Klapac

623Dystrophin Exon 52-Deleted Pigs as a New Animal Model of Duchenne Muscular Dystrophy: Its Characterization and Potential as a Tool for Developing Exon Skipping Therapyyusuke echigoya, nhu Trieu, Kana hosoki, William Duddy, Terence Partridge, eric hoffman, Joe Kornegay, Christopher rogers, Toshifumi yokota

624A Single Neonatal Delivery of an Exon 2 Directed AAV9.U7snRNA Vector Results in Long-Term Dystrophin Expression That Prevents Pathologic Features in the Dup2 Mousenicolas Wein, Tabatha r. Simmons, Felecia Gumienny, Jacqueline yurkoski, nianyuan huang, Francesco Muntoni, Kevin M. Flanigan

625 (Moved to Poster Session ii after Abstract 386)RNAi Therapy for Dominant Limb Girdle Muscular Dystrophy Type 1Alindsay M. Wallace, Carlee r. Giesige, Danielle A. Griffin, louise r. rodino-Klapaca, Scott Q. harper

626Directing Skeletal Myogenic Progenitor Cell Lineage Specification with CRISPR/Cas9 Transcriptional ActivatorsJennifer b. Kwon, Charles A. Gersbach

627Concerted Evolution of Pseudogenes Facilitated Contiguous Deletion of the Canine Dystrophin and TMEM47 Genes by Homologous RecombinationDaniel h. Vanbelzen, Alock Malik, Joe Kornegay, hansell h. Stedman

628Transposons Expressing Full-Length Human Dystrophin Enable Genetic Correction of Dystrophic Mesoangioblasts and iPS-Derived Mesoangioblast-Like CellsMariana loperfido, Susan Jarmin, Sumitava Dastidar, Mario Di Matteo, ilaria Perini, Marc Moore, nisha nair, ermira Samara-Kuko, Takis Athanasopoulos, Francesco Saverio Tedesco, George Dickson, Maurilio Sampaolesi, Thierry VandenDriessche, Marinee K. Chuah

629Intravenous Delivery of a MTMR2-Encoding AAV9 Vector Extends Lifespan and Improves Muscle Function in Mice with X-Linked Myotubular Myopathynathalie Danièle, laura Julien, Christelle Moal, Thibaut Jamet, Alban Vignaud, Ana buj bello

630AAV Transduction of a Truncated Dysferlin Improves DysferlinopathyTelmo llanga, bryan Sutton, nadia nagy, Matthew hirsch

631Rankl Knock-Out Mesenchymal Stromal Cells Have an Unexpected Osteogenic Differentiation Defect Which Is Improved by a RANKL-Expressing Lentiviral VectorCiro Menale, Francesca Schena, lorenzo Diomede, lucia Sergi Sergi, lucia Susani, eleonora Palagano, elisabetta Traggiai, Cristina Sobacchi, Anna Villa

632Alpha-1 Antitrypsin Gene Therapy Prevented Bone Loss in an Ovariectomy Induced Osteoporosis Mouse ModelMohammad A. Akbar, Jay J. Cao, yuanqing lu, David nardo, Mong-Jen Chen, Ahmed S. elshikha, rubina Ahamed, Mark brantly, lung-ji Chang, lexie Shannon holliday, Sihong Song

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633A Reproducible and Reliable Non-Invasive Approach to Assess the Efficacy of Gene Therapy in mdx Mice with Relevance to Clinical Trialsyafeng Song, Mihail Petrov, Shira rosenblum, leon Morales, hansell h. Stedman

634 Abstract Withdrawn

635Effects of Mechanical Strain on Zmpste24-Deficient Prematurely Aged Muscle-Derived Stem CellsAndrea liebowitz, elizabeth Morris, Alex Scibetta, xiaodong Mu, Johnny huard

636Enhanced Skeletal Muscle Regeneration in Mice After Multiple PregnanciesAlex C. Scibetta, Aiping lu, Johnny huard

Cancer-Immunotherapy, Cancer Vaccines III

637Targeting of Myeloid Leukemia by IL-10-Engineered Human CD4+ Tr1 CellsGrazia locafaro, Grazia Andolfi, Fabio russo, barbara Camisa, Fabio Ciceri, Angelo lombardo, Attilio bondanza, Maria Grazia roncarolo, Silvia Gregori

638TGFBeta Signaling Blockade within PSMA Targeted CAR Human T Cells for the Eradication of Metastatic Prostate CancerChristopher Kloss, Jihyun lee, Carl June

639Oncolytic Adenovirus Expressing IFN Alpha Works Synergistically with ChemoradiationAmanda o. Salzwedel, Christopher larocca, Joohee han, Julia Davydova, Masato yamamoto

640Ex Vivo AKT Inhibition Promotes the Generation of Potent CD19CAR T Cells for Adoptive Immunotherapyryan Z. urak, Chinglam W. Wong, elizabeth budde, Christine e. brown, Sandra Thomas, Stephen J. Forman, xiuli Wang

641Highly Efficient, ZFN-Driven Knockout of Surface Expression of the T-Cell Receptor and HLA Class I Proteins in Human T-Cells for Enhancing Allogeneic Adoptive Cell TherapiesGary K. lee, Pei-qi liu, nimisha Gandhi, lynn Truong, Andreas reik, Stephen lam, Sarah J. hinkley, Jeffrey C. Miller, David e. Paschon, lei Zhang, edward J. rebar, Michael C. holmes

642Oncolytic Vaccines with Modified Tumor Epitopes for Cancer ImmunotherapyCristian Capasso, Aniket Magarkar, Victor Cerveca Carrascon, Martin Muller, Mariangela Garofalo, lukasz Kuryk, Davide Cardella, Karita Peltonen, Alex bunker, Vincenzo Cerullo

643Various Forms of CD40L Encoded as an Immune Plasmid Adjuvant Generate Unique Anti-Cancer DNA Vaccine Induced ResponsesMegan Wise, Daniel Villarreal, lumena louis, Jian yan, Matthew Morrow, niranjan Sardesai, David Weiner

644In Vivo Expression of Functional DNA Monoclonal Antibodies Targeting IL-6 and CD126Sarah T. C. elliott, Ami Patel, Seleeke Fllingai, Megan C. Wise, Karuppiah Muthumani, David b. Weiner

645T-Cell Activating Mesenchymal Stem Cells as a Biotherapeutic for HCCArpad Szoor, Zhuyong Mei, Adrian Gee, Andras heczey, Stephen Gottschalk

646Development of a Novel Chimeric Antigen Receptor as a Therapy Against Solid TumorsJohn M. Szarejko, Thomas M. yankee, Gary D. Myers

647Efficient Targeting of T Cell Malignancies In Vitro and In Vivo Using CD4-Specific Chimeric Antigen Receptor (CAR)-Engineered NK CellsKevin G. Pinz, elizabeth yakaboski, Alexander Jares, hua liu, Amelia e. Firor, Masayuki Wada, lai-han leung, xun Jiang, yupo Ma

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648Mitigating Tumor Escape: Tandem Anti-CD20- and CD19 SCFV-Based Chimeric Antigen Receptors (CARs) in Leukemia/Lymphomaying xiong, Darong Wu, boro Dropulic, rimas orentas, Dina Schneider

649Developing FGFR4 Chimeric Antigen Receptor CAR T Cell Therapy Against Rhabdomyosarcomanityashree Shivaprasad, ying xiong, Marielle yohe, Dina Schneider, Jack Shern, Sivasubramanian baskar, Dimiter Dimitrov, Paul Sorenson, rimas orentas, Javed Khan

650Development of a Manufacturing Process Using Monte Carlo Simulations to Support KTE-C19 (Anti-CD19 CAR T Cells) Studies in LeukemiaKenny Choi, Marianna Sabatino, Vijay Chiruvolu, Marc better

651Integration of Dual Signal Inputs Strategies in Novel Chimeric Antigen Receptors to Control the CAR T-Cell FunctionsAlexandre Juillerat, Alan Marechal, Jean Marie Filhol, yannick Valogne, Valérie Guyot, Anne-Sophie Gautron, Julien Valton, Aymeric Duclert, laurent Poirot, Philippe Duchateau

652Culture and Characterization of Tumor-Infiltrating Lymphocytes of the Syrian HamsterMikko Siurala, Markus Vähä-Koskela, riikka havunen, Siri Tähtinen, Simona bramante, Suvi Parviainen, Akseli hemminki

653Sialyl Glycolipid Stage-Specific Embryonic Antigen 4 (SSEA4) - A Novel Target for CAR T Cell Therapy of Solid Cancersrita Pfeifer, Dominik lock, Andrea Aloia, Andreas bosio, Andrew Kaiser, olaf hardt, ian C. D. Johnston

Cancer-Oncolytic Viruses II

654Oncolytic Vesicular Stomatitis Virus Retargeted to the Tumor Stem Cell Marker CD133Dina Kleinluetzum, Alexander Muik, Julia hanauer, Sarah-Katharina Kays, Camilo Ayala-breton, Kah-Whye Peng, Christian J. buchholz

655CD133-Targeted Oncolytic Adenovirus Exhibits Anti-Tumor Effect by Attacking Colon Cancer Stem CellsMizuho Sato-Dahlman, yoshiaki Miura, Masato yamamoto

656Effects of STAT3 Inhibition on the Innate Immune Response to OV TherapyAlexander Weiss, George Koutras, Chelsea bolyard, balveen Kaur

657Enhancing Therapeutic Efficacy of an Oncolytic Virus with a Beta-1 Integrin Blocking Antibody, OS2966Tejaswini reddy nallanagulagari

658Bortezomib Treatment Sensitizes Oncolytic Virus Treated Tumors to NK Cell ImmunotherapyJi young yoo

659Oncolytic Adenovirus Loaded with Bioactive Modified Peptide as a Novel Approach to Treat CancerMariangela Garofalo, barbara iovine, lukasz Kuryk, Cristian Capasso, Mari hirvinen, Andrea Vitale, Marjo yliperttula, Maria Assunta bevilacqua, Vincenzo Cerullo

660Enhance Antitumor Effect by Combining Oncolytic Virus HF10 and Bevacizumab in the Treatment of Human Breast Cancer XenograftGewen Tan, hideki Kasuya, Zhiwen Wu, Tsutomu Fujii, Maki Tanaka, Saori Fukuda, Toyone Kikumori, nengquan Sheng, Zhigang Wang, yasuhiro Kodera

661Synergistic Anti-Tumor Efficacy of Immunogenic Adenovirus ONCOS-102 and Standard of Care Chemotherapy in Preclinical Mesothelioma Modellukasz Kuryk, elina haavisto, Mariangela Garofalo, Cristian Capasso, Mari hirvinen, Sari Pesonen, Tuuli ranki, lotta Vassilev, Vincenzo Cerullo

662An Oncolytic Adenovirus Gene Therapy Targeting Both Tumor Cell Survival and Desmoplasia in Pancreatic Cancerioanna Milenova, emma eriksson, rafael Moreno, ramon Alemany, Angelica loskog

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663Can Oncolytic Measles Virus Targeted to CD20 Recapitulate Any of the Effects of Rituximab in the Treatment of Acute Lymphoblastic Leukemia?Katharine bailey, Aditi Dey, Sarah Aref, lucile broyer, Adele K. Fielding

664Therapeutic Effect of Oncolytic Herpes Simplex Virus Type 1 (G47Δ) in Combination with Chemotherapy on Colorectal CancerShinya Abe, yasushi ino, hiroshi Fukuhara, Miwako iwai, Toshiaki Watanabe, Tomoki Todo

665Therapeutic Efficacy of Third Generation Oncolytic HSV-1 (G47Δ) for Glioma Cells with Stem Cell Propertyhirotaka ito, yasushi ino, Tomoki Todo

666Oncolytic Herpes Virotherapy Induces a Paracrine Death Signal Causing Synergistic Antitumor Efficacy with Aurora A Kinase Inhibitionleslee Sprague, Mark Currier, Tilat rizvi, brooke nartker, Chun-yu Chen, Pin-yi Wang, brian hutzen, Meghan Franczek, Jeffrey ecsedy, Joe Conner, nancy ratner, Timothy Cripe

667Natural Selection of a Neurovirulent Herpes Simplex Virus Type1 Strain via Passage in Human Glioma XenograftsKentaro Fujii

Cancer-Targeted Gene and Cell Therapy II

668Platelets Engineered to Express Interleukin-24 Inhibited Melanoma Tumor Growth in MiceJuan Fang, Ming yao, Weiqing Jing, beicheng Sun, bryon D. Johnson, David A. Wilcox

669rAAV-Based and Intraprostatically Delivered miR-34a Therapeutics for Efficient Inhibition of Prostate Cancer ProgressionJianzhong Ai, yi lu, Jia li, Qin Su, Qiang Wei, hong li, Guangping Gao

670A Novel Approach to Expand Antigen Recognition in Chimeric Antigen Receptorsrobert Moot, Sunil raikar, lauren Fleischer, Melissa Querrey, Christopher Doering, Trent Spencer

671Potent Anti-Tumor Effects of ApDCs (Aptamer Drug Conjugates) for Targeted Therapeutics in Pancreatic CancerSorah yoon, Kai-Wen huang, nagy habib, John rossi

672Hybrid AAV/Phage Vector Enhances Chemotherapy Efficacy Against CancerKeittisak Suwan, effrosyni Tsafa, Justyna Przystal, Paladd Asavarut, Amin hajitou

673Therapeutic Efficacy of Retroviral Replicating Vector (RRV) -Mediated Prodrug Activator Gene Therapy for Pancreatic CancerKazuho inoko, Kei hiraoka, Akihito inagaki, Mizuna Takahashi, Toshihiro Kushibiki, Koji hontani, hironobu Takano, Shoki Sato, Toru nakamura, Takahiro Tsuchikawa, Toshiaki Shichinohe, Douglas J. Jolly, noriyuki Kasahara, Satoshi hirano

674Insertional Mutagenesis to Identify Mechanisms of Cetuximab Resistance in Colorectal Cancerriccardo biavasco, Valentina Pirazzoli, Giulio Spinozzi, Andrea Calabria, erika Tenderini, Fabrizio benedicenti, eugenia Zanella, Andrea bertotti, livio Trusolino, eugenio Montini

675Exploring the Effects of Stroma-Derived PPARB Signaling in Colorectal Tumorigenesiseddie h. P. Tan, Walter Arthur Wahli, nguan Soon Tan

676An Engineered CAR T Cell Platform for Allogeneic Combination ImmunotherapyPhilippe Duchateau, Julien Valton, Alexandre Juillerat, laurent Poirot

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677Evaluation of Tumor Specific Promoters for Use in Conditionally Replicating Adenovirus Mediated Virotherapy of Canine LymphomaAbdul Mohin Sajib, Samantha Morici, Maninder Sandey, Payal Agarwal, bruce Smith

678Neural Stem Cell-Based Gene Therapy for Malignant Brain Tumor by Doubl-Stranded Adeno-Associated Viral Vectorsli-juan Wang, hsin-i Ma

679Heat Stress Elevate Cell Population That Express Cancer Stem Cell Markers in Different Types of Cancer Cell LineAmer T. Tawfeeq Al-Saeedi

680TransfeX-Mediated HSV-tk/Ganciclovir Suicide Gene Therapy in HeLa Cervical Carcinoma and HSC-3, FaDu, and H357 Oral Cancer CellsJennifer G. Cheung, Krystyna Konopka, nejat Düzgüneş

211 (Moved from Poster Session i)Combination of Forced Transduction of P53 and an Agent That Blocks MDM2-p53 Interactions Produces Synergistic Cytotoxicity on Mesothelioma Defective of the INK4A/ARF RegionMasatoshi Tagawa, Takao Morinaga, Zhihan li, Thao T. T. nguyen, boya Zhong, Shuji Kubo, ikuo Sekine, yuji Tada, Koichiro Tatsumi, hideaki Shimada, Kenzo hiroshima

Hematologic & Immunologic Diseases II

681HIV-1 Mediated Insertional Activation of STAT5B and BACH2 Promotes the Formation of a Viral Reservoir in T Regulatory CellsDaniela Cesana, Francesca r. Santoni de Sio, laura rudilosso, Pierangela Gallina, Andrea Calabria, laura Passerini, Silvia nozza, elisa Vicenzi, Guido Poli, Silvia Gregori, Giuseppe Tambussi, eugenio Montini

682Correction of CTLs Cytotoxic Function Defect by SIN-lentiviral Mediated Expression of Munc13-4 in Type 3 Familial Hemophagocytic LymphohistiocytosisTayebeh-Shabi Soheili, ida ricciardelli, Julie riviere, Amandine Durand, els Verhoeyen, Chantal lagresle, Geneviève de Saint basile, Persis Amrolia, isabelle André-Schmutz, Marina Cavazzana

683Foamy Virus Gene Therapy Significantly Restores Lymphocyte Development and Function in SCID-X1 MiceMatthew MacQuivey, Gabrielle Curinga, nick hubbard, iram Khan, Courtnee Clough, Daniel humphrys, Jennifer Adair, Grant Trobridge, Troy Torgerson, Andy Scharenberg, hans-Peter Kiem, David J. rawlings

684Enhanced FVIII AAV Vector Cassette Produces Improved Virus Yields and Supraphysiological FVIII Levels In Vivobrigit riley, Jeff boonsripisal, Alicia Goodwin, eudean Garces, Stephen ballaron, hung Tran, Andrea Kang, lei Zhang, Judy Greengard, richard Surosky, Kathleen Meyer, Dale Ando, Geoff nichol, Michael C. holmes

685Restoration of Antiviral Immunity Following Hematopoietic Stem Cell Transplantation with Virus-Specific T-Lymphocyte TherapyMichael D. Keller, Jamie C. hoover, Patrick J. hanley, Sarah McCormack, Cecilia barese, elizabeth Williams, neha Joshi, Maria Martin-Manso, lauren roesch, Conrad r. Cruz, Allistair Abraham, Kirsten Williams, blachy Davila, evelio Albuerne-Perez, David Jacobsohn, Catherine M. bollard

686Forced Fetal-Globin Reactivation Mediated by the TALE-ldb1 Fusion ProteinJun Zhan, irudayam Maria Johnson, Arthur nienhuis

687Development of a Safe and Effective Combinatorial Foamy Virus Vector for HIV Gene TherapyArun K. nalla, Theodore Williams, Athena lemon, rahaf Qamou, elizabeth everson, Grant D. Trobridge

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688Supplemental Immune Suppression Is Required for AAV-F8 ITI in Hemophilia A Mice with Pre-Existing InhibitorsDavid Markusic, brett Palaschak, irene Zolotukhin, roland herzog

689 Abstract Withdrawn

690Development of a Clinical Lentiviral Vector for Gene Therapy of SCID-X1Sabine Charrier, Valentina Poletti, Samia Martin, bernard Gjata, Matthias hebben, Alban Vignaud, Fang Zhang, Karen buckland, Michael rothe, Axel Schambach, bobby Gaspar, Adrian Thrasher, Fulvio Mavilio

691 Abstract Withdrawn

692Genome Editing for Personalized Gene Therapy of IVSI-110 Beta-ThalassemiaPetros Patsali, Claudio Mussolino, Constantinos loucari, Coralea Stephanou, Michael Antoniou, Toni Cathomen, Carsten W. lederer, Marina Kleanthous

693Single Cell Dynamics Causes Pareto-Like Effect in Stimulated T Cell PopulationsAlice Moussy, Jérémie Cosette, Fanny onodi, Adrien Auffret-Cariou, Thi My Anh neildez-nguyen, Andras Paldi, Daniel Stockholm

Immunological Aspects of Gene Therapy II: AAV Vectors

694Anti-Transgene Cellular Immune Repsonses Can Be Induced by Subretinal Gene Transfer with rAAV in a Dose-Dependent MannerJulie M. Vendomele, Quentin Khebizi, Mirella Mormin, Sabrina Donnou, Catherine Poinsignon, Anne Galy, Sylvain Fisson

695An AAV8 Mutant with Better Transduction in Murine Muscle and Nasal Airway Than AAV8Qiang Wang, Peter bell, hongwei yu, Christine Draper, Deirdre McMenamin, Maria P. limberis, James M. Wilson

696TLR9 Signaling Mediates Transgene Antibody FormationScott Ashley, Deirdre McMenamin, Christine Draper, James M. Wilson

697Phenotypic and Functional Characterisation of Human Anti-AAV CD8+ T Cells Using MHC Class I Tetramer-Associated Magnetic EnrichmentCeline Vandamme, Marie Devaux, nicolas Jaulin, Mickaël Guilbaud, Johanne le Duff, leslie hesnard, hanane Djamai, Marc bonneville, Philippe Moullier, xavier Saulquin, oumeya Adjali

698Effective Depletion of Pre-Existing Anti-AAV Antibodies Requires Broad Immune Targetinghaiyan Fu, Aaron S. Meadows, Victoria Velazquez, Marybeth Camboni, ricardo Pineda, F. Jason Duncan, Christopher M. Walker, Douglas M. McCarty

699Effective AAV9 Vector Delivery to Nasal Mucosa for Protection Against Airborne Challenge with Influenza A and BMarco Crosariol, Ghazal Stity, omua Ahonkhai, erik Wielechowski, Angelica C. Medina-Jaszek, Arbans Sandhu, Julie Johnston, Martin lock, Anna Tretiakova, James M. Wilson, Maria P. limberis

700Successful Repeated Hepatic Gene Delivery in Mice and Non-Human Primates Achieved by Sequential Administration of AAV5 and AAV1 Vector SerotypesAnna Majowicz, David Salas, nerea Zabaleta, estefania rodríguez-Garcia, Gloria Gonzalez Aseguinolaza, harald Petry, Valerie Ferreira

701Successful In Vivo Re-Administration of AAV with the Use of Two-Step AAV InjectionAnna Majowicz, harald Petry, Valerie Ferreira

702Detection and Isolation of Immune Cells After Intramuscular AAV Injection in Mouse and HumanGwladys Gernoux, Alisha Gruntman, Terence r. Flotte, Christian Mueller

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Vector and Cell Engineering/Manufacturing II

703Development of RD-MolPack-trLNGFR/dCK.DM.S74E Stable Packaging Cells Claudia Piovan, erica Giuliani, Stefano Corna, Cinzia Scavullo, Sergio bossi, Monika Pema, Jeffrey A. Medin, Monty McKillop, bryan Au, Claudio bordignon, Gian Paolo rizzardi, Chiara bovolenta

704Enhanced Lentiviral Production Through Rational Design of Mammalian Host CellsJean-Francois Gelinas, Deborah r. Gill, Stephen C. hyde

705Enhancement of CAR Expression of piggyBac Transposon-Engineered T Cells by Stimulation with Viral AntigensDaisuke Morita, Miyuki Tanaka, nobuhiro nishio, nozomu Kawashima, yusuke okuno, Matthew h. Wilson, Cliona M. rooney, yoshiyuki Takahashi, yozo nakazawa

706Consistent Viral Vector Manufacturing for Phase III Using iCELLis 500 Fixed-Bed TechnologyMinna Karhinen, elisa M. nurminen, Piia Valonen, outi rautsi, Vesa huusko, Kati heikkilä, Janne Weisell, Achim Muller, hanna P. lesch, outi närvänen, Minna M. hassinen, Minna Pollen, Steve Parrish, robert Shaw, nigel r. Parker, Seppo ylä-herttuala

707Attenuated HIV Does Not Amplify in Primary Human T-Lymphocytes During a Model Ex Vivo Gene Therapy ProcedurePratima Cherukuri, Scott Witting, lilith reeves

708Process Development of Lentiviral Vectors for Large Scale Production Using a HEK293 Producer Cell LineAlexandre Audy, Marc-André robert, Parminder Chahal, rénald Gilbert, bruno Gaillet

709Characterization of Nanoparticles in Lentiviral Vector Preparationserik hansen, Kristen lee, Suzanna Melotti, Jesse Milling, Maya Miatkowski, James Warren, Mercedes Segura

710Surface Adsorptive Loss of rAAV on Materials Used in cGMP ManufacturingMonica Jiang, Colleen M. Dupuis, Amy A. Medeiros, Samuel C. Wadsworth, K. reed Clark, Christopher J. Morrison

711Development of a Clinically-Acceptable Lentiviral Vector for Cystic Fibrosis Airway Gene TherapyAlexandra McCarron, Chantelle Mcintyre, Martin Donnelley, Patricia Cmielewski, David Parsons

712BIUX2X2neha Tiwari, Jill beilowitz, Carlos Sampson, Dorothy Peterson, Aaron Carnes, Jim Williams

713Optimization and Scale-Up of a Manufacturing Process for Clinical-Grade Adenovirus-Based VectorsMaria Fe C. Medina, Shabnam Shoaebargh, natasha Kazhdan, uma Sankar, David r. latulippe, brian lichty

714Use of a Modular Workstation for Reduced Biosafety Risks Also Increased Cellular YieldsAlicia D. henn, Shannon Darou, randy yerden

Clinical Protocol Development, Regulatory Interactions, and Ethics

715Giant Axonal Neuropathy - The Role of Natural History Studies in Gene Transfer Therapy Trial DesignDiana bharucha-Goebel, Minal Jain, Melissa Waite, Tanya lehky, reghan Foley, Jonathan D. Marra, Wadih Zein, Carsten G. bonnemann

716 Abstract Withdrawn

717Revising the Common Rule: Changes to the Federal Policy Protecting Human Subjectsbambi Grilley

718Cell Therapy Commercialization: An Assessment of Translational BarriersDavid Pettitt, Georg hollander, David brindley

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719Stability Could Be a Weak Point for Gene Therapy SponsorsDominique Kissick

720Data-Driven Development to Deployment of Recombinagenic to Reprogramming DrivesFaisal reza

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Saturday, May 7, 2016Oral Abstract Session 410AAV Vectors and Pre-Clinical Analysis10:15 AM - 12:15 PMroom: Virginia Suite

721 10:15 AMLong-Term (2-8 years) Body-Wide Expression of AAV9-Minidystrophin Gene in Golden Retriever Muscular Dystrophy Dogs After Regional Limb Vein InjectionJuan li, Joe Kornegay, Chunping Qiao, hui Zheng, Jianbin li, ruhang Tang, yi Dai, xiao xiao

722 10:30 AMAAV Capsid Evolution for Enhanced Antibody Delivery to Human Skeletal Muscle for Use in Next-Generation HIV Vaccines and Muscle Gene Therapiesnicole K. Paulk, Gregory Charville, Katie Maguire, Katja Pekrun, yue Zhang, Matthew Tiffany, Jose Vilches-Moure, Gordon lee, Joseph Shrager, Thomas rando, Mark A. Kay

723 10:45 AMAAV Mediated Cancer Targeting: Systemic Trafficking to Tumor Is More Important Than Vector Tumor Cell Interactionyuan lu, Damien Marsic, Chen ling, Maria V. Guijario, Padraic P. levings, Glyn D. Palmer, Anthony Dacanay, rachael S. Watson-levings, Viktoria hyddmark, Margaret e. White, Sergei Zolotukhin, Steven C. Ghivizzani

724 11:00 AMA Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-Associated Virus Serotypes upon Cerebrospinal Fluid Administration in Pigsnicolina C. Sorrentino, Veronica Maffia, Sandra Strollo, Vincenzo Cacace, noemi romagnoli, Anna Manfredi, Domenico Ventrella, Francesco Dondi, Francesca barone, Massimo Giunti, Anne-renee Graham, yan huang, Susan l. Kalled, Alberto Auricchio, Maria l. bacci, enrico M. Surace, Alessandro Fraldi

725 11:15 AMAssessment of CSF Route for Gene Delivery in Sandhoff Mice Using AAV9 Expressing an Hexosaminidase Isoenzyme.Patrick Thompson, Karlaina osmon, evan Woodley, Subha Karumuthil-Melethil, Steven J. Gray, Jagdeep S. Walia

726 11:30 AMPrevention of Muscular Dystrophy by an AAV Vector Encoding a Nonimmunogenic Protein Based on the Evolution of Utrophin and Dystrophinyafeng Song, Alock Malik, Andy F. Mead, Marilyn Mitchell, Mihail Petrov, leonard Su, leon Morales, Shira rosenblum, Tejvir Khurana, Joe n. Kornegay, hansell h. Stedman

727 11:45 AMCharacterizing the Adeno-Associated Virus 1 Sialic Receptor Binding Site and Its Overlap with Antigenic Epitopeslin-ya huang, Ami Patel, robert ng, edward b. Miller, Sujata halder, robert McKenna, Aravind Asokan, Mavis Agbandje-McKenna

728 12:00 PMTissue-Directed Transgene Engineering for AAV and Lentivector Gene Therapy Approachesharrison C. brown, Phillip M. Zakas, Stephan n. George, ernest T. Parker, h. Trent Spencer, Christopher b. Doering

Oral Abstract Session 411Targeted Genome Editing: Methods and Technology10:15 AM - 12:15 PMroom: Washington 1-2

729 10:15 AMInheritable Silencing of Endogenous Gene by Hit-and-Run Targeted Epigenetic EditingAngelo Amabile, Alessandro Migliara, Paola Capasso, Mauro biffi, luigi naldini, Angelo lombardo

730 10:30 AMPermanent Correction of Diverse Muscular Dystrophy Mutations in Human Cardiomyocytes by MyoeditingChengzu long, hui li, Viktoriia Kyrychenko, huayu Zhou, Samadrita bhattacharyya, Cristina e. rodriguez, John M. Shelton, Pradeep Mammen, Jay Schneider, rhonda bassel-Duby, eric n. olson

731 10:45 AMHigh-Fidelity CRISPR-Cas9 Nucleases with No Detectable Genome-Wide Off-Target Effectsbenjamin P. Kleinstiver, Vikram Pattanayak, Michelle S. Prew, Shengdar Q. Tsai, nhu T. nguyen, J. Keith Joung

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732 11:00 AMCRISPR/Cas9 Mediates Highly Efficient Gene Editing in Long-Term Engrafting Human Hematopoietic Stem/Progenitor CellsJack M. heath, Aditi Chalishazar, Christina S. lee, William Selleck, Cecilia Cotta-ramusino, David bumcrot, Jennifer l. Gori

733 (Moved to oral Session 330)Somatically Repairing Compound Heterozygous Recessive Mutations by Chromosomal Cut-and-Paste for In Vivo Gene TherapyDan Wang, haiwei Mou, Karen Tran, Jia li, blake y. Wang, Dominic J. Gessler, Phillip Wl Tai, Qin Su, Wen xue, Guangping Gao

120 11:15 AM (Moved from Poster Session i)Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9Kshitiz Singh, hanneke evens, Melvin rincón, nisha nair, Shilpita Sarcar, ermira Samara-Kuko, Marinee K. Chuah, Thierry VandenDriessche

734 11:30 AMPre-Clinical Development and Qualification of ZFN-Mediated Disruption of CCR5 in Human Hematopoietic Stem and Progenitor Cellsrodica Stan, David DiGiusto, Paula Cannon, Colin exline, lijing li, Anitha rao, Jianbin Wang, Gary lee, Philip D. Gregory, nancy Gonzalez, Victoria bedell, Michael C. holmes, John A. Zaia

735 11:45 AMFunctional Restoration of Dystrophin Protein in HiPSC-Derived Skeletal Myotubes and Cardiomyocytes After CRISPR/Cas9-Mediated Deletion of 530-725kb of DMDCourtney S. young, Michael r. hicks, natalia V. ermolova, haruko nakano, Majib Jan, Shahab younesi, Atsushi nakano, Stanley F. nelson, M. Carrie Miceli, Melissa J. Spencer, April D. Pyle

736 12:00 PMSilencing Substituting the RHODODPSIN Gene to Treat Blinding DisordersSalvatore botta, elena Marrocco, nicola de Prisco, Fabiola Curion, Mario renda, Martina Sofia, Mariangela lupo, Maria laura bacci, Carlo Gesualdo, Settimio rossi, Francesca Simonelli, enrico Maria Surace

Oral Abstract Session 412Oligonucleotide Therapeutics II10:15 AM - 12:15 PMroom: Thurgood Marshall W

737 10:15 AMRNAi Induced Hepatotoxicity Results from a Functional Depletion of the First Synthesized Isoform of miR-122Paul n. Valdmanis, Shuo Gu, Kirk Chu, lan Jin, Feijie Zhang, elizabeth M. Munding, yue Zhang, yong huang, huban Kutay, Kalpana Ghoshal, leszek lisowski, Mark A. Kay

738 10:30 AMEngineered MicroRNA Silences C9ORF72 Variants in BAC Transgenic MouseGabriela Toro Cabrera, owen M. Peters, Tania F. Gendron, Alisha Gruntman, robert h. brown, Christian Mueller

739 10:45 AMrAAV Delivered MicroRNA Therapeutics Towards Efficacious Treatment of Corneal Neovascularizationyi lu, Jianzhong Ai, Phillip Tai, Dominic J. Gessler, Qin Su, Qiang Zheng, Phillip D. Zamore, xun xu, Guangping Gao

740 11:00 AMThe TLR9 Agonist EnanDIM(R) - Evaluation of New Enantiomeric Oligonucleotides for Cancer Immunotherapy In Vitro and In VivoKerstin Kapp, Matthias Schroff, burghardt Wittig, Manuel Schmidt

741 11:15 AMDevelopment of LNA Gapmer Oligonucleotide Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansionyoshitsugu Aoki, raquel Manzano, Shin’ichi Takeda, Kevin Talbot, Matthew Wood

742 11:30 AMA Therapeutic miRNA for Brain DisordersKai C. Sonntag, Woori Kim, yenarae lee, Donna l. McPhie, Kwang-Soo Kim, Gordana Vitaliano, bruce M. Cohen

743 11:45 AMMinicircles Are Similar to Plasmids in Providing High Level, Long-Term Expression in the LungDeborah r. Gill, reto P. bazzani, Mary M. Connolly, Martin Schleef, Stephen C. hyde, ian A. Pringle

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744 12:00 PMOptimizing Conditions for Aptamer Folding Using a High-Throughput Aptamer Fluorescence Binding and Internalization (AFBI) AssayDavid D. Dickey, Paloma h. Giangrande, William h. Thiel

Oral Abstract Session 413Cancer-Immunotherapy, Cancer Vaccines III10:15 AM - 12:15 PMroom: Thurgood Marshall n

745 10:15 AMUpdated Phase 1 Results from ZUMA-1: A Phase 1-2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-C19 (Anti-CD19 CAR T Cells) in Subjects with Refractory Aggressive Non-Hodgkin Lymphoma (NHL)Frederick l. locke, Sattva S. neelapu, nancy l. bartlett, Tanya Siddiqi, Julio C. Chavez, Chitra M. hosing, Armin Ghobadi, lihua e. budde, Adrian bot, John M. rossi, Marika Sherman, lynn navale, Meg elias, Jeff Wiezorek, William y. Go

746 10:30 AMGo-TCR: Inducible MyD88/CD40 (iMC) Enhances Proliferation and Survival of Tumor-Specific TCR-Modified T Cells, Increasing Anti-Tumor EfficacyTsvetelina P. hoang, Aaron Foster, Tania rodriguez, David Torres, An lu, Jeannette Crisostomo, lorenz Jahn, Mirjam h. M. heemskerk, David M. Spencer

747 10:45 AMTowards the Clinical Application of BCMA CAR T Cells: The Importance of Reduced Tonic Signaling and Methods to Enhance Memory T CellsKevin Friedman, holly horton, Molly Perkins, Shannon Grande, Tracy Garrett, richard Morgan

748 11:00 AMA Clinical Trial Using Third Generation CD19 Targeting CAR T Cells for Relapsed Lymphoma and LeukemiaGunilla enblad, hannah Karlsson, Jessica Wenthe, Kristina i. Wikstrom, Magnus essand, barbara Savoldo, Gianpietro Dotti, Martin hoglund, helene hallbook, Malcolm K. brenner, hans hagberg, Angelica loskog

749 11:15 AMEscape from Tumor Dormancy Following Gene- or Viro-Therapies Is Mediated by Acquisition of a Phenotype in Which Innate Immune Surveillance Actively Drives Tumor Cell RecurrenceTim Kottke, Diana rommelfanger, Kevin G. Shim, Jose Pulido, Jill Thompson, Karishma rajani, oleg bobr, Peter Selby, elizabeth ilett, Alan Melcher, richard Vile

750 11:30 AMAnti-Tumor Effects of Anti-CD20 Chimeric Antigen Receptor Therapy in Canine B Cell Lymphoma PatientsM. Kazim Panjwani, Josephine S. Gnanandarajah, Martha A. Maloneyhuss, nicola J. Mason

751 11:45 AMImproving CAR T Cell Function by Reversing the Immunosuppressive Tumor Environment of Pancreatic CancerSujita Sukumaran, Somala Mohammed, norihiro Watanabe, Pradip bajgain, Salma Ansari, usanarat Anurathapan, helen e. heslop, Cliona r. rooney, Malcolm K. brenner, Ann M. leen, Juan F. Vera

752 12:00 PMSingle Chain TCR Gene Editing in Adoptive Cell Therapy for Multiple MyelomaSara Mastaglio, Pietro Genovese, Zulma Magnani, elisa landoni, barbara Camisa, Giulia Schiroli, elena Provasi, Angelo lombardo, Andreas reik, nicoletta Cieri, Martina rocchi, Giacomoq oliveira, Giulia escobar, Monica Casucci, bernhard Gentner, Antonello Spinelli, Anna Mondino, Attilio bondanza, luca Vago, Maurilio Ponzoni, Fabio Ciceri, Micheal holmes, luigi naldini, Chiara bonini

Oral Abstract Session 414Hematologic & Immunologic Diseases II10:15 AM - 12:15 PMroom: Thurgood Marshall S

753 10:15 AMLentiviral-Mediated Gene Correction of Mobilized Peripheral Blood Progenitors and Repopulating Cells from FA-A PatientsPaula río, Susana navarro, Guillermo Guenechea, Anne Galy, rebeca Sánchez, María l. lamana, rosa yañez, José A. Casado, José C. Segovia, Jordi Surrallés, ricardo lópez, nagore García de Andoaín, Pedro ruiz, Albert Catalá, Cristina Díaz de heredia, Julián Sevilla, Juan A. bueren

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754 10:30 AMExploring the Human Hematopoietic Hierarchy Through Retroviral Integration Sites Tracking in the Wiskott Aldrich Syndrome Gene Therapy Trialemmanuelle Six, laure Caccavelli, Cecile roudaut, Frances Male, yinghua Wu, nicolas Cagnard, Alessandra Magnani, Fabien Touzot, elisa Magrin, Anne Galy, isabelle André-Schmutz, Salima hacein-bey Abina, Jean-Philippe Jais, Charles C. berry, Frederic D. bushman, Marina Cavazzana

755 10:45 AMOne-time Gene Therapy for Hereditary Angioedemaodelya e. Pagovich, Ting Qiu, Adele S. Whaley, Anthony r. russo, Jonathan rosenberg, bishnu P. De, Carlo russo, Maria J. Chiuchiolo, Dolan Sondhi, Stephen M. Kaminsky, ronald G. Crystal

756 11:00 AMStable Amelioration of Hemophilia B in Dogs by Intravenous Administration of Lentiviral Vectors Expressing Hyper-Functional Factor IXAlessio Cantore, Michela Milani, Tongyao liu, Patrizia Della Valle, Armando D’Angelo, Thierry VandenDriessche, Marinee Chuah, haiyan Jiang, Timothy nichols, luigi naldini

757 11:15 AMStable In Vivo Transduction of Primitive Hematopoietic Stem Cells After Mobilization and Intravenous Injection of an Integrating Gene Transfer VectorMaximilian richter, roma yumul, Donna Palmer, Philip ng, Zsuzsanna izsvák, roberto Cattaneo, Jing liu, Anja ehrhardt, Wolfgang uckert, Thalia Papayannopoulou, André lieber

758 11:30 AMA Nonhuman Primate Transplantation Model to Evaluate Gene Editing Strategies Aimed at Inducing Fetal Hemoglobin Production for the Treatment of Hemoglobinopathiesolivier humbert, hans-Peter Kiem

759 11:45 AMReduction of HLH-like Manifestations in Murine Model of Munc13-4 Deficiency Following Lentiviral Gene Transfer into Hematopoietic Stem and Progenitor CellsTayebeh-Shabi Soheili, Fernando Sepulveda, Amandine Durand, Julie rivière, Samia Martin, Geneviève de Saint basile, Marina Cavazzana, isabelle André-Schmutz

760 12:00 PMOptimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus MacaquesJenny A. Greig, Qiang Wang, Amanda l. reicherter, erin bote, Deirdre McMenamin, Christine Draper, Shu-Jen Chen, Alexandra l. hanlon, Tamara Goode, K. reed Clark, Samuel Wadsworth, lili Wang, James M. Wilson

Oral Abstract Session 415Immunological Aspects of Gene Therapy II10:15 AM - 12:15 PMroom: Thurgood Marshall e

761 10:15 AMTargeted Killing of HIV Infected Cells Using CCR5-Disrupted Anti-HIV-CAR T CellsGuillermo S. romano ibarra, Malika hale, Karen Sommer, iram Khan, Jaya Sahni, Christopher Peterson, hans-Peter Kiem, Jordan Jarjour, Alexander Astrakhan, Andrew M. Scharenberg, David J. rawlings, Thor A. Wagner

762 10:30 AMA Quantitative Imaging Tool Box for the Functional Analysis of Chimeric Antigen Receptor - T Cell Immune Synapse Helps Identify Novel Structural and Functional Features of CAR T CellsMalini Mukherjee, Maksim Mamonkin, nabil Ahmed, Malcolm brenner, Jordan orange

763 10:45 AMHIV-Specific T Cells Can Be Expanded from Virus-Naive Donors to Target a Range of Viral Epitopes: Implications for a Cure Strategy After Allogeneic HSCTShabnum Patel, Sharon lam, Conrad russell Cruz, Kaylor Wright, Christina Cochran, richard Ambinder, elizabeth Shpall, Catherine bollard

764 11:00 AMDosing and Re-Administration of Intravenous Lentiviral Vector for Liver-Directed Gene Transfer in Young Rhesus Monkeys and ADA-Deficient MiceDenise A. Carbonaro-Sarracino, Alice F. Tarantal, Chang i. lee, Michael l. Kaufman, Michele Martinez, Danielle n. Clark, Colin Koziol, Donald b. Kohn

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AbSTrACT DireCTory AnD AuThor inDex — 61

765 11:15 AMDetection of Treatment-Resistant Infectious HIV After Genome-Directed Antiviral Endonuclease TherapyDaniel Stone, harshana S. De Silva Feelixge, harlan l. Pietz, Pavitra roychoudhury, Alex l. Greninger, Joshua T. Schiffer, Martine Aubert, Keith r. Jerome

766 11:30 AMGenetic Correction of Ifnγr1 Deficiency in Hematopoietic Cells Repairs the Cellular Phenotype of Mendelian Susceptibility to Mycobacterial Diseasenico lachmann, Adele Mucci, Miriam hetzel, robert Meineke, olga K. halle, Mark P. Kühnel, ralph Goethe, Sebastian brennig, Daniel brand, reinhold Förster, Axel Schambach, Thomas Moritz

767 11:45 AMA Novel Humanized Mouse Model for Testing Anti-HIV T-Cell Gene Therapy Strategies Kevin G. haworth, Christina ironside, hans-Peter Kiem

768 12:00 PMThe CRISPR/Cas9 System as an Anti-Viral Treatment to Prevent Primary Infection by HCMV Positive Hematopoietic Stem CellsJanina Gergen, Flora Coulon, Alison Creneguy, Jean-Marie heslan, ignacio Anegon, Tuan huy nguyen, Franck halary, Fabienne haspot

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 62

Author IndexAagaard, lars ............................................................ 589

Aartsma-rus, Annemieke .............................................. 504

Abdulkerim, hayat ...................................................... 576

Abdul Majid, Asma .................................................... 208

Abdul razak, hayder .................................................. 634

Abe, Shinya .............................................................. 664

Abel, Tobias ................................................................ 59

Aboody, Karen .......................................................... 208

Aboody, Karen S. ............................................... 209, 443

Abraham, Allistair ....................................................... 685

Abraham, Sojan ......................................................... 574

Abrams, Gary ............................................................. 98

Abu Abed, omar S. ................................................... 600

Abu Zaid, Mohammad ................................................ 508

Aceves-Chimal, José l. ................................................. 246

Achilly, nathan P. ......................................................... 46

Ackermann, Mania ............................................. 234, 522

Ackley, Amanda ......................................................... 532

Adachi, Kei ........................................... 12, 96, 255, 540

Adachi, Kumi............................................................. 308

Adair, Jennifer .................................................... 284, 683

Adair, Jennifer e. ........................................................ 283

Adamo, richard F. ...................................................... 174

Adams, Alexa .............................................................. 26

A. Davies, lee ........................................................... 431

Adjali, oumeya ................................................. 503, 697

Adkar, Shaunak S. ...................................................... 318

Aebischer, Patrick ....................................................... 606

Aeran, rangoli ........................................................... 607

AFM-sponsored Duchenne Consortium ............................ 503

Afzal, Saira ....................................................... 232, 470

Agarwal, Payal .................................................. 517, 677

Agarwal, S. .............................................................. 401

Agarwal, Suneet ........................................................ 568

Agbandje-McKenna, Mavis .................. 256, 294, 541, 727

Aghi, Manish K. ........................................................... 61

Agrawal, Sudhir ......................................................... 153

Aguado, Jesus ...........................................................186

Agudelo, Daniel .........................................................139

Aguilar, brenda ..........................................198, 204, 275

Aguilar, T. .................................................................445

Aguirre, Gustavo D. ....................................................617

Ahamed, rubina ........................................................632

Ahmad, Shahzad .......................................................232

Ahmadi, neeki ..........................................................354

Ahmed, nabil ...................................................392, 762

Ahn, hyo Min............................................................112

Ahokhai, omua ...........................................................60

Ahonkhai, omua ................................................189, 699

Ai, Jianzhong .............................................290, 669, 739

Aishwarya, Veenu ......................................................586

Aitamer, Marine .........................................................578

Aiuti, Alessandro ........................................226, 280, 531

Akahori, yasushi .........................................................193

Akbar, Mohammad .......................................................84

Akbar, Mohammad A. ................................................632

Akbuğa, Jülide ...........................................................152

Akkina, ramesh .........................................................532

Alabanza, leah M. ......................................................74

Al-Abbasee, raed ......................................................290

Alan, Saadet .............................................................152

Albano, luisa.........................................................37, 42

Alberquilla, omaira ....................................................228

Albert, Jessica ..............................................................68

Alberti, Micol ..............................................................53

Albuerne-Perez, evelio .................................................685

Aldossary, Ahmad ........................................................23

Aldrin-Kirk, Patrick ...............................................256, 542

Aleman, Tomas ..........................................................270

Alemany, ramon ................................................516, 662

Alexander, ian ...........................................................169

Alexander, ian e. ................................................298, 541

Alfano, lindsay ..................................................480, 497

Alferiev, ivan S. ..........................................................174

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 63

Author IndexAlghamri, Mahmoud S. ...............................................176

Ali, robin ..................................................................256

Alizadeh, Darya ........................................................247

Alkuraya, Fowzan S. ...................................................360

Allegri, Gabirella .......................................................355

Allen, Cornell ..............................................................41

Allen, James ..............................................................501

Allen, rachel ...............................................................71

Aloia, Andrea ............................................................653

Alonso-Camino, Vanesa .................................................63

Al-Saeedi, Amer T. Tawfeeq. ........................................679

Al-Shammari, Ahmed M. ..............................................416

Alt, Frederick .............................................................568

Alton, eric W. F. W. ...................................................534

Altrock, Philipp M. ......................................................314

Alvanou, Maria ..........................................................426

Álvarez, lara .............................................................558

Alves, Anna ...............................................................301

Alvey, Cory ...............................................................511

Al-Zaidy, Samiah ................................................480, 497

Amabile, Angelo ........................................................729

Amaishi, yasunori .......................................................193

Amankulor, nduka ......................................................520

Amarasekara, hiruni ...................................................311

Amartino, hernan .......................................................250

Ambinder, richard ......................................................763

Ambler, Gareth ..........................................................452

Amendola, Mario .......................................................135

Amor, Fatima .............................................................135

Amreddy, narsireddy ..................................................489

Amrolia, Persis ...........................................................682

Anagnostopoulos, Achilles ...................................291, 426

Anagnou, nicholas P. .................................................222

Andersen, Sofie .........................................................533

Ando, Dale ...............................................................684

Andolfi, Grazia ..........................................................637

André-Schmutz, isabelle .......225, 425, 560, 682, 754, 759

Anduaga, Javier .........................................................165

Anegon, ignacio ........................................................768

Angenent-Mari, nicolas...............................................335

Anguela, xavier M. ......................................................52

Annoni, Andrea ...................................................51, 286

Annunziata, Patrizia ......................................................47

Ansari, Salma ....................................................190, 751

Antoniani, Chiara ...............................................135, 559

Antoniou, Michael ..............................................230, 692

Antoniou, Panayotis ....................................................222

Antony, Jeyaraj ...................................................200, 394

Antrilli, Thomas ..........................................................185

Anurathapan, usanarat ...............................................751

Aoki, yoshitsugu .........................................................741

A. Pringle, ian............................................................431

Araki, Ayame ............................................................395

Aramburo, Soraya ......................................................209

Aref, Sarah .........................................................65, 663

Arenas, Victor M. .......................................................246

Arenas-luna, V. ..........................................................453

Argaw, Takele ...........................................................214

Arguello, Annie ..........................................................186

Arif, Muhammad ..................................................60, 189

Armentano, Donna ...............................................92, 387

Armstrong, emily ................................................612, 619

Armstrong, robin ........................................................579

Arnaud, Cécile ..........................................................231

Arndt, Tara ..................................................................88

Arndt, Tara Arndt ........................................................299

Arnold, Madeline .........................................................46

Arnold, W Dave ........................................................480

Arnson, benjamin .........................................................49

Aronovich, elena l. .....................................................439

Aronson, Joshua P. ......................................................598

Arroyo, Jonathan ........................................................206

Arsenault, Jason .........................................................368

Arumugam, Paritha .......................................................21

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 64

Author IndexArumugam, Paritha i. ....................................................35

Asada, Koutaro ..........................................................144

Asavarut, Paladd ........................................................672

Ashley, Scott ..............................................................696

Ashtari, Manzar .........................................................265

Aslanidi, George .............................10, 11, 91, 196, 548

Aslanidi, Georgy ..................................................95, 215

Asokan, Aravind ........................8, 30, 55, 482, 504, 727

Astrakhan, Alexander ....................44, 277, 323, 398, 761

Ataeijannati, yasaman .................................................266

Atasheva, Svetlana .......................................................13

Athanasopoulos, Takis .................................503, 628, 634

Attinà, Maria luisa .....................................................691

Au, bryan .................................................................703

Aubert, Martine ..........................................................765

Aubourg, Patrick .................................................250, 365

Audrain, Mickael .........................................................59

Audy, Alexandre ................................................341, 708

Auffret-Cariou, Adrien ..................................................693

Auricchio, Alberto .................................28, 187, 468, 724

Ayala-breton, Camilo ..................................................654

Aysola, Manjula .........................................................466

Ayuso, eduard ...............................................34, 50, 302

Aziz-bose, razina ......................................................250

babu, Anish ..............................................................489

baccarini, Alessia b. .....................................................70

bacchetta, rosa .........................................................123

bacci, Maria l. ..........................................................724

bacci, Maria laura .............................................320, 736

bachtarzi, houria .......................................................500

badie, behnam ..................................198, 204, 247, 275

bae, Seongho ...........................................................350

baekelandt, Veerle ......................................................175

bagarazzi, Mark ........................................................505

bagel, Jessica h. ..................................................56, 611

bago, Juli ..........................................................442, 528

bailey, Katharine ..................................................65, 663

bailey, rachel M. .......................................................604

bailus, barbara ............................................................26

bain, owen ..............................................................452

bair, Thomas .............................................................148

bajgain, Pradip ..........................................190, 451, 751

bak, rasmus o. .....................................39, 43, 127, 533

baker, Chris ..............................................................265

ballantine, Josiah ............................................................3

ballaron, Stephen .......................................................684

balyasnikova, irina .......................................................76

banala, Vibhu ............................................................294

banerjee, Abhik .........................................................252

banerjee, ipsita ..........................................................239

bank, Alexander ........................................................397

bankiewicz, Krystof S. .................................................621

bankiewicz, Krystoff ....................................................618

banks, Glen b. ..........................................................380

bankstahl, Jens P. ........................................................522

bao, Gang ...............................................................131

barbeau, xavier .........................................................561

barese, Cecilia ....................................................75, 685

barish, Michael ..........................................................275

barish, Michael e. ......................................198, 247, 443

barker, roger .............................................................367

barone, Francesca ......................................................724

barone, Samuel b. .....................................................248

barravecchia, Michael ................................................490

barrera, luis ......................................................124, 576

barrett, John A. ..........................................................509

barry, elizabeth ..................................................301, 329

bartlett, nancy l. .......................................................745

bartolaccini, Sara .......................................................286

bartolucci, Pablo ........................................................279

bartsevich, Victor ................................................138, 200

bartsevich, Victor V. ............................................394, 579

baruchel, André .........................................................250

barzel, Adi ..................................................................48

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 65

Author Indexbasche, Mark ............................................................256

baskar, Sivasubramanian .............................................649

bass, Caroline e. .......................................................376

bassel-Duby, rhonda ...................................................730

basso ricci, luca ........................................................531

bataille, Carole ............................................................19

batshaw, Mark l. .......................................................481

batsis, ioannis ............................................................426

baturevych, Alex ................................................170, 572

bauer, Daniel e. .........................................................282

bauer, Thomas r. .......................................................285

baumeister, Suzanne .....................................................71

baylin, Stephen ..........................................................237

bazán-A, C. ..............................................................453

bazzani, reto P. .........................................................743

beard, Clayton W. .............................................138, 394

beauséjour, Christian ...................................................321

bechinger, burkhard ....................................................536

bedell, Victoria ..........................................................734

bedoya, Felipe ..........................................................203

beedle, Aaron ...........................................................377

beekman, Jeffery M. ...................................................534

beekman, Jeffrey ........................................................175

beggs, Alan ..............................................................498

behr, Michael ....................................................194, 410

beilowitz, Jill ..............................................................712

bell, eric ...................................................................170

bell, Jason b. .............................................................439

bell, Peter .................................................346, 481, 695

bellayr, ian h. ............................................................245

belmonte, Paul ...........................................................543

beloglazova, irina ......................................................447

beltraminelli, Tim ........................................................327

beltran, William A. .....................................................617

belur, lalitha ........................................................25, 369

bemelmans, Alexis-Pierre ................................................59

benati, Daniela ..........................................................488

benedicenti, Fabrizio ..............................2, 212, 537, 674

benedict, William F. ......................................................72

bengtsson, niclas e. ...................................................501

bennett, Antonette .......................................................256

bennett, Jean .....................185, 189, 265, 270, 553, 556

bennicelli, Jeannette l. .........................................553, 556

benoist, Christophe .....................................................560

benosman, ryad ........................................................268

benson, Janet.............................................................472

berchel, Mathieu ........................................................597

beretta, Stefano .........................................................537

berezovski, Maxim V. ..................................................151

bergmann, Thorsten ..........................................14, 15, 18

berinstein, elliot M. .....................................................407

berkhout, ben ....................................................331, 610

berlin, Jacob ..............................................................209

berman, robert ............................................................26

bernard-Marissal, nathalie ...........................................606

bernaudin, Françoise ..................................................231

bernier, Alice .............................................................292

bernstein, howard ......................................................573

berry, Charles C. .......................................................754

berry, Garrett ...............................................................30

berry, Katherine .................................................480, 497

berthelette, Patricia ...............................................92, 387

bertil, emilie ..............................................................390

bertotti, Andrea ..........................................................674

bessard, Marie-Andrée ................................................560

bestas, burcu .............................................................581

better, Marc ......................................................287, 650

beuzard, yves ............................................................279

bevilacqua, Maria Assunta ...........................................659

bhagat, lakshmi .........................................................153

bharathan, Mini .........................................................391

bharucha-Goebel, Diana .............................................715

bhattacharyya, Samadrita ............................................730

bhattacharyya, Sharmistha ...........................................146

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 66

Author Indexbhoj, Vijay ................................................................399

bi, xiaohong .............................................................240

bi, yanyu ..................................................................202

biasco, luca ..............................................................531

biavasco, riccardo .....................................................674

bidot, Samuel ............................................................267

bielamowicz, Kevin ....................................................392

bielefeld, eric C. ........................................................612

biffi, Alessandra .........................................................537

biffi, Mauro ...............................................................729

bigger, brian .....................................................256, 364

bijvelds, Marcel .........................................................175

bilbao, ignacio ..........................................................300

binder-Scholl, Gwendolyn K. ........................................192

binley, Katie ................................................................33

birchall, Martin A. ......................................................452

bird, Andrew .............................................................311

biswas, Arijit .............................................................296

biswas, Monaro ...........................................................78

biswas, roopa ...........................................................146

bjerkvig, rolf .............................................................475

björklund, Tomas ................................................256, 542

black, Diane .........................................................21, 35

blanche, Stéphane .....................................................279

blattner, Carolin .........................................................405

blessa, Thais .............................................................499

blessing, Daniel r. ......................................................463

blevins, Teri ...............................................................170

blits, bas ...................................................................618

blondeau, Johanna .....................................................689

blouin, Véronique .........................................................34

blum, Shany ..............................................................598

blum, William ............................................................276

blumenkranz, Mark S. .................................................248

bobela-Aebischer, Julianne ...........................................606

bobr, oleg ................................................................749

bockstael, olivier .......................................................610

boehler, Jessica F. .......................................................584

boehme, Philip ...............................................14, 17, 105

boelens, Jaap Jan .......................................................404

boestaneau, Alina ......................................................510

bofill-De ros, xavier ....................................................154

boisgerault, Florence .............................................77, 595

bolduc, Véronique ......................................................150

boldyreva, Maria A. ...................................................448

boldyreva, Marya ......................................................447

bolfer, luiz ................................................................180

bollard, Catherine ......................................................763

bollard, Catherine M. ...........................................75, 685

bolli, roberto .............................................................178

bolukbasi, Mehmet F. ..................................................326

bolyard, Chelsea ...............................................520, 656

bondanza, Attilio .......................................512, 637, 752

bongarzone, ernesto r. .................................................57

bongers, Cassandra A. ...............................................172

bonifacino, Aylin ........................................................523

bonini, Chiara ...................................................512, 752

bönnemann, Carsten G. ......................................150, 715

bonner, Melissa .........................................221, 229, 458

bonner, Melissa A. .....................................................281

bonneville, Marc ........................................................697

boonsripisal, Jeff .........................................................684

booth, Claire .............................................................325

bordeleau, louis-Jean ..................................................603

bordignon, Claudio ....................................130, 512, 703

borel, Florie .........................................................90, 167

bories, Dominique ......................................................231

borquez-ojeda, oriana ...............................................459

bortolussi, Giulia ........................................................158

borzone, roberta .........................................................47

bosch, Fatima ......................................................50, 348

bosio, Andreas ..........................................................653

bosnjak, Masa...........................................................495

bossi, Sergio .....................................................130, 703

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AbSTrACT DireCTory AnD AuThor inDex — 67

Author Indexbosticardo, Marita ......................................................280

bot, Adrian .......................................................287, 745

bote, erin ..................................................................760

bothmer, Anne ...........................................................576

botta, Salvatore .................................................320, 736

bouch, Karen .............................................................184

boucher, elisabeth ......................................................473

bouillé, Pascale ..........................................................538

bouquet, Céline ...........................................83, 267, 268

bourg, nathalie .........................................................377

bourget, Philippe ........................................................279

boutry, Sébastien ........................................................610

bouzelha, Mohammed ..................................................34

bovolenta, Chiara ........................................37, 130, 703

boyd, Christopher A. ..................................................534

boyd, Surina ...............................................................82

boye, Sanford l. ........................................................620

boye, Shannon e. .......................................................620

boyer, Jean................................................................505

boyko, Alisa ..............................................................432

bradbury, Allison M. .....................................................56

bradley, Stephen ........................................................258

brady, roscoe ...........................................................521

bragonzi, Alessandra ..................................................226

bram, richard ............................................................197

bramante, Simona ..............................................194, 652

brand, Daniel ............................................................766

brantly, Mark .............................................................632

brasca, Stefano .........................................................537

braun, Armin .............................................................476

braun, lyndsey ...........................................................480

breakefield, xandra o. ...............................................567

breger, ludivine ..........................................................610

brendel, Christian ...............................................282, 314

brendler, Charles b. ....................................................417

brenneman, Mark ...............................................259, 262

brenner, Malcolm ...............................................513, 762

brenner, Malcolm K. .....69, 190, 210, 392, 451, 748, 751

brennig, Sebastian .............................................522, 766

brentjens, renier J. ......................................................393

bridle, byram W. .................................................66, 422

brimble, Mark A. ................................................547, 571

brindley, David ..........................................................718

bringas, John .............................................................618

brito, Alfonso .............................................198, 204, 275

brod, Florian ...............................................................59

broderick, Kate ..................................428, 435, 440, 505

broderick, Kate e. ......................................................433

brombin, Chiara ........................................................226

brooks, brian P. ............................................................46

brooks, elizabeth D.....................................................311

broucque, Frederic .......................................................34

brown, Alexander.......................................................136

brown, Audrey ...........................................................200

brown, Audrey e. .......................................................394

brown, brian ...............................................................70

brown, Cassandra ......................................................227

brown, Christine .................................................275, 406

brown, Christine e. .............................198, 204, 247, 640

brown, Devin .............................................................230

brown, harrison C. ............................................456, 728

brown, James ............................................................468

brown, nolan..............................................................87

brown, robert h. .................................................90, 738

broxmeyer, hal e. .......................................................508

broyer, lucile .............................................................663

brugières, Pierre .........................................................367

brunetti-Pierri, nicola .....................................................47

brunger, Jonathan M. ..........................................318, 502

brunson, brandon .......................................................605

bruscaggin, Valentina ..................................................691

bu, Jie ......................................................................301

buccholz, Christian J. ....................................................59

buch, Pranali .............................................................473

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 68

Author Indexbuchholz, Christian J. ..................................201, 506, 654

buchholz, Tatjana ...............................................306, 592

buchholz, Veit r. ........................................................506

buckland, Karen .........................................................690

buckley, Suzanne M. K. ...............................305, 437, 609

buckvold, Megan .........................................................25

budde, elizabeth ........................................................640

budde, lihua e. ..........................................................745

budzynski, ewa ....................................................88, 299

bueren, Juan A. ..........................................228, 558, 753

buggage, ronald .......................................................249

buj bello, Ana ...................................................578, 629

buj-bello, Ana ............................................................322

buj-bello, Anna ..........................................................498

bukhai, Kanit ...............................................................10

bulysheva, Anna ........................................................491

bumcrot, David ..................................124, 317, 572, 732

büning, hildegard ......................................................306

bunker, Alex ..............................................................642

buquicchio, Frank .......................................................572

burghardt, elliot ..........................................................263

burghes, Arthur h. ......................................................480

burke, robert e. .........................................................370

burnett, John C. ..........................................................172

burnham, brenda .........................................................92

burtner, Christopher .....................................................284

burton, Matthew.........................................................374

buscara, laurine .........................................................498

busch, Dirk h. ...........................................................506

busch, Korie ................................................................49

busch, Steve ..............................................................399

bushman, Frederic D. ..................................................754

bussy, Cyrill ...............................................................450

butler-browne, Gillian ..................................................500

buttery, Philip .............................................................367

buttimer, Melissa ........................................................473

byrd, Tiara ................................................................392

byrne, barry ..............................................................372

byrne, barry J. ...........................................180, 382, 472

byrne, James A. .........................................................347

byrne, leah C. ...........................................................617

Cacace, Vincenzo ......................................................724

Caccavelli, laure .......................225, 231, 425, 689, 754

Caffrey, elisa .............................................................365

Cagnard, nicolas ......................................................754

Cai, hongliang .........................................................509

Cai, yujia .................................................127, 533, 574

Cairo, Mitchell S. .........................................................75

Calabria, Andrea ...................2, 212, 529, 537, 674, 681

Calcedo, roberto ...........................................82, 88, 299

Calero-Garcia, Miguel ................................................140

Callejas, David ............................................................50

Camarena, Joab ....................................................39, 43

Camboni, Marybeth ...................................162, 352, 698

Camisa, barbara .......................................512, 637, 752

Campbell, Dakota ..............................................221, 229

Campbell-Thompson, Martha ..........................................24

Campochiaro, Peter ....................................................249

Campo Fernandez, beatriz ..................................118, 230

Canales, Mathieu .......................................................327

Cancedda, laura .........................................................53

Cancelas, Jose A........................................................444

Candotti, Fabio..........................................................280

Cannon, Paula ...........................................................734

Cannon, Paula M. ......................................................330

Cantero, Gloria ...........................................................45

Cantore, Alessio ................................................286, 756

Canver, Matthew C. ...................................................282

Cao, huibi ...............................................................324

Cao, Jay J. ................................................................632

Cao, Pengpeng .........................................................209

Capasso, Cristian ..............................408, 642, 659, 661

Capasso, Paola .........................................................729

Capo, Valentina ...........................................................42

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 69

Author IndexCarbonaro, Denise .......................................................32

Carbonaro-Sarracino, Denise A. ...................................764

Cardella, Davide ...............................................408, 642

Cardozo, brynn .........................................................167

Carette, Jan e. ...................................................293, 478

Carey, brenna .......................................................21, 35

Carlier, Pierre ............................................................503

Carlile, Marc .............................................................600

Carlon, Marianne S. ...................................................175

Carlson-Stevermer, Jared ......................................335, 575

Carmo, Marlene ........................................................140

Carmona, Vitor ..........................................................582

Carnes, Aaron ...........................................................712

Carpentier, Arnaud .....................................................527

Carpizo, Darren .........................................................262

Carrig, Sean .............................................................550

Carter, barrie .............................................................171

Cartier-lacave, nathalie ................................................59

Carvalho, Carla .........................................................452

Carvalho, livia ......................................29, 80, 594, 608

Casado, José A. ........................................................753

Castellanos, ruth M. ...................................................482

Castellanos rivera, ruth M. ..........................................504

Castello, raffaele .........................................................47

Castiello, Maria C. ......................................................42

Castiello, Maria Carmina ............................................280

Castleton, Anna ...........................................................65

Casucci, Monica ...............................................512, 752

Catalá, Albert ............................................................753

Catalano, Jennifer G. ..................................................245

Cathomen, Toni..................................................325, 692

Cattaneo, roberto ..............................................106, 757

Catz, Sergio D. .............................................................4

Cavazzana, Marina ................ 225, 231, 279, 425, 559, 560, 682, 689, 754, 759

Cecchini, Sylvain .......................................................541

Cederbaum, Steven D. ..................................................45

Cemazar, Maja .........................................................495

Cepeda, Diana .........................................................607

Certo, Michael ..........................................................277

Cerullo, Vincenzo ...............................408, 642, 659, 661

Cerveca Carrascon, Victor ...........................................642

Cesana, Daniela ............................................2, 529, 681

Chahal, Parminder .............................................292, 708

Chalishazar, Aditi .......................................................732

Chalk, Claudia ......................................................21, 35

Chamberlain, Jeffrey S. ................................380, 499, 501

Chamberlain, Joel r. ...................................................501

Chamberlain, nicolas .................................................280

Chamoun-emanuelli, Ana M. ........................................602

Champion, Kim ..........................................................452

Chan, Frieda .............................................................284

Chan, hon Fai ...........................................................242

Chan, Jennifer A. ........................................................601

Chan, Jeremy Soon Kiat ...............................................216

Chan, Jerry K. y. ........................................................296

Chan, Ken y. .............................................................252

Chandler, randy J. ................................48, 168, 359, 613

Chang, brenda ..........................................198, 204, 275

Chang, esther h. .........................................................72

Chang, Katherine M. ..................................................347

Chang, lung-Ji ...................................................195, 632

Chang, Wen-Chung ...........................................204, 406

Channon, Keith ............................................................19

Chapdelaine, Pierre ....................................................561

Chapman, Michael S. ...................................12, 293, 478

Chariou, Paul ..............................................................89

Charles, Severine ...............................................158, 595

Charlins, Paige ..........................................................532

Charrier, Sabine .........................................................690

Charton, Karine .........................................................377

Charville, Gregory .....................................................722

Chateau, Morgan ......................................................330

Chavas, Joel ..............................................................268

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 70

Author IndexChavez, Julio C. ........................................................745

Chaw, Ching ............................................................600

Chee, Dickson S. .......................................................477

Chellappan, Malathi ...................................................525

Chen, Allshine ...........................................................489

Chen, Alvin .......................................................365, 471

Chen, Cheng ............................................................202

Chen, Chun-yu...................................................518, 666

Chen, haifeng .............................................................97

Chen, hsu-yu .............................................................330

Chen, Kong y. ...........................................................353

Chen, Mong-Jen ...................................................84, 632

Chen, Myra J. ............................................................601

Chen, Shu-Jen ............................................................760

Chen, yi-Wen ............................................................153

Chen, yifeng .............................................................185

Chen, Zhilei C. ..........................................................602

Chenegros, Guillaume ................................................268

Cheng, Jason ............................................................483

Cheng, Seng .............................................249, 301, 329

Cheng, Seng h. ..................................................92, 621

Cherel, yan ...............................................................503

Cherif, Khadija ..........................................................321

Cherqui, Stephanie ...............................32, 163, 336, 350

Cherukuri, Pratima ......................................................707

Cheung, Gordon Weng Kit ..........................................455

Cheung, Jennifer G. ....................................................680

Chevronnay, héloïse P. Gaide ......................................336

Chien, yin-hsiu ..........................................................372

Childers, Martin .........................................................498

Chin, Mario P. ...........................................................545

Chiocca, ennio A. ......................................................667

Chipman, Paul ...........................................................294

Chiruvolu, Vijay ..................................................287, 650

Chitranshi, nitin .........................................................188

Chiuchiolo, Maria ......................................................471

Chiuchiolo, Maria J. ...................................................755

Chiyo, Tomoko ..................................................337, 378

Cho, Jun-ho ..............................................................165

Choi, eun-young.........................................................615

Choi, Jang-Gi ............................................................574

Choi, Joung-Woo ...............................................113, 177

Choi, Jung-Woo .................................................110, 115

Choi, Kenny ..............................................................650

Choi, Kyujoo .............................................................527

Choi, Margaret ............................................................85

Choi, Moon Jung ...............................................155, 590

Choi, uimook ......................................................41, 557

Choolani, Mahesh ......................................................296

Chorny, Michael ........................................................174

Chosa, Keisuke ..........................................................122

Chou, Janice .............................................................165

Chou, Janice y. ..........................................................350

Choudhury, Shreyasi ...................................................620

Chouteau, Myriam .....................................................425

Chretien, Stany ..........................................................279

Christiansen, lauryn ............................................221, 229

Chroscinski, edward A. ...............................................227

Chtarto, Abdelwahed .................................................610

Chu, Alice ..................................................................61

Chu, Kirk ..................................................................737

Chuah, Marinee ........................................................756

Chuah, Marinee K. .............................................120, 628

Chudasama, Priya ......................................................134

Chulay, Jeffrey D. ...........................................24, 88, 299

Chung, C. ................................................................401

Chung, Janet .............................................................458

Church, Kathleen ................................................480, 497

C. hyde, Stephen ......................................................431

Ciceri, Fabio .............................................512, 637, 752

Cieri, nicoletta ..........................................................752

Ciesielska, Agnieszka .................................................621

Cioroch, Milena...........................................................19

Cirak, Sebahattin .......................................................584

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 71

Author IndexCittaro, Davide ..........................................................531

Clark, Danielle n. ......................................................764

Clark, K. r. .......................................................460, 710

Clark, K. reed ...................................................304, 760

Clark, Karen C. .........................................................263

Clark, reed ...............................................................227

Clarke, Don ..............................................................430

Clark-Matott, Joanne ...................................................376

Claver, bep ...............................................................610

Cleaver, brian ............................................................472

Clegg, D. o. ............................................................445

Clement, nathalie ................................................94, 472

Clough, Courtnee .......................................132, 569, 683

Cloughesy, Timothy .......................................................61

Cmielewski, Patricia ....................................................711

Cocchiarella, Fabienne ...............................................488

Cochran, Christina .....................................................763

Cock, Terrie-Anne ...............................................145, 260

Cockle, Julia ..............................................................197

Coghlan, Sarah .........................................................184

Cogné, benjamin .......................................................390

Cohen, bruce M. .......................................................742

Cohen, Justus b. .................................................111, 114

Collaud, Fanny ............................................77, 158, 595

Collins, Casey P. ............................................................6

Collins, McKensie ..............................................124, 572

Collins, robert h. .......................................................276

Comander, Jason ....................................................29, 80

Combal, Jean-Philippe ...........................................83, 267

Conlon, Thomas .........................................................472

Conlon, Thomas J. ......................................................180

Conner, Joe ...............................................................666

Connolly, Mary M. .....................................................743

Considine, Michael ....................................................237

Constable, ian J. ........................................................248

Contestabile, Andrea ....................................................53

Contreras-ramos, Alejandra .........................................246

Conway, Anthony .................................................37, 432

Cook, Philip ..............................................................265

Cooksley, renee D. ....................................................485

Cooper, Aaron r. .......................................................118

Cooper, Jonathan D. .....................................................27

Cooper, laurence J. n. .......................................278, 509

Cope, leslie ..............................................................237

Corat, Marcus ...........................................................527

Corat, Marcus A. F. ....................................................564

Corbau, romuald .......................................................185

Corcoran, Sarah ........................................................480

Corna, Stefano ..................................................130, 703

Corti, Manuela ..........................................................472

Cosette, Jérémie .........................................................693

Cossu, Giulio ............................................................384

Cost, Gregory J. ..................................................37, 432

Cotta-ramusino, Cecilia .......................................576, 732

Coulombe, nathalie ...................................................292

Coulon, Flora ............................................................768

Counsell, John r. ........................................................609

Courtoy, Pierre J. ................................................163, 336

Couto, linda b. ............................................................31

Couture, l. ................................................................445

Couture, larry ............................................................244

Couzin, Chloé ...........................................................231

Cowley, Christopher J. .........................................612, 619

Cox, Andrew.............................................................167

Cram, Sara ...............................................................309

Creneguy, Alison ........................................................768

Cripe, Timothy ...........................................................666

Cripe, Timothy P. ........................................................518

Crisostomo, Jeannette ..................................................746

Crosariol, Marco........................................................699

Crosson, Sean M. ......................................................159

Cruz, C. russell ...........................................................75

Cruz, Conrad r. ........................................................685

Cruz, Conrad russell ..................................................763

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 72

Author IndexCrystal, Jessica ..........................................................272

Crystal, ronald ....................................................36, 309

Crystal, ronald G. .............................365, 471, 474, 755

Cui, Shengjie ..............................................................98

Cullis, Pieter ..............................................................614

Culme-Seymour, emily J. ...............................................452

Cunha-Santos, Janete ..................................................582

Cunningham, Sharon ..................................................169

Curiel, David T. ..................................................517, 519

Curinga, Gabrielle .............................................316, 683

Curion, Fabiola ..................................................320, 736

Currier, Mark .....................................................518, 666

Currlin, Seth ..............................................................159

Cyckowski, laura .......................................................265

Czink, elena S. ............................................................68

Dacanay, Anthony ......................................................723

da Cunha, Mélanie F. .................................................175

Dai, lisheng ..............................................................154

Dai, Mei ...................................................................521

Dai, yi .....................................................................721

Daley, heather .............................................................71

Dalkara, Deniz ..........................................................268

Dallas, Michael .........................................................505

Dalle, bruno ..............................................................464

Dalle, Jean-hugues .....................................................250

Damha, Masad J. .......................................................586

Dane, Allison P. ..........................................................298

Dang, W. .................................................................445

Dang, Wei ...............................................................244

Dang, ying ...............................................................574

D’Angelo, Armando ....................................................756

Danièle, nathalie .......................................................629

Dansereau, Colleen ....................................................250

Dao, Tao ..................................................................393

D’Apuzzo, Massimo ...................................................247

Darou, Shannon .........................................................714

Das, Asha ...................................................................61

Das, Atze T. ......................................................331, 610

Dasgupta, Anindya .....................................................402

Dashkoff, Jonathan ......................................................616

Dassie, Justin P. ..........................................................263

Dastidar, Sumitava ......................................................628

Davey, roshni ............................................................138

David, McCarty .........................................................402

David, robert ............................................................338

Dávid, Stephan ............................................................15

Davidoff, Andrew M. ..........................................547, 571

Davidson, robert J. .......................................................52

Davidsson, Marcus .....................................................542

Davies, Jane C. .........................................................534

Davies, lee A. ...........................................................534

Davila, blachy ...........................................................685

Davis, Timothy h. .......................................................131

Davulco, omar ..........................................................293

Davulcu, omar ..........................................................478

Davydova, Julia ..................................413, 421, 519, 639

De, bishnu ..................................................................36

De, bishnu P. .....................................365, 471, 474, 755

de Alencastro, Gustavo ...............................................539

Dean, David A ..........................................................490

Dearborn, Josh T. ..........................................................27

De boeck, Christiane ..................................................175

Debyser, Zeger ..........................................................175

DeClercq, Joshua J. .....................................................133

Degli-esposti, Mariapia A. ...........................................248

Déglon, nicole ..................................................327, 463

de haar, Colin ..........................................................404

de Jong, Jondavid .......................................108, 181, 422

de Jonge, hugo .........................................................175

DeKelver, russell .................................................484, 485

Dekkers, Johanna F..............................................175, 534

Delacono, Cheryl .......................................................249

Delatycki, Martin b. ....................................................374

de lázaro, irene .................................................384, 450

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 73

Author IndexDel’Guidice, Thomas ..................................................603

Delhove, Juliette M. .....................................................437

Delhove, Juliette M. K. M. ............................................305

Dell’Aquila, Fabio.........................................................28

Della Valle, Patrizia .....................................................756

de Mollerat du Jeu, xavier ............................................462

de Montalembert, Mariane ..........................................279

de Montigny, Charline ..................................................54

Deniger, Drew C. ...............................................272, 391

Denton, nicholas .......................................................518

Deplazes, Sereina ......................................................169

de Prisco, nicola ...............................................320, 736

Dequesne, Thomas .....................................................327

De ravin, Suk see.................................................41, 557

Dergilev, Konstantin ....................................................447

Dergilev, Konstantin V. .................................................448

de Saint basile, Geneviève ..................................682, 759

Deshmukh, harshavardhan ...........................................131

Deshotels, Matthew r. .................................................375

De Silva Feelixge, harshana S. .....................................765

Dessainte, Michel .......................................................464

Devaux, Marie ...........................................................697

Dever, Daniel .............................................................123

Dever, Daniel P. ......................................................39, 43

Deverman, benjamin e. ...............................................252

De Witte, olivier ........................................................610

Dey, Aditi............................................................65, 663

Dheer, yogita .............................................................188

Diaconu, iulia ............................................................277

Diao, yong ...............................................................545

Diaz, rosa ..................................................................63

Díaz de heredia, Cristina ............................................753

Dickey, David D. ................................................263, 744

Dickson, George ................................500, 503, 628, 634

Dickson, Patricia i. ..............................................430, 615

Dickson, Patti .............................................................364

Diep, Jonathan ...................................................293, 478

Díez, begoña ............................................................558

DiGiusto, David .........................................................734

Di Matteo, Mario .......................................................628

Dimitrov, Dimiter .........................................................649

Diomede, lorenzo ......................................................631

Dionisio, Francesca ....................................................531

Di Paolo, nelson C. .....................................................13

DiPersio, John ............................................................276

DiPietro, Marti .............................................................52

Di Salvo, Maria T. ......................................................488

Discenza, Claire ........................................................186

Discher, Dennis ..........................................................511

Di Serio, Clelia ..................................................226, 531

Di Tomaso, Tiziano .......................................37, 130, 286

DiTommaso, Tia .........................................................573

Djamai, hanane ........................................................697

Djerroudi, lounes ........................................................225

Dmitriev, igor .............................................................517

Dodd, J. ...................................................................445

Doering, Christopher ...................................207, 402, 670

Doering, Christopher b. .......................................456, 728

Doerner, Johannes ......................................................105

Dolgachev, Vladislav ...................................................173

Domingo, ron u. ...............................................156, 591

Domingues, Cleyton ....................................................354

Domire, Jacqueline S. ..................................................386

Donahue, robert ........................................................523

Donate, Amy .....................................................358, 491

Donate, Amy l. ..........................................................360

Dondi, Francesco .......................................................724

Dong, biao ...............................................................545

Dong, lijin ................................................................266

Dong, linda ..............................................................399

Dong, lujia ...............................................................195

Donnelley, Martin .......................................................711

Donnou, Sabrina ........................................................694

Donohue, Kristen ........................................................262

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 74

Author IndexDopeso-reyes, iria G. .................................................370

Dörner, Johannes ..........................................................14

Dotson, Cedric D. ......................................................159

Dotti, Gianpietro ..........................................76, 507, 748

Douar, Anne M. .........................................................268

Doudna, Jennifer A. ....................................................716

Douek, Daniel C. .......................................................272

Douglas, Gillian ...........................................................19

Drakopoulou, ekati .....................................................222

Draper, Christine ................................227, 695, 696, 760

Driscoll, Christopher ..............................................64, 197

Dropulic, boro ...................................................532, 648

D’souza, Saritha .............................................................4

Du, Fang ..................................................................459

Duan, Dongsheng ..............................................482, 499

Duan, Sean x. ...........................................................499

Duchateau, Philippe ............................................651, 676

Duclert, Aymeric .........................................................651

Duddy, William ..........................................................623

Dudgeon, Crissy ........................................................262

Duebel, Jens ..............................................................268

Dufour, Valerie ...........................................................617

Dumitru, raluca ..........................................................528

Dunbar, Cynthia .................................................523, 527

Dunbar, Cynthia e. .....................................................564

Duncan, Christine .......................................................250

Duncan, F. J. ..............................................................698

Duong, Thu T. ....................................................553, 556

Du Pasquier, renaud ...................................................327

Dupont, Jean-baptiste ..................................................390

Dupuis, Colleen M. ............................................460, 710

Durand, Amandine .............................................682, 759

Durand, romain .........................................................390

Duthoit, Christine ........................................................538

Dutta, roop ...............................................................285

Duttagupta, Priyanka ...................................................261

Düzgüneş, nejat ........................................................680

Dvorchik, igor ............................................................497

Dynan, William S. ......................................................563

earley, lauriel F. ...........................................................12

eaton, Samuel C. .......................................................251

ebina, Wataru ...........................................................568

echigoya, yusuke .......................................................623

ecsedy, Jeffrey ...........................................................666

edelblute, Chelsea ......................................................491

edelman, Aleksander ..................................................175

edes, inan .................................................................506

edwards, heather ......................................................361

efimenko, Anastasia y. ................................................448

ehrhardt, Anja ........................ 14, 15, 17, 18, 103, 104, 105, 106, 194, 410, 757

ehrke-Schulz, eric .............................14, 15, 18, 103, 104

eichler, Florian ...........................................................295

eichler, Florian S.........................................................250

eidenschink, ben ........................................................413

einsele, hermann .........................................................73

elias, ivet ....................................................................50

elias, Meg ................................................................745

eliott, Dean ...........................................................29, 80

elkordy, Amal A. ........................................................600

elkouby, liron...............................................................52

ella Menye, Jean-rene .................................................170

ellinwood, n. M. .......................................................439

elliot, Gene .................................................................46

elliott, Gene ..............................................................357

elliott, Sarah T. C. ......................................264, 433, 644

ellis, Scott ...................................................................33

ellison, Stuart M. ........................................................364

elMallah, Mai ...........................................................167

el nemer, Wassim ......................................................279

el refaey, Mona ...................................................20, 577

elsedawy, noura .........................................................67

elshikha, Ahmed...........................................................84

elshikha, Ahmed S. .....................................................632

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 75

Author Indexelverman, Matthew .....................................................498

embree, heather ........................................................532

emerson, Corey ...........................................................13

emter, Craig ..............................................................499

enblad, Gunilla ..........................................................748

endo-Takahashi, yoko ..................................................337

engeland, Christine e. ...........................................68, 423

epping, Madeline ......................................................357

epstein, benjamin e. ...................................................119

eriksson, emma ..................................................516, 662

ermolova, natalia V. ...................................................735

escobar, Giulia ..........................................................752

espejo, Andrew .........................................................101

espinoza, Diego ........................................................523

espinoza, Fernando l. .................................................205

esposito, Martina .......................................................179

essand, Magnus ........................................................748

estrada, Amara h. .....................................................180

ettyreddy, Adarsh r. ....................................................502

eubank, Tim ..............................................................518

evans, Annicka ............................................................89

evans-Galea, Marguerite V. ..........................................374

evens, hanneke .........................................................120

everson, elizabeth ......................................................687

everson, elizabeth M. .....................................................1

evgin, laura ........................................................63, 197

ewend, Matt .............................................................528

excoffon, Katherine J. D. A. ..................................176, 554

exline, Colin ..............................................................734

eyquem, Justin ............................................................274

Fairbanks, Carolyn .................................................25, 86

Fairchild, Craig ..........................................................473

Fakhiri, Julia...............................................................254

Falabella, Paolo ...........................................................31

Falcone, laura ...........................................................512

Falk, Darin J. .............................................................382

Falzarano, Darryl .......................................................436

Famà, rosella ............................................................691

Fan, Fan ...................................................................309

Fan, yong .................................................................239

Fang, Juan ................................................................668

Fang, Justin ...............................................................572

Farinelli, Giada ..........................................................226

Faris, Kaitlyn ..............................................................472

Farmer, Andrew .........................................................313

Fehse, boris ...............................................................475

Feldman, heinrich ......................................................436

Felsburg, Peter J. ........................................................284

Fenard, David ...................................................464, 536

Fenelon, Gilles ...........................................................367

Feng, yinglu ................................................................95

Ferla, rita .................................................................468

Fernandez-Zapico, Martin ............................................413

Ferrari, Giuliana .........................................................235

Ferrari, Samuele ...........................................................37

Ferre, Tura ...................................................................50

Ferreira, Valerie ..................................310, 424, 700, 701

Ferrone, Soldano ........................................................507

Ferrua, Francesca .......................................................280

Ferry, Arnaud .............................................................500

Fielding, Adele K. ................................................65, 663

Figliola, Matthew J......................................................278

Figueiredo, Constanca ................................................476

Figueiredo, Monica D. ................................................387

Fil, Daniel .................................................................364

Filareto, Antonio .........................................................312

Filhol, Jean Marie .......................................................651

Filipeanu, Catalin .......................................................375

Finer, Mitchell ............................................................229

Finocchiaro, Gaetano .................................................507

Fire, Andrew .............................................................289

Firor, Amelia e. ..........................................................647

Firrman, Jenni .............................................................545

Fischer, Andrew J. .......................................................612

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 76

Author IndexFishbein, ilia ..............................................................174

Fisson, Sylvain ...........................................................694

Fitoussi, Serge .....................................................83, 267

Fitzgerald, Caitlin .......................................................611

Fitzgerald, Julie A. ..............................................612, 619

Fitzpatrick, Zachary ....................................................595

Flagiello, Thomas .......................................................365

Flanigan, Kevin ..........................................................497

Flanigan, Kevin M. .............................................328, 624

Flannery, John G. .......................................................617

Flaschel, erwin ...........................................................465

Fleischer, lauren .................................................207, 670

Flingai, S. .................................................................401

Flingai, Seleeke .........................................264, 428, 433

Fllingai, Seleeke .........................................................644

Flores, linda ..............................................................209

Flotte, Terence r. ..........................................24, 183, 702

Fogel, Jonathan ..........................................................282

Fol, romain .................................................................59

Foley, reghan ............................................................715

Follenzi, Antonia ........................................................691

Foltz, Steven J. ...........................................................377

Fong, Mei .................................................................313

Forman, Stephen ........................................................275

Forman, Stephen J. .....................198, 204, 247, 406, 640

Forsayeth, John ..........................................................621

Förster, reinhold .........................................................766

Fortin, Marylene .........................................................192

Foster, Aaron .............................................................746

Fouladi, naghmeh .......................................................33

Fousek, Kristen ...........................................................392

Foust, Kevin D. ...........................................480, 612, 619

Fraldi, Alessandro .......................................................724

Franczek, Meghan .....................................................666

Fraysse, bodvael ........................................................503

French, Martyn A. ......................................................248

Frey ii, William h. ........................................................25

Freytag, Svend o. ......................................................514

Friedman, Alan ..........................................................237

Friedman, Kevin .........................................................747

Friehs, Karl ................................................................465

Frock, richard ............................................................568

Fröhling, Stefan ..........................................................134

Fronza, raffaele .................................................232, 470

Fu, haiyan ................................................162, 352, 698

Fu, Jun ..................................................................14, 17

Fu, richard y. ................................................5, 434, 441

Fu, xinping ................................................................126

Fuchsluger, Thomas .....................................................306

Fuerst, yaron ..............................................................598

Fujii, Kentaro .............................................................667

Fujii, Tsutomu .............................................................660

Fujiwara, hiroshi ........................................................193

Fukuda, Saori ............................................................660

Fukuhara, hiroshi ...............................................412, 664

Fuller, Dave ...............................................................472

Furling, Denis .............................................................322

Fusaro, Mathieu .........................................................231

Gabriel, richard ................................................121, 232

Gabrion, Aurélie ........................................................425

Gagné, Jack ..............................................................353

Gaide Chevronnay, heloise P. ......................................163

Gaillet, bruno ....................................321, 341, 555, 708

Gaj, Thomas .............................................................125

Galanis, evanthia .........................................................62

Galen, James e. .........................................................191

Galetto, roman ..........................................................397

Galfin, Tim ................................................................186

Galimberti, Stefania ....................................................468

Galinsky, ilene .............................................................71

Gallina, Pierangela.............................................529, 681

Gallinger, Julia o. ......................................................448

Galperin, Anna ..........................................................170

Galvin, Alice .............................................................269

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 77

Author IndexGaly, Anne .................83, 267, 464, 536, 694, 753, 754

Gammon, elizabeth ....................................................517

Gandhi, nimisha .......................................................641

Ganjawala, Tushar .......................................................98

Ganser, Arnold ..........................................................476

Gao, Guangping .......... 7, 9, 58, 81, 90, 157, 290, 295, 349, 351, 366, 570, 669, 733, 739

Gao, huiping ............................................................202

Gao, xiuhua..............................................................191

Gao, xueqin .............................................240, 446, 449

Gao, yandi .........................................................20, 577

Garachtchenko, Tatiana...............................................313

Garces, eudean .........................................................684

García, Miquel ....................................................50, 348

Garcia-bravo, Maria ...................................................228

García de Andoaín, nagore ........................................753

Garcia-Gomez, Maria ................................................228

García-V, P. ...............................................................453

Gargaun, Ana ...........................................................151

Garnier, Alain ....................................................321, 603

Garofalo, Mariangela .........................408, 642, 659, 661

Garrett, Tracy ............................................................747

Gartner, Jared ............................................................272

Gartner, Jared J. .........................................................391

Gaskin, Chantelle .........................................................88

Gasmi, Mehdi ...........................................................607

Gaspar, bobby ..........................................................690

Gaspar, h. bobby ......................................140, 250, 325

Gaucher, eric A. ........................................................456

Gautron, Anne-Sophie .................................................651

Gayon, régis ............................................................538

Gazquez, Cristina ......................................................164

Gee, Adrian ..............................................................645

Gee, James ...............................................................265

Geekiyanage, hirosha ..................................................62

Gehrke, Stephan ........................................................186

Geldres, Claudia .........................................................74

Gelinas, Jean-Francois .................................................704

Gemberling, Matthew .................................................482

Gendron, Tania F. .......................................................738

Gendron, William ........................................................64

Generous, Alex ..........................................................297

Genovese, Pietro ....................................37, 42, 130, 752

Genovesse, Pietro.......................................................558

Gentner, bernhard ................................37, 226, 235, 752

Georgakopoulou, Afroditi ............................................291

George, Stephan n....................................................728

Georger, Christophe ...................................................390

Georgomanoli, Maria .................................................222

Gérard, Catherine ..............................................321, 565

Gerasch, laura ..........................................................469

Gerdts, ethan ............................................................204

Gerdts, ethan A. ........................................................406

Gergen, Janina ..........................................................768

Gernoux, Gwladys .........................................24, 90, 702

Gersbach, Charles A. ...................... 311, 317, 318, 385, 482, 502, 504, 525, 626

Gessler, Dominic J. .......58, 157, 349, 351, 366, 733, 739

Gesualdo, Carlo ................................................320, 736

Ghassemi, Saba ........................................................203

Ghivizzani, Steven C. .................................................723

Ghobadi, Armin .........................................................745

Gholizadeh, Shervin ...................................................368

Ghoshal, Kalpana ......................................................737

Giangrande, Paloma h. ..............................148, 263, 744

Gibson, Alana l. ........................................................613

Gicquel, evelyne ........................................................377

Giese, Morgan ..........................................................429

Giesige, Carlee r. .....................................................625

Giffear, Mary ............................................................505

Gijsbers, rik ..............................................................175

Gilbert, Jonathan ........................................................573

Gilbert, rénald ..........................292, 321, 341, 555, 708

Gilchrist, Megan ........................................................208

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 78

Author IndexGiles, April r. ..............................................................82

Gil-Farina, irene .........................................................296

Gilger, brian ..............................................................544

Gill, Deborah r. .........................................534, 704, 743

Gill, Saar ..........................................................273, 399

Gilmore, William b.....................................................347

Ginn, Samantha l. ......................................................298

Gisch, Donald .....................................................44, 288

Gissen, Paul ..............................................................250

Gittes, George K. .......................................................303

Giuliani, erica .............................................37, 130, 703

Giunti, Massimo .........................................................724

Gjata, bernard ..........................................390, 503, 690

Glass, Katherine A......................................................502

Glazyrin, yury e. ........................................................151

Glimm, hanno ...........................................................134

Glorioso, Joseph ........................................................520

Glorioso, Joseph C. ............................................111, 114

Gloskowski, Sebastian ................................................124

Gluck, Sarah .............................................................269

Gluckman, eliane .......................................................231

Gluhanyuk, evgeny V. .................................................448

Gnanandarajah, Josephine S. .......................................750

Go, William y. ..........................................................745

Godbout, Jonathan P. ..................................................619

Goddard, Melissa ......................................................498

Goedland, Madelyn ...................................................575

Goethe, ralph ...........................................................766

Gogishvili, Tea .............................................................73

Goins, William ..........................................................520

Goins, William F. .......................................................111

Gojo, Satoshi ............................................................340

Goldberg, rebecca ....................................................173

Golshani, Peyman ........................................................45

Goltsman, haim .........................................................598

Gombash, Sara e. .............................................612, 619

Gomi, rika..................................................................22

Gonçalves, Manuel A. ................................................404

Gong, yi ..................................................................295

Gonoi, reina .............................................................395

Gonzales, Paul ..........................................................509

Gonzalez, iria .............................................................99

Gonzalez, nancy ......................................................734

Gonzalez Aseguinolaza, Gloria ............................310, 700

González-Aseguinolaza, Gloria ..............99, 164, 300, 370

Goode, Tamara .........................................227, 346, 760

Goodman, Spencer ....................................................336

Goodwin, Alicia ........................................................684

Goodwin, Marianne ...................................................123

Goraczniak, rafal ..............................................259, 262

Gori, Jennifer l. ..........................................................732

Gorospe, Myriam .......................................................146

Gosiker, bennett .........................................................358

Goss, James r. ..........................................................184

Goss, Kendrick ..........................................................229

Goto, Fumihiro ...........................................................224

Gotta, Gregory ..........................................................124

Gottschalk, Stephen ..............................................76, 645

Gouble, Agnès ..........................................................397

Gouello, Gaetane ......................................................367

Gould, Gregory C. ....................................................554

Gourdon, Geneviève ..................................................322

Govindasamy, lakshmanan ............................................82

Grabowski, Gregory...................................................521

Gradinaru, Viviana .....................................................252

Graebner, Allison K. ...................................................376

Graham, Anne-renee ..................................................724

Graham, Michael ...............................................260, 500

Graham, Stuart l. .......................................................188

Grande, Shannon ......................................................747

Grandi, Paola ...........................................................520

Grange, robert ..........................................................498

Gravel, William-Édouard .............................................565

Gray, John T. .............................................................547

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 79

Author IndexGray, Steven .....................................................363, 551

Gray, Steven J. .............................57, 362, 604, 611, 725

Gray-edwards, heather ...............................................605

Green, Jordan J. .........................................................494

Greenberg, Phil .........................................................288

Greengard, Judy ........................................................684

Greeno, edward ........................................................421

Gregori, Silvia ...................................................637, 681

Gregory, Philip ..........................................221, 229, 427

Gregory, Philip D. ........................................37, 734, 558

Gregory, richard i. .....................................................282

Greig, Jenny A. ..........................................................760

Greninger, Alex l........................................................765

Grez, Manuel ............................................................234

Grier, Alexandra ........................................................398

Griesenbach, uta .......................................................534

Griffin, Danielle..........................................................622

Griffin, Danielle A. .............................................386, 625

Grifoll, iris ...................................................................50

Grilley, bambi ............................................................717

Grimm, Dirk ..............................................254, 302, 470

Grønli, Janne .............................................................475

Gros, Alena ..............................................................272

Gross, Amanda l. ......................................................605

Grosse, Stefanie.........................................................302

Grosskopf, Anna ........................................................107

Gruber, harry ..............................................................61

Gruber, harry e. ........................................................205

Gruenert, Dieter .........................................................122

Gruenert, Dieter C. .....................................................332

Gruenert, luke ...........................................................332

Grünert, Anja ............................................................306

Gruntman, Alisha ...............................................702, 738

Gruntman, Alisha M..............................................24, 183

Gu, Shuo ..........................................................154, 737

Guay, David .............................................................603

Guda, Swaroopa .......................................................282

Guedan, Sonia ..........................................................515

Guenechea, Guillermo ................................................753

Guenther, Caitlin ..........................................................89

Guerrero, David T. ......................................................174

Guidry, Jessie J. ..........................................................375

Guijario, Maria V. ......................................................723

Guilak, Farshid ..................................................318, 502

Guilbaud, Mickaël .....................................................697

Guillaume, Corre .......................................................464

Guise, Theresa...........................................................417

Gulati, neetu ..............................................................13

Gumienny, Felecia ..............................................328, 624

Gunderson, Samuel ............................................259, 262

Guo, Charles C. ..........................................................72

Guo, hua .................................................................574

Guo, landys Z. ..........................................................178

Guo, linjie ........................................................191, 396

Guo, Ping .................................................................303

Guo, Siqi..................................................................491

Gupta, Ankit ..............................................................326

Gupta, Vivek .............................................................188

Gurda, brittney l. .......................................................611

Gurruchaga, Jean Marc ...............................................367

Guschin, Dmitry y. ........................................................37

Gutierrez-Martinez, Paula.............................................568

Gutiérrez-V, i. .............................................................453

Gutova, Margarita .....................................................443

Guyot, Valérie............................................................651

Guzman, Monica l. ....................................................397

György, bence ..................................................567, 594

haavisto, elina ..........................................................661

häberle, Johannes ..............................................169, 355

habib, nagy ............................................................671

hacein-bey Abina, Salima ...........................................754

hacein-bey-Abina, Salima ............................279, 425, 689

hack, Anniesha .........................................................459

hackett, Perry b. ........................................................439

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 80

Author Indexhadaczek, Piotr .........................................................621

haddad, Marie reine .................................................356

hadland, brandon K. ..................................................562

hafler, brian ..........................................................29, 80

hagberg, hans..........................................................748

hagin, David ......................................................40, 316

hajitou, Amin ............................................................672

hakim, Chady ...........................................................499

hakim, Chady h. .......................................................482

halary, Franck ...........................................................768

halder, Sujata ...........................................................727

hale, Ashley................................................................19

hale, Malika .....................................................398, 761

hall, bryan C. ...........................................................439

hall, John K. ..............................................................501

hallbook, helene .......................................................748

halle, olga K. ...........................................................766

hallwirth, Claus V. ..............................................298, 541

hamada, hizuki ........................................................580

hamada, Katsuyuki ....................................................420

hamada, Motoharu ....................................................213

hamel, Amanda ................................................221, 229

hamon, loic ..............................................................536

hampson, David r. ....................................................368

han, Fei ...................................................................182

han, Joohee ..............................................................639

han, Jun-Ming ...........................................................219

han, renzhi ........................................................20, 577

han, Sang-oh ......................................................49, 381

han, Su-yeon ............................................................102

han, Zongchao .................................................339, 342

hanauer, Julia ............................................................654

hanenberg, helmut ....................................................508

hanley, Patrick J. ..................................................75, 685

hanlon, Alexandra l. ..................................................760

hannan, Anthony J. ....................................................374

hansen, Carl .............................................................614

hansen, erik ..............................................................709

hansen, Gesine .........................................................522

hanson, leah ..............................................................25

hantraye, Philippe ......................................................367

happle, Christine .......................................................522

harada, yuichirou ......................................................144

hardin, Jeremy ...........................................................432

harding, Cary o. ......................................................355

hardt, olaf ...............................................................653

hargrove, Phillip ........................................................530

harkness, Ty ..............................................................335

harnof, Sagi .............................................................598

harper, Scott Q. ................................................386, 625

harrang, James ..........................................................492

harrington, elizabeth ..................................................353

harrington, Kevin .........................................................63

harris, reuben .............................................................64

harshbarger, Todd l. ...................................................247

hart, Stephen ..............................................................23

hart, Stephen l. .........................................................588

hartnor, Morgan ........................................................542

harvey, brandon ........................................................591

harvey, brandon K. ............................................156, 218

harvey, Mario ...........................................................603

harvie, Pierrot ...........................................................170

hasegawa, Mamoru ..................................................534

hashii, yoshiko ..........................................................395

hasle, nicholas .........................................................398

haspot, Fabienne .......................................................768

hassinen, Minna M. ...........................................461, 706

hattenbach, Jacob D. .................................................353

haugwitz, Michael .....................................................313

haurigot, Virginia .........................................................50

haurigot, Virginia A. ...................................................348

hauschka, Stephen D. .................................................380

hauswirth, William W. .........................................88, 299

havlicek, David F. ........................................................36

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 81

Author Indexhavunen, riikka .........................194, 405, 410, 515, 652

haworth, Kevin ....................................................44, 429

haworth, Kevin G. .............................................283, 767

hayashita-Kinoh, hiromi ......................................337, 378

hayward, Samuel b. ..................................................133

he, Qing ..................................................................459

he, ran ............................................................7, 9, 570

he, Zhenning ....................................................227, 481

heath, Jack M. ..........................................................732

heathward, emily J. ....................................156, 218, 591

hebben, Matthias ..............................................503, 690

heczey, Andras .................................................396, 645

heemskerk, Mirjam h. M. ...........................................746

heeney, Matthew M. ..................................................282

heffner, Garrett ..........................................................229

hegde, Meenakshi .....................................................392

heidbuechel, Johannes ..................................................68

heier, Jeff ..................................................................249

heikkilä, Kati .............................................................706

heilbronn, regine.......................................................549

hekele, Armin ....................................................138, 394

heller, Kristin .....................................................379, 622

heller, loree ..............................................................495

heller, richard ...........................................................491

hemmila, Mark r. ......................................................173

hemminki, Akseli ................194, 405, 410, 418, 515, 652

henckaerts, els ..........................................................256

hendel, Ayal .........................................................39, 43

henderson, Mark J. ....................................................218

henn, Alicia D. ..........................................................714

herath, Shan .............................................................634

hergott, lucas ............................................................327

hermann, Kip ............................................................347

hermine, olivier ........................................................279

hernandez, laura .........................................................32

hernández, S ............................................................453

hernández, Salomón ..................................................246

hernandez-Alcoceba, rubén ................................164, 300

herzog, roland .................................................472, 688

herzog, roland W. ................................................78, 79

heslan, Jean-Marie .....................................................768

heslop, helen e. ........................................190, 451, 751

hesnard, leslie ..........................................................697

hetherington, nicola A. ..............................................541

hetzel, Miriam ...................................................522, 766

hicks, Martin J. ..........................................................587

hicks, Michael r. .......................................................735

hickstein, Dennis D. ....................................................285

higgins, Tracy e. ........................................................534

high, Katherine A. ..........................................31, 52, 185

hinderer, Christian ......................................................346

hingtgen, Shawn .......................................242, 442, 528

hinkley, Sarah J. ........................................................641

hinton, D r. ..............................................................445

hirai, yukihiko ...........................................................308

hirano, Satoshi ..........................................................673

hiraoka, Kei ..............................................................673

hiriyanna, Suja ..........................................................266

hironaka, Takashi .......................................................534

hiroshima, Kenzo .......................................................211

hirsch, Matthew .............87, 93, 319, 394, 544, 579, 630

hirvinen, Mari ...................................................659, 661

hmitou, isabelle .........................................................225

ho, Ching yi ...............................................................97

ho, Michelle ...............................................................89

hoang, Tsvetelina P. ....................................................746

hoban, Megan D. .....................................................118

hochberg, Jessica ........................................................75

hocum, Jonah D.........................................................1, 6

hoeben, rob C. ........................................................404

hoeve, James n. Ver. ............................................88, 299

hoffman, Andrew .......................................................167

hoffman, brad e. .........................................................79

hoffman, eric ............................................................623

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 82

Author Indexhoffman, eric P. .........................................................584

hoffmann, Victoria ......................................................357

hogan, Daniel ...........................................................403

hogan, Mark ............................................................497

hogarth, Marshall W. .................................................584

hoglund, Martin ........................................................748

hogrel, Jean-yves .......................................................503

holditch, Sara ...................................................297, 307

holditch, Sara J. ........................................................172

holic, nathalie ..........................................................536

holland, Steven M. ....................................................285

hollander, Georg .......................................................718

hollenhorst, Monika i. .................................................175

holley, rebecca .........................................................364

holliday, lexie S. .......................................................632

hollis, roger P. ..................................................118, 230

holmes, Michael ................................................427, 558

holmes, Michael C. .............. 37, 38, 42, 133, 286, 432, 484, 485, 641, 684, 734

holmes, Micheal ........................................................752

holt, Jeff r. ................................................................269

honaker, yuchi ..........................................................398

hong, Jinwoo ....................................109, 112, 113, 116

hong, So gun ....................................................523, 527

hongmei l., li ...........................................................565

hontani, Koji .............................................................673

hood, Tiffany ............................................................466

hook, Kathryn ...........................................................612

hoover, Jamie C. .......................................................685

hoq, Fahmida .............................................................75

hornick, Andrew l. .....................................................438

horton, holly .....................................221, 229, 277, 747

horvath, Christopher ...................................................229

hosing, Chitra M. ......................................................745

hosoki, Kana ............................................................623

hossain, Jubayer A. ....................................................475

hotta, Akitsu ..............................................................565

houghton, benjamin C. ...............................................325

houston, Mike ...........................................................170

howard, Doug ..........................................................218

howe, Steven J. .........................................................609

hoxie, James .............................................................427

hoyler, birgit ...............................................................68

hsu, Charlie y. ..........................................343, 344, 601

hsu, D. ....................................................................445

hsu, David ................................................................244

hsu, Patrick D. ...........................................................317

hu, Jim .....................................................................324

hu, Shiu-lok ................................................................38

hu, yang ..................................................................271

hu, yu-Chen ..............................................................448

huang, hannah .........................................................328

huang, haoliang .......................................................271

huang, Jing li ............................................................519

huang, Kai-Wen ........................................................671

huang, lin-ya ............................................................727

huang, nianyuan ......................................................624

huang, Wei ......................................................157, 396

huang, yan ...............................................................724

huang, yong .............................................................737

huard, Johnny ...................240, 303, 446, 449, 635, 636

hubbard, brandon T. ..................................................168

hubbard, nicholas ...............................................44, 284

hubbard, nicholas W. .................................................40

hubbard, nick ..................................................316, 683

huber, nina ..............................................................252

huckfeldt, rachel .......................................................270

hudecek, Michael ........................................................73

hudry, eloise .............................................................616

huff, Amanda ..............................................................63

hughes, Michael ........................................................305

hui, Daniel J. .............................................................185

huls, Mary h. ...........................................................278

humayun, M. ............................................................445

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 83

Author Indexhumbert, olivier ........................................284, 288, 758

humbert-Claude, Marie ...............................................610

humeau, laurent ........................................................505

humphries, Daniel ......................................................283

humphries, Margaret ....................................................24

humphrys, Daniel ...............................................284, 683

hung, King .......................................................132, 569

hung, Michelle e. ......................................................496

hunter, David W. .......................................................439

huo, Jeffrey ...............................................................237

hurton, lenka V. .........................................................278

husain, Syed r. .........................................................214

huston, nick .............................................................317

hutzen, brian ....................................................518, 666

huusko, Vesa .............................................................706

hux, Jo Ann ...............................................138, 200, 394

huynh, Mai T. ...........................................................596

huynh, nguyen P. T. ...................................................502

hwu, Wuh-liang ........................................................372

hyddmark, Viktoria .....................................................723

hyde, Stephen C. ......................................534, 704, 743

hyland, Kendra A. .....................................................439

hyman, bradley T. ..............................................567, 616

iacovino, Michelina ....................................................430

iaffaldano, laura ........................................................179

ibanşez, Carlos ............................................................61

ibañez, Carlos e. .......................................................205

ibrahim Aibo, Daher ...................................................399

iché, Alexandra .........................................................538

ide, Kanako ..............................................................241

ignatova, irena ............................................................31

ijichi, nobuhiro ..........................................................414

ikeda, hiroaki ............................................................193

ikeda, norihiko ..........................................................144

ikeda, yasuhiro ..............................8, 172, 297, 307, 543

ilett, elizabeth ..............................................63, 197, 749

im, Chan Su ......................................................155, 590

imagawa, Kiwamu .............................................238, 378

inagaki, Akihito ..........................................................673

incao, Arturo A. .........................................................613

ingelsson, Martin ........................................................567

ingraham, Phoebe ......................................................388

innes, Alastair J. .........................................................534

ino, yasushi ...............................................412, 664, 665

inoko, Kazuho ...........................................................673

inoue, Makoto ...........................................................534

inskeep, Venette .........................................................521

iovine, barbara ..........................................................659

iozzo, renato V. .........................................................417

irons, eric ....................................................................13

ironside, Christina .......................................................767

ishibashi, hidetoshi .....................................................337

ishii, Akiko ................................................................308

ishii, Takehiko ............................................................593

ishikawa, yoshi ..................................................293, 478

itaka, Keiji ................................................................593

itano, Karen ..............................................................144

ito, hirotaka ..............................................................665

ivics, Zoltan .................................................................73

iwai, Miwako ....................................................412, 664

iyombe-engembe, Jean-Paul ..........................................561

izsvák, Zsuzsanna ..............................................106, 757

izumiya, yoshihiro ...........................................................8

Jackson, robert ............................................................92

Jacobsen, Kari ...................................................413, 519

Jacobsohn, David .......................................................685

Jae, lucas .................................................................478

Jae, lucas T. ..............................................................293

Jaeger, Dirk .........................................................68, 423

Jaen, Maria luisa .........................................................50

Jaffrès, Paul-Alain ........................................................597

Jahn, lorenz ..............................................................746

Jain, Minal ................................................................715

Jais, Jean-Philippe .......................................................754

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 84

Author IndexJakobsen, bent K. .......................................................192

Jakobsen, Martin r. ....................................................127

James, rachel ............................................................579

Jamet, Thibaut ............................................................629

Jamie-ramirez, Cristina ................................................415

Jan, Majib ................................................................735

Janssens, Virginie ........................................................163

Jantz, Derek .......................................138, 200, 394, 579

Janz, Sebastian ............................................................14

Jares, Alexander .........................................................647

Jarjour, Jordan ....................................277, 323, 398, 761

Jarmin, Susan .............................................500, 628, 634

Jaulin, nicolas ...........................................................697

Javier, h. ..................................................................445

Jayaram, hari ............................................124, 572, 576

Jenkins, Gregory ........................................................499

Jensen, eric ...............................................................421

Jensen, Michael C. .....................................................247

Jeong, hwa yeon ...............................................155, 590

Jeong, in ho ......................................................155, 590

Jerome, Keith r. .........................................................765

Jiang, haiyan ............................................................756

Jiang, hua ................................................................202

Jiang, Jun ..................................................................315

Jiang, Monica............................................................710

Jiang, xun .................................................................647

Jimenez, Veronica .........................................................50

Jin, Aiwu ...................................................................126

Jin, lan .....................................................................737

Jing, Weiqing ............................................................668

Joffe, Maxwell .............................................................85

Jofra hernandez, raisa ................................................226

Johana, nuryanti ........................................................296

Johnson, bryon D. .......................................................668

Johnson, Calvin A. ......................................................156

Johnson, l. V. .............................................................445

Johnson, laura A. .......................................................510

Johnson, Mark ...........................................................173

Johnson, Mark r. ........................................................305

Johnson, r. Michael ....................................................554

Johnston, ian ..............................................................455

Johnston, ian C. D. .....................................................653

Johnston, Jennifer M. ...................................................128

Johnston, Julie ............................................................699

Joigneaux, Maeva ......................................................689

Jolly, Douglas J. ............................................61, 205, 673

Jones, Jon ..................................................................572

Jones, Michael ...........................................................360

Jones, Simon .............................................................364

Joshi, bharat ..............................................................214

Joshi, neha .........................................................75, 685

Joung, J. K. ................................................................731

Juillerat, Alexandre ..............................................651, 676

Julien, laura ...............................................................629

June, Carl .........................................................203, 638

June, Carl h. .....................................................510, 515

Jung, Moonjung .........................................................527

Jung, Soo-Jung ...........................................109, 113, 115

Kaadt, erik ................................................................589

Kabadi, Ami M. .........................................................525

Kabasakal, levent ......................................................152

Kai, hirofumi .............................................................122

Kailasan, Shweta .......................................................254

Kaiser, Andrew ..................................................455, 653

Kaler, Stephen G. ...............................................356, 615

Kalervo, Aino ............................................................194

Kaliberov, Sergey .......................................................519

Kalkanis, Steven n. ......................................................61

Kalled, Susan l. .........................................................724

Kaloyannidis, Panayotis ...............................................426

Kalurupalle, Swathi .....................................................146

Kamdem, Annie .........................................................231

Kamen, Amine ...................................................292, 555

Kami, Daisuke ...........................................................340

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AbSTrACT DireCTory AnD AuThor inDex — 85

Author IndexKaminsky, Stephen ................................................36, 309

Kaminsky, Stephen M. .........................365, 471, 474, 755

Kan, Shih-hsin ............................................364, 430, 615

Kan, yuet W. .............................................................332

Kaneda, yasufumi .......................................................149

Kanerva, Anna ..........................................................418

Kang, Andrea ............................................................684

Kang, Peter b. ...........................................................360

Kang, Seong Jae ................................................155, 590

Kao, Shih-Chu ...........................................143, 145, 260

Kaplan, Mark h. ........................................................508

Kaplitt, Michael .........................................................186

Kapp, Kerstin .............................................................740

Kapur, reuben ...........................................................508

Karda, rajvinder ........................................305, 437, 609

Karhinen, Minna ........................................................706

Karlsson, hannah .......................................................748

Karponi, Garyfalia .....................................................291

Karumuthil-Melethil, Subha .....................57, 363, 551, 725

Karuppiah, Muthumani ........................................264, 479

Kasahara, noriyuki.......................................61, 217, 673

Kasahara, yuko n. .............................................238, 378

Kasala, Dayananda ....................................109, 113, 115

Kasaraneni, nagarjun .................................................602

Kashentseva, elena .....................................................517

Kashiwazako, Mike ....................................................144

Kaspar, brian K. .........................................480, 497, 612

Kasuya, hideki ..........................................................660

Kataoka, Kazunori ......................................................593

Katayama, Takane ......................................................395

Katnani, husam A. .....................................................598

Kato, Koichi ..............................................................337

Katsuko, Sudo ...........................................................144

Kattenhorn, lisa ..........................................................227

Katz, nathan.............................................................346

Kaufman, Michael l. ...........................................230, 764

Kaur, balveen ............................................415, 520, 656

Kawai, Toshinao ........................................................224

Kawakami, eriko ........................................................413

Kawakami, hirotaka ...................................................414

Kawashima, nozomu .................................................705

Kay, Mark .................................................257, 289, 539

Kay, Mark A. ...............................48, 129, 253, 722, 737

Kays, Sarah-Katharina .................................................654

Kazhdan, natasha .....................................................713

Keaton, Amelia ..........................................................433

Keaton, Amelia A. ..............................................264, 479

Kebriaei, Partow ........................................................278

Kehoe, Daniel............................................................466

Kei Kishimoto, Takashi ...................................................77

Keiser, Megan S. .......................................................583

Kelleher, Michelle .........................................................33

Keller, Michael .............................................................75

Keller, Michael D. .......................................................685

Kemna, Christoph .......................................................506

Kenderian, Saad S. ....................................................273

Keravala, Annahita .....................................................607

Kerr, Alastair G. ...........................................................19

Keys, Megan e. .........................................................206

Khalil, Andrew ...........................................................335

Khan, Amir ........................................................479, 505

Khan, Amir S. ............................................................436

Khan, iram ....................................40, 44, 316, 683, 761

Khan, iram F. .............................................132, 398, 569

Khan, Javed ..............................................................649

Khan, Sabeena ..........................................................186

Khan, Taimoor ...........................................................159

Khandekar, neeta ......................................................298

Khawaja, hunain .......................................................153

Khebizi, Quentin ........................................................694

Kholodilov, nick ........................................................370

Khosla, Maninder .......................................................419

Khoury, hanna J. ........................................................276

Khurana, Tejvir ...........................................................726

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 86

Author IndexKielhack, bridgette ......................................................206

Kiem, hans-Peter ......................... 38, 44, 283, 284, 288, 429, 683, 758, 761, 767

Kikumori, Toyone ........................................................660

Kiley, James ...............................................................312

Kilfoil, Georgina ........................................................258

Kilpatrick, Julie ...........................................................247

Kim, Goo-young .........................................................165

Kim, hyun-Jung ..........................................................102

Kim, J. J. ...........................................................401, 436

Kim, Jaesung .............................................................110

Kim, Jung-Woong .......................................................266

Kim, Kwang-Soo ........................................................742

Kim, leo A. ...........................................................29, 80

Kim, Min Woo ..................................................155, 590

Kim, Miriam y. ...........................................................273

Kim, ok-sun ...............................................................102

Kim, Sung Wan .................................110, 113, 115, 177

Kim, Woori ...............................................................742

Kim, yoon-Sang .................................................166, 530

Kim, young-Jo ............................................................282

Kimura, Miyuki ...........................................................255

King, Jean .................................................................157

Kingman, Stephanie ....................................................347

Kinoh, hiroaki ...........................................................593

Kinoh, hiromi h. ........................................................238

Kissel, John T. ............................................................480

Kissick, Dominique ......................................................719

Kitagawa, Koichi ...............................................117, 395

Kitani, Tomoya ...........................................................340

Kitto, Kelley .................................................................86

Klann, Tyler S. ...........................................................525

Klatt, Denise ..............................................................282

Kleanthous, Marina .....................................................692

Klein, Pierre ...............................................................500

Kleinluetzum, Dina ......................................................654

Kleinstiver, benjamin P. ................................................731

Klempner, Mark .........................................................428

Kley, rudolf A. ...........................................................104

Klichinsky, Michael .....................................................273

Kloss, Christopher .......................................................638

Kneževiş, lea .............................................................404

Knight, Kristopher A. ...................................................456

Knipping, Friederike ....................................................121

Knop, David r. ....................................................88, 299

Knowles, helen ..........................................................452

Kobinger, Gary ..................................................436, 555

Kobinger, Gary P. .......................................................479

Kochenderfer, James n. .................................................74

Kodera, yasuhiro ........................................................660

Kodippili, Kasun .........................................................499

Koeberl, Dwight D. .......................................49, 311, 381

Kohara, hiroshi ..........................................................236

Kohlenberg, lucille......................................................575

Kohn, Donald b. ..........................32, 118, 230, 250, 764

Kok, rene .................................................................295

Kollu, nag ................................................................579

Kolovskaya, olga S. ...................................................151

Komarova, Mariia ......................................................151

Kompella, uday b. .....................................................489

Kondratov, oleksandr ..................................................549

Kong, D. ..................................................................445

Kong, Derek ..............................................................244

Kong, il-Keun ...............................................................16

Konhilas, John P. .........................................................172

Koniar, brenda l. ........................................................485

Konopka, Krystyna .....................................................680

Konstantinova, Pavlina .................................................546

Koo, Taeyoung ..................................................503, 634

Koodie, lisa ..............................................................413

Koonin, eugene V. ......................................................541

Koontz, Sherry .............................................................41

Koponen, Taina .........................................................461

Kornegay, Joe ......................85, 383, 388, 623, 627, 721

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 87

Author IndexKornegay, Joe n. .......................................................726

Korte, Martin ...............................................................59

Kortylewski, Marcin ....................................................261

Kosai, Ken-ichiro ................................................241, 414

Koski, Anniina ...........................................................418

Kostarelos, Kostas ...............................................384, 450

Kotin, robert M. .........................................................541

Kottke, Tim ............................................63, 64, 197, 749

Koutras, George ........................................................656

Kozarsky, Karen .............................25, 86, 346, 369, 468

Koziol, Colin .....................................................230, 764

Kozlowski, Miroslaw ...................................................273

Kozyak, benjamin W. .................................................383

Krat, Alexey V. ...................................................151, 151

Kraynyak, Kim ...................................................436, 440

Kraynyak, Kimberly .....................................................505

Kraynyak, Kimberly A. .................................................479

Krenciute, Giedre .........................................................76

Krhac levacic, Ana .....................................................493

Krishnan, A. ..............................................................445

Krishnan, Aparna .......................................................244

Krishnan, Suma ..........................................................509

Krishnankutty, ranjith ...................................................389

Krisky, David .............................................................184

Krouse, Allen .............................................................523

Kruspe, Sven .............................................................263

Krzysztoş, rafał ..........................................................599

Kubo, Shuji .......................................................211, 217

Kudchodkar, S. ..........................................................401

Kudchodkar, Sagar b. .................................................436

Kuhlmann, Anne-Sophie ...............................................429

Kühn, Jens .................................................................338

Kühnel, Mark P. ..................................................522, 766

Kumar, bhavna ..........................................................415

Kumar, Parameet ........................................................146

Kumar, Pawan ...........................................................415

Kumar, Sandeep ..........................................................79

Kumaran, r. ileng .......................................................563

Kume, Akihiro ....................................................223, 243

Kundu, nabanita .......................................................354

Kunkiel, Jessica ..........................................................522

Kuo, Caroline y. ........................................................118

Kuraoka, Mutsuki ........................................................238

Kurbanov, Suhrab .......................................................191

Kuroda, Masahiko ......................................................144

Kurtzberg, Joanne .........................................................93

Kuryk, lukasz .....................................408, 642, 659, 661

Kushibiki, Toshihiro......................................................673

Kutay, huban ............................................................737

Kwilas, Anna r. .........................................................214

Kwon, Jennifer ...........................................................525

Kwon, Jennifer b.........................................................626

Kwon, Joonhyun .........................................................165

Kyba, Michael ...........................................................312

Kyostio-Moore, Sirkka............................................92, 387

Kyrychenko, Viktoriia ...................................................730

labranche, Celia ........................................................440

labruna, Giuseppe .....................................................179

lächelt, ulrich ............................................................493

lachmann, nico ............................21, 35, 234, 522, 766

lagresle, Chantal .......................................................682

lagresle-Peyrou, Chantal ..............................................425

lagüe, Patrick ............................................................561

lai, Chooi-May ..........................................................248

laio, Sophia ..............................................................298

lam, Michael ..............................................................89

lam, Sharon ..............................................................763

lam, Stephen .............................................................641

lamana, María l. .......................................................753

lamouroux, lucille .......................................................538

lanciego, José l. ..................................................99, 370

landau, Dustin J. ........................................................311

landegger, lukas ........................................................269

landi, Daniel .............................................................392

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 88

Author Indexlandoni, elisa ............................................................752

langford-Smith, Alex....................................................364

langsdorf, Aliete ....................................................29, 80

lannfelt, lars ..............................................................567

lanthier, Stephane ......................................................292

laoharawee, Kanut .......................................86, 484, 485

lape, Janel ........................................................200, 394

lara-M, l. .................................................................453

lara-Martínez, luis A. ..................................................246

larcher, Thibaut ..........................................................503

larghero, Jerome ........................................................250

larocca, Chris ...........................................................413

larocca, Christopher ..........................................519, 639

larochelle, Andre .......................................................285

lata, Jaclyn .......................................................373, 605

latella, Maria C. ........................................................488

latournerie, Virginie ....................................................498

lattanzi, Annalisa ...............................................135, 559

latulippe, David r. .....................................................713

lau, Athena .................................................................32

laustsen, Anders .........................................................127

lavisse, Sonia ............................................................367

law, benedict ............................................................147

lawitz, eric ...............................................................258

lawlor, Michael .........................................................498

lawrence, Jeanne .......................................................315

lawson, Tristan ...........................................................466

le, S. Q. ..................................................................615

le, Steven Q. ............................................................430

leach, Sarah .............................................................316

leach, Stacey ............................................................499

leap, David J. ................................................................1

leaver, Timothy ..........................................................614

le bec, Christine ................................................498, 503

lebel, Francois ...........................................................509

lebkowski, Jane S. ......................................................276

leborgne, Christian .......................................................77

leboulch, Philippe ................................................10, 279

lebuhotel, Céline ........................................................397

lech, Patrycja J. .........................................................416

lecomte, emilie ..........................................................390

leddon, Jennifer .........................................................518

lederer, Carsten W. ....................................................692

le Duff, Johanne .........................................................697

lee, Albert ................................................................122

lee, baeckseung ................................................323, 398

lee, Chang i. ............................................................764

lee, Christina .............................................................576

lee, Christina S. .........................................................732

lee, Ciaran M. ..........................................................131

lee, Gary .........................................................133, 734

lee, Gary K. .............................................................641

lee, Gordon ..............................................................722

lee, hyunmi ..............................................................309

lee, Janet ....................................................................41

lee, Jessica ...............................................................505

lee, Jihyun .................................................................638

lee, John ...................................................................415

lee, Kristen ................................................................709

lee, lily ....................................................................313

lee, ni-Chung ...........................................................372

lee, Patrick C. ...........................................................347

lee, Sang r. ................................................................16

lee, Seung Koo .........................................................147

lee, Sunyeong ...........................................................102

lee, Won Jai .............................................................112

lee, Wonsig ..............................................................109

lee, yenarae .............................................................742

lee, young Mok .........................................................165

lee, young Sook ........................................................177

lee, youngmok...........................................................350

lee, yu-May ..............................................................372

leen, Ann M. ............................................190, 451, 751

lefrere, Francois .........................................................279

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AbSTrACT DireCTory AnD AuThor inDex — 89

Author Indexle Gall, Tony .............................................................597

léger, Adrien .............................................................390

legg, Charles r. ........................................................499

leggiero, eleonora .....................................................179

le Guiner, Caroline .....................................................503

lehalpere, Annaig .....................................................249

le heron, Anita ..........................................................634

lehky, Tanya ..............................................................715

lehtivarjo, Anna-Kaisa .................................................461

leitner, Theo ................................................................15

lemcke, heiko ...........................................................338

lemon, Athena ...........................................................687

lengler, Johannes ........................................................286

lenz, Jack .....................................................................2

león, xavier ........................................................50, 348

leonard, Joshua n. .....................................................496

leonardelli, lorena ......................................................531

leong, Kam W. .........................................................242

leopold, David ..........................................................265

lepetit, helene ...........................................................367

lepetit-Stoffaes, Jean-Pascal ..........................................603

lerner, Paul ................................................................616

leronni, Daniela .................................................114, 520

le roy, Florence G. ....................................................578

lesch, hanna ............................................................461

lesch, hanna P. .........................................................706

leslie, George J. ........................................................427

lesniak, Maciej ..........................................................208

leung, Chris P. .............................................................72

leung, hang .............................................................323

leung, lai-han ...........................................................647

leung, Wai-hang .......................................................277

levings, Padraic P. ......................................................723

levivier, Marc ............................................................610

levy, robert J. ............................................................174

lewis, Gretchen .........................................221, 229, 458

lewis, Sarah ......................................................379, 497

li, Allen ....................................................................170

li, Aofei ....................................................................520

li, baozheng ...............................................................91

li, Danning .................................58, 157, 349, 351, 366

li, Ding ....................................................................178

li, haitang ................................................................586

li, hong ...................................................................669

li, hongxia ...............................................................220

li, hui ......................................................................730

li, Jia ...........................................................9, 669, 733

li, Jianbin ..................................................................721

li, Jingjing .................................................................563

li, Juan .....................................................................721

li, Jun ...................................................11, 95, 215, 548

li, Kesang .................................................................202

li, lijing ....................................................................734

li, lingli ....................................................................357

li, linhong ...................................................................41

li, Mengxin ...............................................................570

li, Min .....................................................................220

li, Mu ......................................................................462

li, ni .......................................................................438

li, Patrick W. .............................................................133

li, Pingjuan ...............................................................566

li, Qianhong .............................................................178

li, Shaoyong ...............................................................81

li, Songtao ..................................................49, 311, 381

li, Tiansen .................................................................266

li, Weiying ...............................................................167

li, yan ......................................................109, 110, 113

li, yuping ..................................................................614

li, Zhihan ..................................................................211

li, Zonghai ................................................................202

liang, Feisi ................................................................271

liang, T. Jake .............................................................527

liang, xueyun ............................................................182

liao, Jiyuan ...............................................................236

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 90

Author Indexliao, xiao-hui ............................................................163

liau, Gene ................................................................138

lichty, brian ...............................................................713

lieber, André .........................................14, 17, 106, 757

liebowitz, Andrea ......................................................635

liebowitz, Andrea b. ..................................................446

liefhebber, Jolanda .....................................................546

liikanen, ilkka ............................................................418

likhite, Shibi ..............................................................480

lim, lawrence ........................................................29, 80

limberis, Maria P. ...............................................695, 699

limsirichai, Prajit .........................................................125

lin, C. M. .................................................................445

lin, hang ..................................................................524

lin, xin .....................................................................490

lin, youngshun ...........................................................527

linde, ian ......................................................................6

lindell, Per ................................................................258

linden, r. Michael ......................................................256

ling, Changquan ...........................................11, 95, 215

ling, Chen ............................. 10, 11, 91, 95, 196, 215, 220, 271, 371, 548, 723

liou, benjamin ...........................................................521

lipshutz, Gerald S. ...............................................45, 347

lisowski, leszek..........................................................737

liu, Chen ..................................................................393

liu, Daofeng ..............................................................396

liu, hua ....................................................................647

liu, Jie ......................................................................614

liu, Jilin .....................................................................101

liu, Jing ..........................................14, 15, 17, 106, 757

liu, li-Wen ................................................................149

liu, Pei-qi ..................................................................641

liu, Tongyao ..............................................................756

liu, xiao-bo .................................................................45

liu, xiaoming .............................................................182

liu, yu-Chen ..............................................................195

liu, yun ....................................................................185

llanga, llanga .............................................................93

llanga, Telmo ............................................................630

llewellyn, George n. ..................................................330

loader, Julie .................................................................33

loboa, elizabeth ........................................................442

lobry, Tatiana ..............................................................32

locafaro, Grazia ........................................................637

lock, Dominik ............................................................653

lock, Martin ..............................................................699

locke, Frederick l. ......................................................745

lockey, Timothy D. ......................................................100

lockhart, Paul J. ..........................................................374

lockwood, Sarah .........................................................26

logan, Grant .............................................................298

lombardo, Angelo ..37, 42, 130, 286, 558, 637, 729, 752

long, Chengzu ..........................................................730

long, Joseph ..............................................................118

loov, Camilla ............................................................567

loperfido, Mariana .....................................................628

lopez, P. M. ..............................................................445

lópez, ricardo ...........................................................753

lopez-Franco, esperenza ...............................................99

lopez-lemus, uriel A. ..................................................532

lopez-Manzaneda, Sergio ...........................................228

lópez-Martínez, M. ....................................................453

lopez-rodriguez, elena ...............................................522

loredo-Mendoza, l. ....................................................453

lorenz, birgit .............................................................137

loring, heather S. ......................................................183

lo Scrudato, Mirella ....................................................322

loskog, Angelica ........................................516, 662, 748

lou, Meng ................................................................575

louboutin, Jean-Pierre ..................................................346

loucari, Constantinos ..................................................692

louis, lumena ............................................................643

l.oulette, Dominique ...................................................565

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 91

Author Indexlourenço, Francisco ....................................................180

low, Walter C. ..............................................25, 86, 369

lowdell, Mark W. ......................................................452

lowe, emily ...............................................................287

lowes, linda .....................................................480, 497

lowther, Daniel e. .......................................................192

lu, Aiping .................................240, 303, 446, 449, 636

lu, An ......................................................................746

lu, Dao-Pei ................................................................195

lu, Jiamiao ................................................................289

lu, JunMei .................................................................371

lu, Qi ........................................................................98

lu, Taihe ...................................................................571

lu, xiangping ..............................................................85

lu, yi .........................................................81, 669, 739

lu, yuan ...................................................................723

lu, yuanqing .......................................................84, 632

ludewig, Susann ..........................................................59

lukason, Michael ...............................................301, 329

lumaquin, Dianne .......................................................118

lund, Troy C. .............................................................250

lundberg, Cecilia .......................................................610

luo, li ....................................................................7, 81

luo, yanping .............................................................515

luo, yicheng..............................................................252

luo, yonglun ..............................................................127

lupo, Mariangela ...............................................320, 736

luther, Samantha ........................................................466

lutrey, Molly ..............................................................218

lutzko, Carolyn ..........................................................444

lux, Christopher T. ......................................................562

lv, Guijin ..................................................................240

lyle, Meghan J. ..................................................434, 441

lyubarsky, Arkady .......................................................270

Ma, hong ........................................................7, 9, 570

Ma, hongming ..........................................................574

Ma, hsin-i .........................................................219, 678

Ma, Wenqin ...............................................................91

Ma, yupo .................................................................647

Ma, Zhijun ........................................................281, 571

Machado-Aranda, David .............................................173

Mack, David .............................................................498

Mackall, Crystal l. ......................................................192

Maclachlan, Timothy ..................................................399

Macleod, Daniel .......................................................200

Macleod, Daniel T. ....................................................394

MacQuivey, Matthew .................................................683

Madathilparambil, Suresh ............................................173

Maddalena, Andrea .....................................................28

Madhavan, Sarina .....................................................482

Maeder, Morgan l. ....................................................124

Maeshima, ruhina ......................................................588

Maffia, Veronica ........................................................724

Magalon, Jeremy .......................................................231

Magarkar, Aniket .......................................................642

Maggioni, luca ...........................................................50

Magnani, Alessandra ..........................................231, 754

Magnani, Zulma ........................................................752

Magno, Aaron l. .......................................................248

Magrin, elisa .....................................231, 279, 559, 754

Maguire, Albert .........................................189, 265, 270

Maguire, Albert M. ....................................................185

Maguire, Casey .................................................594, 616

Maguire, Casey A. .....................................................595

Maguire, Katie ..........................................................722

Mahimkar, rajeev .......................................................171

Maiti, Sourindra .........................................................278

Maiyo, Fiona C. ........................................................142

Maizonnier, natacha ..................................................377

Majdoul, Saliha .........................................................536

Majeti, ravindra ....................................................39, 43

Majowicz, Anna ................................300, 310, 700, 701

Makarevich, Pavel i. ...................................................448

Male, Frances ...........................................................754

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AbSTrACT DireCTory AnD AuThor inDex — 92

Author IndexMalech, harry ...................................................224, 527

Malech, harry l. ..................................................41, 557

Malerba, Alberto .......................................500, 503, 634

Malik, Alock ........................................85, 389, 627, 726

Malik, Punam ............................................................285

Maloneyhuss, Martha A. .............................................750

Maltais, Chantale .......................................................565

Mammen, Pradeep .....................................................730

Mamonkin, Maksim ..............................69, 210, 392, 762

Mandal, Pankaj K. .....................................................568

Mandapati, Savitri ......................................................299

Manfredi, Anna .........................................................724

Mangion, Mathias ......................................................341

Manko, Kelsey ...........................................................360

Manning-bog, Amy .............................................484, 485

Manoli, irini ......................................................353, 357

Mansilla-Soto, Jorge ....................................................274

Mañu, Maria M. ........................................................228

Manzano, raquel .......................................................741

Mao, Tin ..................................................143, 145, 260

Marco, eugenio .........................................................124

Marcó, Sara .............................................................348

Marcozzi-Pierce, Kathleen ............................................505

Marechal, Alan ..........................................................651

Maria Johnson, irudayam .....................................166, 686

Marigo, Valeria..........................................................488

Marin, Virna ................................................................37

Marino, Michael P. .............................................214, 566

Marino, Sandra ...........................................................97

Marinova, ekaterina ...................................................396

Markusic, David .........................................................688

Marlowe, Jennifer .......................................................399

Marolleau, béatrice ....................................................390

Marra, Jonathan D. .....................................................715

Marrocco, elena ................................................320, 736

Marshall, Kathleen ......................................................265

Marshall, Mike ............................................................57

Marsic, Damien .................................................549, 723

Marteau, Jean-brice ............................................464, 468

Martier, raygene ........................................................546

Martin, Aaron ............................................................200

Martin, Douglas .................................................361, 373

Martin, Douglas r. .....................................................605

Martin, lori ...............................................................399

Martin, Samia ...........................322, 498, 560, 690, 759

Martin, ulrich ............................................................234

Martin, William .........................................................377

Martindale, Jennifer ....................................................146

Martinez, Michele ......................................................764

Martinez-Calderón, Andrés r. .......................................246

Martin-Manso, Maria ..................................................685

Martino, Ashley T. ......................................................550

Martuza, robert l. ......................................................107

Marumoto, Tomotoshi ..................................................236

Marzulli, Marco .................................................111, 520

Maslow, Joel .............................................................436

Mason, Chris ............................................................452

Mason, Joe .................................................................19

Mason, nicola J. .......................................................750

Masso, F. ..................................................................453

Masso, Felipe ............................................................246

Mastaglio, Sara .........................................................752

Mastis, bryan ....................................................301, 621

Masuda, Chiaki .................................................238, 337

Masurier, Carole ........................................................503

Matalon, reuben ..........................................58, 351, 366

Matern, lindsay K. .........................................................6

Matsui, Akitsugu .........................................................593

Matsui, hideto ...........................................................161

Mattar, Citra ..............................................................296

Matus-nicodemos, rodrigo ..........................................272

Matuszewska, Kathy .....................................................66

Maurer, Anna ..............................................................34

Mavilio, Fulvio ...........135, 230, 498, 503, 559, 560, 690

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AbSTrACT DireCTory AnD AuThor inDex — 93

Author IndexMawatari-Furukawa, yuki .............................................217

Mazerolles, Fabienne ..................................................560

McAlister, Michelle .......................................................26

McCall, Angela l. ......................................................382

McCarron, Alexandra .................................................711

McCarthy, David ..........................................................23

McCarty, David .........................................................207

McCarty, Douglas M. .................................162, 352, 698

McCloskey, laura .........................................................33

McColly, Markus ........................................................497

McCormack, Sarah ....................................................685

McCormick, Sarah .......................................................75

McCoy, Jay ...............................................................435

McCray, Paul b. ........................................................438

McCreedy, bruce ...............................................200, 394

McCullough, K. Tyler...................................................620

Mc Dermott, John .......................................................364

Mcelvaney, noel G. .....................................................24

McGettigan, Shannon e. .............................................515

Mcintyre, Chantelle .....................................................711

Mcivor, r. S. .....................................................439, 485

Mcivor, r. Scott ......................................25, 86, 369, 484

Mckay, Tristan r. ................................................305, 437

McKenna, Mavis A.....................................................254

McKenna, robert .......................................................727

McKillop, Monty ........................................................703

McKintosh, Jenny ........................................................296

Mclaughlin, lauren .......................................................75

McMenamin, Deirdre ..................227, 481, 695, 696, 760

McMichael, Ashley .......................................................41

Mcnally, David J. ......................................................100

Mcnamara, James o. ................................................263

McPherson, leslie .......................................................299

McPhie, Donna l. .......................................................742

Mead, Andrew ............................................................85

Mead, Andy .............................................383, 388, 389

Mead, Andy F. ..........................................................726

Meadows, Aaron .......................................................162

Meadows, Aaron S. ...........................................352, 698

Meadows, eric ..........................................................379

Meagher, Michael ......................................................547

Meagher, Michael M. .................................................100

Medeiros, Amy A. ..............................................460, 710

Medin, Jeffrey A. ................................................407, 703

Medina, Maria Fe C. .................................................713

Medina-Jaszek, Angelica .......................................60, 189

Medina-Jaszek, Angelica C. .........................................699

Mefferd, Adam ..........................................................311

Meffre, eric ...............................................................280

Mehtala, Jonathan G. .................................................304

Mei, yanai ................................................................371

Mei, Zhuyong ............................................................645

Meineke, robert .........................................................766

Meister, Michael ........................................................254

Melcher, Alan ..............................................63, 197, 749

Melchiori, luca ..........................................................192

Melenhorst, Jos ..........................................................203

Meliani, Amine ....................................................77, 595

Melotti, Suzanna ................................................473, 709

Menale, Ciro.............................................................631

Mendell, Jerry ....................................................379, 622

Mendell, Jerry r. ................................................480, 497

Mendell, Josh ............................................................379

Mendoza, Daniel .......................................................205

Mendoza, Janess ...............................................428, 440

Meneghini, Vasco ......................................................559

Meng, hui ................................................................498

Meo, oriana .............................................................235

Mepani, rina ............................................................124

Merelli, ivan ......................................................529, 537

Merkel, Carrie e. .......................................................556

Merkert, Sylvia ...........................................................234

Merlin, Simone ..........................................................691

Merling, randall ........................................................527

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AbSTrACT DireCTory AnD AuThor inDex — 94

Author IndexMerling, randall K. .....................................................557

Metais, Jean-yves .......................................................564

Metelitsa, leonid ........................................................396

Metelitsa, leonid S. ....................................................191

Meunier, Cécile .........................................................327

Meunier, Sandrine ................................................83, 267

Mevel, Mathieu ...........................................................34

Meyer, Kathleen .................................................484, 684

Meyer, Kathrin ...........................................................480

Meyer, nancy l. ..........................................12, 293, 478

Mi, Tiejuan ...............................................................278

Miao, Carol h.......................................5, 434, 441, 492

Miao, John ................................................................509

Miao, linqing ............................................................271

Miari, reem ..............................................................598

Miatkowski, Maya......................................................709

Miccio, Annarita ........................................135, 230, 559

Miceli, M. Carrie .......................................................735

Michor, Franziska .......................................................314

Mier, heather A. A. ........................................... TeST0001

Mietzsch, Mario .................................................294, 549

Migliara, Alessandro...................................................729

Migliavacca, Maddalena ............................................226

Mikkelsen, Jacob G. ...........................................127, 533

Mikkelsen, Jacob Giehm ..............................................574

Milanesi, luciano .......................................................529

Milani, Michela .................................................286, 756

Milenova, ioanna ...............................................516, 662

Miletic, hrvoje ...........................................................475

Miller, edward b. .......................................................727

Miller, Jeffrey C. .........................................................641

Miller, natalie ...........................................................497

Miller, Paul e. ......................................................88, 299

Miller, robert J. ..........................................................148

Miller, Weston P. ........................................................250

Millikan, randall e. .......................................................72

Milling, Jesse .............................................................709

Milone, Michael ........................................................203

Milone, Michael C. ....................................................399

Milsom, Michael D. ....................................................282

Min, Seok-hong .........................................................620

Mineno, Junichi ..................................................193, 400

Mingozzi, Federico ..........49, 53, 77, 158, 498, 503, 595

Minopoli, renato........................................................187

Mintz, e. Kathy ............................................................75

Miranda, Carlos ........................................................480

Miskey, Csaba ............................................................73

Miskin, James ............................................................367

Mitchell, Marilyn ..................................................85, 726

Mitra, rajendra n. .............................................339, 342

Mitrophanous, Kyriacos .........................................33, 367

Mitsui, Kaoru .............................................................241

Miura, yoshiaki ..........................................................655

Miyahara, yoshihiro ....................................................193

Miyake, Koichi ..........................................................160

Miyake, noriko .........................................................160

Miyake, Takashi .........................................................580

Miyake, Tetsuo ...........................................................580

Miyamoto, Shohei ..............................................236, 409

Mizukami, hiroaki ......................................223, 243, 400

Mo, Fengfeng ............................................................220

Moal, Christelle .........................................................629

Mock, ulrike..............................................................455

Modestov, Andrey A. ..................................................151

Mohammed, Somala ..................................................751

Mohiti-Asli, Mahsa......................................................442

Molan, Amy ................................................................64

Molas, Maria ............................................................348

Monahan, Sean .........................................................170

Mondino, Anna .........................................................752

Monette, Sebastien .....................................................365

Monjezi, razieh ...........................................................73

Monpoux, Fabrice ......................................................279

Montefiori, David .......................................................440

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AbSTrACT DireCTory AnD AuThor inDex — 95

Author IndexMontier, Tristan...........................................................597

Montini, eugenio ............2, 212, 235, 529, 537, 674, 681

Montus, Marie ...........................................................503

Mookherjee, Suddhasil ................................................266

Moon, Chang yoon ....................................................115

Moon, Kyoung-Sik ......................................................102

Mooney, rachael .......................................................208

Moore, Constance .....................................................157

Moore, Marc ............................................................628

Moore-Clingenpeel, Melissa .........................................497

Moot, robert .....................................................207, 670

Morales, leon ....................................383, 388, 633, 726

Moraly h., yonit ........................................................598

Morbach, henner .......................................................280

Moreira, Dayson ........................................................261

Morel, laurence ...........................................................84

Morell, Montse ..........................................................313

Moreno, Daniel .........................................................164

Moreno, rafael ..................................................516, 662

Morgan, Jessica .........................................................270

Morgan, richard ........................................................747

Morici, Samantha .......................................................677

Morinaga, Takao .......................................................211

Morishita, ryuichi .......................................................580

Morita, Daisuke .........................................................705

Moritz, Thomas ..............................21, 35, 234, 522, 766

Morizono, hiroki ........................................................481

Mormin, Mirella .........................................................694

Morreale, Michael T. ..................................................566

Morris, elizabeth ................................................449, 635

Morris, elizabeth r. ....................................................446

Morris, James ............................................................301

Morris, John ..............................................................579

Morris, John C. ..........................................................413

Morris, Kevin V...........................................................532

Morrison, Christopher J. ...............................304, 460, 710

Morrison, Kyle ...........................................................492

Morrow, Matthew ......................................440, 505, 643

Morsy, Salma G. ........................................................543

Morton, Christopher ....................................................547

Moser, rachel ............................................................394

Moss, S. ...................................................................576

Motas, Sandra...........................................................348

Mottais, Angélique .....................................................597

Mou, haiwei .............................................................733

Moullec, Sophie .........................................................503

Moullier, Philippe ..................................34, 390, 503, 697

Moussy, Alice ............................................................693

Movaghar, Arezoo .....................................................575

Movsas, benjamin ......................................................514

Mrdalj, Jelena ............................................................475

Mu, Dakai ................................................................295

Mu, xiaodong ...........................................................635

Mucci, Adele ....................................................522, 766

Mudri, Sherri .............................................................572

Mueller, Christian ...................24, 90, 167, 183, 702, 738

Muench, robert .........................................................257

Muenzer, Joseph ........................................................352

Muharemagic, Darija ..................................................151

Muik, Alexander ................................................201, 654

Mukherjee, Malini ................................69, 210, 392, 762

Muller, Achim ............................................................706

Muller, Martin ....................................................408, 642

Müller, robert n. .......................................................610

Müller, ulrike C. ...........................................................59

Müller-Kuller, uta ........................................................234

Munding, elizabeth M. ................................................737

Munoz, Sergio .............................................................50

Munshi, Anupama ......................................................489

Munshi, nikhil .............................................................71

Muntoni, Francesco ....................................................624

Munye, Mustafa ...........................................................23

Murad, Joana ..............................................................71

Murad, John P. ...........................................................204

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 96

Author IndexMurakami, naoka ......................................................352

Muralidharan, ranganayaki .........................................489

Murdock, bryan .........................................................605

Muriel-ramos, Manuela ...............................................228

Murillo, oihana .........................................................164

Murlidharan, Giridhar .............................................30, 55

Muro, Andres ............................................................158

Murphy, Kelly ............................................................153

Murray, Margaret .......................................................509

Murrell, Julie ..............................................................466

Murrey, Darren...........................................................162

Musolino, Patricia l. ....................................................250

Mussolino, Claudio.............................................325, 692

Musunuru, Kiran .........................................................481

Muthumani, Kar .........................................401, 428, 440

Muthumani, Karrupiah .........................................433, 436

Muthumani, Karuppiah ................................................644

Muzyczka, nicholas .....................................................95

Mwizerwa, olive .......................................................598

Myers, Gary D. .........................................................646

Mykkänen, Jenni.........................................................461

na, youjin ........................................................112, 113

nagabhushan Kalburgi, Sahana ...................................362

nagai, yoko .............................................................236

nagamine, Claude ....................................................478

nagamine, Claude M. ...............................................293

nagaraju, Kanneboyina ..............................................584

nageswararao, Amulya .............................................197

naggie, Susanna .......................................................258

nagy, nadia ............................................................630

nair, nisha .......................................................120, 628

naka, Tetsuji .............................................................117

nakabayashi, Kazuhiko ..............................................224

nakai, hiroyuki .....................................12, 96, 255, 540

nakamura, Toru .........................................................673

nakanishi, yoichi .......................................................409

nakano, Atsushi ........................................................735

nakano, haruko ........................................................735

nakashima, hiroshi ....................................................667

nakazawa, yozo .......................................................705

nakazawa, yumiko ....................................................224

naldini, luigi ...................... 37, 42, 130, 235, 280, 286, 512, 558, 729, 752, 756

nalla, Arun K. ...........................................................687

nallanagulagari, Tejaswini reddy .................................657

nam, yein ................................................................384

nambiar, bindu ...........................................................92

nanni, Marina ............................................................53

naphade, Swati ........................................................336

nardo, David ............................................................632

nartker, brooke..........................................................666

närvänen, outi .........................................................706

nasimuzzaman, Md ...................................................285

naskar, Shovan ...........................................................53

nass, Shelley ..............................................................92

natarajan, Pachai ........................................................41

nathwani, Amit .........................................................296

nathwani, Amit C. .............................................100, 547

navai, Shoba ...........................................................392

naval, Mohamad ......................................................346

navale, lynn .....................................................287, 745

navarro, Susana ................................................228, 753

navarro-betancourt, J. .................................................453

navarro-betancourt, José r. ..........................................246

navarro-blasco, iñigo .................................................164

nayak, ramesh .........................................................444

neal, Tawny .............................................................607

nearing, Marie .........................................................584

neelapu, Sattva S. .....................................................745

negishi, yoichi ..........................................................337

negre, helene ............................................................71

negre, olivier ...................................................221, 229

neides, Mitchell G. ....................................................619

neil, Garry A. ...........................................................598

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 97

Author Indexneildez-nguyen, Thi My Anh .......................................693

neill, Thomas ............................................................417

nelson, Christopher ....................................................482

nelson, Christopher e. ................................................504

nelson, Stanley F. ......................................................735

nelvagal, hemanth r. ...................................................27

neman, Josh .............................................................204

nettelbeck, Dirk .................................................194, 410

neven, bénédicte ......................................................279

newCombe, hannah ...................................................41

newgreen, Donald F. .................................................374

ng, Joanne .......................................................305, 609

ng, Philip .........................................................106, 757

ng, robert ................................................................727

nguyen, nhu T. .........................................................731

nguyen, Quynh .........................................................380

nguyen, Thao T. T. .....................................................211

nguyen, Tuan h. .......................................................768

nichol, Geoff ............................................................684

nichols, Timothy ........................................................756

nicholson, Michael ....................................................200

nicholson, Michael G. ...............................138, 394, 579

nickolay, lauren ........................................................455

nicolas, Carmencita e. ...........................................39, 43

nienhuis, Arthur .........................................................686

nienhuis, Arthur W. ............................................166, 530

nierkens, Stefan ........................................................404

niibori, yosuke ..........................................................368

nikiforow, Sarah ..........................................................71

nikonov, Sergei .........................................................270

nikonova, elena ........................................................265

nishikawa, Tomoyuki ..................................................149

nishimoto, Kevin ........................................................276

nishio, nobuhiro .......................................................705

nitahara-Kasahara, yuko .............................................337

noble, Alex ..............................................................478

noda, Masahi ..........................................................161

noel, roxane ......................................................83, 267

norden, Diana M. .....................................................619

norelli, Margherita .....................................................512

nork, T. Michael ..................................................88, 299

novak, James S. ........................................................584

nowrouzi, Ali ............................................................134

nozza, Silvia ............................................................681

nunez-Cruz, Selene ....................................................203

nunoi, hiroyuki .........................................................224

nurminen, elisa M......................................................706

nwokafor, ugochi ......................................................209

nye, Chris ................................................................572

oakland, Mayumi ......................................................438

obama, yuki .............................................................414

o’brien, Timothy ........................................................346

oda, Tsugumi ............................................................395

odak, Ashlesha .........................................................274

odom, Guy l. ...................................................380, 501

o’Donnell, Patricia .......................................56, 284, 611

o’Geen, henriette ........................................................26

oh, eonju .................................................................113

oh, Jung-eun .............................................................177

ohmine, Ken .............................................................400

ohmori, Tsukasa ........................................................223

ohno, Shin-ichiro .......................................................144

oikawa, Keiki ...........................................................144

okada, hironori ........................................308, 337, 378

okada, Takashi .........................160, 238, 308, 337, 378

okamoto, Sachiko .....................................................193

okamura, Kohji .........................................................224

okano, Teruo ............................................................161

okolie, onyi .....................................................442, 528

oksanen, Minna ........................................................418

okuno, yusuke ...........................................................705

old, Matthew ............................................................415

oldham, robyn A. A. .................................................407

o’leary, Claire ..........................................................256

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 98

Author Indexo’leary, heather A. ....................................................508

olivares, Simon .........................................................278

oliveira, Giacomoq ...................................................752

oliver, James .............................................................287

oliviera, Amanda ......................................................421

olson, eric n. ...........................................................730

olson, erik r. ............................................................439

olzsko, Miles e. ............................................................1

omakobia, eugene ....................................................452

o’Malley, Mark .........................................................184

o’Meara, Tara ..........................................................250

ong, Sze hwee ........................................................374

ongulade, blessing ....................................................375

onodera, Masafumi ...........................................224, 308

onodi, Fanny ............................................................693

onojafe, ighovie F. .......................................................46

orange, Jordan .........................................................762

orange, Jordan S. ........................................69, 210, 392

orchard, Paul J. .........................................................250

orentas, rimas ..................................................648, 649

o’riordan, Catherine ..................................................621

o’riordan, Catherine r. ................................................92

orito, yuki ................................................................193

orkin, Stuart h. .................................................282, 314

ory, Daniel S. .............................................................56

osada, Kensuke ........................................................593

osborn, Mark............................................................121

osmon, Karlaina ................................................551, 725

osmon, Karlaina J. l. ..................................................363

ostertag, Derek ...........................................................61

ou, li ..............................................................484, 485

ou, Qinghui .............................................................178

ou, Wu ...................................................................214

ouedrani, Amani .......................................................225

ouellet, Dominique l. ..........................................321, 561

ouellet, eric ..............................................................614

ousterout, David G. ...................................................482

ozawa, Keiya ...........................................223, 243, 400

ozbas Turan, Suna .....................................................152

ozkan, naziye..........................................................152

ozog, Stosh ..................................................................4

ozono, Keiichi ..........................................................395

Pablo Perez-Pinera, Pablo ............................................311

Pacak, Christina .........................................................358

Pacak, Christina A. .............................................180, 360

Pacouret, Simon ...........................................................34

Paczesny, Sophie .......................................................508

Páez, A. ...................................................................453

Pagès, Jean-Christophe ................................................538

Pagovich, odelya ........................................................36

Pagovich, odelya e. ...........................................471, 755

Paker, Asif M. ............................................................250

Pal, Summanta ...........................................................261

Pala, Francesca .........................................................280

Palagano, eleonora ....................................................631

Palaschak, brett..........................................................688

Paldi, Andras .............................................................693

Palfi, Stéphane ...........................................................367

Palisoc, Maryknoll ......................................................527

Palmer, Donna ...........................................................757

Palmer, Glyn D. ..........................................................723

Palmer, lance ............................................................281

Palomar, nuria ..........................................................256

Pan, Dao ..................................................................521

Pan, Quintin ..............................................................415

Pan, Weihong ...........................................................101

Pan, xiaorong ............................................................202

Pan, xiufang ......................................................482, 499

Pan, Zhuo-hua .............................................................98

Panch, Sandhya r. .....................................................523

Pandey, Sachchida nand ............................................153

Pandha, hardev ...........................................................63

Pang, bifeng .............................................................269

Panjwani, M. Kazim ...................................................750

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 99

Author IndexPanneerselvam, Janani .................................................489

Papadimitriou, Stergios ................................................426

Papadopoulou, Anastasia ............................................426

Papanikolaou, eleni P. .................................................222

Papayannopoulou, Thalia ............................106, 526, 757

Parfyonova, yelena .....................................................447

Parfyonova, yelena V. .................................................448

Park, Anthony ............................................................204

Park, Anthony K. ........................................................406

Park, Daniel h. ..................................................264, 479

Park, Sang-Jin ............................................................102

Park, Tea Soon ..........................................................237

Park, yong Serk ..................................................155, 590

Park, young K. ...........................................................436

Parker, ernest T. ..................................................456, 728

Parker, nigel r. ..................................................461, 706

Parkhurst, Maria r. .....................................................272

Parrini, Martina ............................................................53

Parrish, Steve .....................................................461, 706

Parsons, David ...........................................................711

Parsons, Geoffrey b. ...................................................458

Partridge, Terence .......................................................623

Partridge, Terence A. ..................................................584

Parviainen, Suvi .................194, 405, 410, 418, 515, 652

Pasca, Sergiu P. .........................................................252

Paschal, Amber ..........................................................170

Paschon, David e. ......................................................641

Pasetto, Anna ....................................................272, 391

Passerini, laura ..........................................................681

Pastore, lucio ............................................................179

Pastre, David .............................................................536

Patel, Ami .........................264, 428, 433, 479, 644, 727

Patel, imari ................................................................587

Patel, J. .....................................................................445

Patel, Jennil ...............................................................244

Patel, Keyur ...............................................................258

Patel, Shabnum ..........................................................763

Pather, Sarshan ..........................................................237

Patsali, Petros ............................................................692

Pattanayak, Vikram .....................................................731

Paul, biswajit ...............................................................44

Paulk, nicole K. .........................................................722

Paulo, Sara ...............................................................461

Paulsen, bruna S. .......................................................568

Pavan, William J. .......................................................613

Pavani, Giulia....................................................135, 559

Payen, emmanuel .................................................10, 279

Pedersen, Kelly M. .....................................................369

Peebles, Donald .........................................................437

Peggs, Karl ...............................................................455

Pegues, Melissa A. .......................................................74

Pei, xuetao ................................................................233

Pekrun, Katja .....................................................539, 722

Pellegatta, Serena ......................................................507

Pellerin, luc ...............................................................327

Pellin, Danilo .............................................................531

Peltonen, Karita ..................................................408, 642

Pema, Monika ...................................................130, 703

Penaud-budloo, Magalie .......................................34, 302

Peng, Kah-Whye ................................................411, 654

Pennesi, Mark ..............................................................96

Pereira de Almeida, luís ..............................................582

Peretz, yoav ..............................................................192

Perez, Arianne ...........................................................287

Perez-Pinera, Pablo .....................................................136

Perini, ilaria ...............................................................628

Perkins, Molly ............................................................747

Perlingeiro, rita ..........................................................312

Perocheau, Dany ........................................................437

Perocheau, Dany P. .............................................305, 609

Perriard, Guillaume .....................................................327

Perrier, Anselme .........................................................327

Peshwa, Madhusudan V. ...............................................41

Pesonen, Sari ............................................................661

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 100

Author IndexPetek, Christopher ......................................................480

Peters, owen M. ........................................................738

Peterson, Christopher ..................................................761

Peterson, Christopher W. ...............................................38

Peterson, David ............................................................56

Peterson, Dorothy .......................................................712

Peterson, James J. .......................................................620

Petri, Karl ..................................................................121

Petrik, James J. .............................................................66

Petrov, Mihail ......................................85, 388, 633, 726

Petry, harald .....................310, 424, 546, 618, 700, 701

Pettitt, David ..............................................................718

Petzold, Andreas ..........................................................14

Pfeifer, rita ................................................................653

Phadke, Tanushree ......................................................576

Pham, Christina ..........................................................200

Phelan, Dean .............................................................374

Phillip, brian ..............................................................455

Phua, Terri .................................................................477

Picaud, Serge ............................................................268

Pichiorri, Flavia ..........................................................415

Pierciey, F. John ..........................................................229

Pierreux, Christophe e. ................................................163

Pietersz, Kimberly l. ....................................................424

Pietz, harlan l. ..........................................................765

Pignata, Antonella ......................................................392

Pignataro, Diego ........................................................370

Pignataro, Jose D. ........................................................99

Piguet, Françoise ..........................................................54

Pillay, Sirika .......................................................293, 478

Pineda, ricardo .........................................162, 352, 698

Pinz, Kevin G. ...........................................................647

Piovan, Claudia .................................................130, 703

Piras, bryan A. ...........................................................100

Pirazzoli, Valentina .............................................212, 674

Pirollo, Kathleen F. ........................................................72

Pittaluga, Stefania.......................................................527

Pivirotto, Philip ...........................................................621

Plantinga, Maud ........................................................404

Plovie, eva ............................................................29, 80

Plummer, emily ...........................................................428

Plyler, r. ...................................................................401

Podetz-Pedersen, Kelly .................................................485

Podetz-Pedersen, Kelly M. ..............................................25

Podetz-Petersen, Kelly M. .......................................86, 484

Poinsignon, Catherine .................................................694

Poirot, laurent ....................................................651, 676

Pokiniewski, Katie A. ...................................................545

Polacino, Patricia .........................................................38

Poletti, Valentina .................................................230, 690

Poli, Guido ...............................................................681

Pollen, Minna ............................................................706

Pomerantseva, irina.....................................................598

Pompeo Marinho, Felipe .............................................617

Pondarré, Corinne ..............................................231, 279

Ponzoni, Maurilio .......................................................752

Popplewell, linda .......................................................634

Porter, Caroline ..........................................................513

Porter, Forbes D. .........................................................613

Porteus, Matthew ........................................123, 128, 559

Porteus, Matthew h. .......................................39, 43, 563

Portnow, Jana ............................................................247

Posey, Avery D...........................................................510

Pouillot, Severine ........................................................468

Poulard, Karine ..........................................................498

Povlich, Samantha F. ...................................................304

Powers, John ...............................................................12

Powers, John M. ...........................................................96

Pozsgai, eric .............................................................622

Pradhan, isha ............................................................239

Pramov, Aleksandar ....................................................226

Prasad, Kavita ...........................................................259

Pravdo, Piers l. ..........................................................252

Prestil, ryan ...............................................................575

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 101

Author IndexPrew, Michelle S. .......................................................731

Priceman, Saul J. ................................................204, 406

Prickett, Todd D. .................................................272, 391

Prieto, Jesus ...............................................................164

Prieto, Jesús ...............................................................300

Prieve, Mary .............................................................170

Pringle, ian A.....................................................534, 743

Prior, Thomas W. ........................................................480

Prociuk, Maria .....................................................56, 611

Prosser, Megan ..........................................................206

Provasi, elena ............................................................752

Provenzale, James M. ...................................................56

Pruneau, Didier ..........................................................268

Przybilla, Michael J. ....................................................485

Przystal, Justyna ..........................................................672

Psatha, nikoleta .........................................................526

Puccio, hélène ............................................................54

Pule, Martin...............................................................455

Pulido, Jose .........................................................63, 749

Punreddy, Sandhya .....................................................466

Purdon, Terence .........................................................393

Puri, raj K. ........................................................214, 245

Purvis, Annie .............................................................249

Puschnik, Andreas ......................................................478

Puschnik, Andreas S. ...................................................293

Putta, Mallikarjuna ......................................................153

Pyle, April D. .............................................................735

Pyles, benjamin ............................................................26

Pytel, Kamila M. ........................................................534

Pythoud, Catherine .....................................................327

Qabar, Maher ...........................................................170

Qamou, rahaf ...........................................................687

Qasim, Waseem ........................................................455

Qi, ruirui ..................................................................220

Qiao, Chunping ........................................................721

Qin, Qiuyue ................................................................82

Qin, Wanru ............................................................7, 81

Qiu, Jianming ............................................................254

Qiu, Ting ..................................................................755

Qu, Jinrong ...............................................................459

Quach, lucy ......................................................309, 474

Querrey, Melissa ........................................................670

Quinn, Thomas ..........................................................313

Quinones-hinojosa, Alfredo ..........................................494

rabkin, Samuel D. ......................................................107

racine, Trina .............................................................479

radeke, robert ..................................................484, 485

rädler, Joachim o. .....................................................599

rae, Dustin T. .................................................................6

rafi, Mohammad .........................................................56

rafiq, Sarwish ...........................................................393

raghavendran, Krishnan ..............................................173

rahim, Ahad A. .................................................256, 305

rahman, Jahedur ........................................................256

raikar, Sunil ..............................................................670

raikar, Sunil S. ...........................................................207

raines, lydia .................................................................3

rajani, Karishma ............................................63, 64, 749

rakoczy, elizabeth P....................................................248

ralle, Martina ............................................................356

ralph, Scott G. ..........................................................367

ramachandran, Pavitra S. ............................................270

ramadan, Abdulraouf .................................................508

ramesh, rajagopal .....................................................489

ramos, Stephanie .......................................................264

ramsay, euan ............................................................614

ran, F. Ann ...............................................................482

rancourt, Derrick e. ....................................343, 344, 601

randle, Ashley ...........................................................361

randle, Ashley n. ......................................................605

rando, Thomas ..........................................................722

ranki, Tuuli ................................................................661

rantoglou, eirini .........................................................291

ranucci, Serena .........................................................226

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 102

Author Indexrao, Anitha ...............................................................734

rapoport, Aaron P. .....................................................192

rath, Tim ..................................................................134

ratner, nancy ............................................................666

rautsi, outi ...............................................................706

ravindra Kumar, Sripriya ..............................................252

rawlings, David .........................................................284

rawlings, David J. ..40, 44, 132, 316, 398, 569, 683, 761

raymond, Gerald V. ...................................................250

readler, James M. ......................................................176

rebar, edward J. ........................................................641

recchia, Alessandra ...................................................488

reddy, Timothy e. .......................................................525

reeves, emer P. ............................................................24

reeves, lilith ......................................................457, 707

refetoff, Samuel .........................................................163

reicherter, Amanda l. .................................................760

reider, Jake .................................................................71

reik, Andreas ....................................................641, 752

reimann, Christian ......................................................425

reinhart, bonnie .................................................111, 114

reinisch, Andreas ...................................................39, 43

reiser, Jakob ......................................................214, 566

renda, Mario ....................................................320, 736

renella, raffaele ................................................282, 314

ressler, Julie A. ...........................................................247

reul, Johanna ............................................................201

reuschel, emma l. ..............................................436, 479

reutenauer, laurence .....................................................54

rey, Maria ................................................................327

reza, Faisal ..............................................................720

r. Gill, Deborah .........................................................431

ribeil, Jean Antoine .....................................................231

ribeil, Jean-Antoine .....................................................279

ribera, Albert ............................................................348

ricciardelli, ida ..........................................................682

richard, isabelle.........................................................377

richardson, Celeste ....................................................399

richardson, Max ........................................................427

richfield, eric .............................................................259

richie, Christopher .....................................................218

richie, Christopher T. ..........................................156, 591

richter, Maximilian .............................................106, 757

rico, Alberto J. .....................................................99, 370

riecken, Kristoffer .......................................................475

riedl, Maureen ....................................................25, 369

riemann, Keith ...........................................................428

rieux-laucat, Frederic ..................................................560

rigby, Susan .............................................................466

rijal, Pramila .............................................................431

riley, brigit ................................................................684

riley, James ...............................................................427

rincón, Melvin ...........................................................120

río, Paula .........................................................558, 753

rittinghausen, Susanne ................................................476

ritz, Jerome .................................................................71

rivière, isabelle ..........................................................459

rivière, Julie .......................................................682, 759

rizvi, Tilat .................................................................666

rizzardi, Gian Paolo ...........................................130, 703

rizzo, Skylar .....................................................358, 360

rizzo, William b. .......................................................166

robbins, Joan M. .................................................61, 205

robbins, Paul F. ..................................................272, 391

roberge, Joannie ........................................................603

robert, Marc-André ............................................555, 708

robin, Schroll ............................................................444

robinson, Anastasia r. ................................................375

robinson, Paulette M. ...........................................88, 299

robinson-hamm, Jacqueline n. .............................317, 504

roboz, Gail J.............................................................397

roca, Carles .............................................................348

rocca, Celine ..............................................................32

rocca, Celine J. .................................................163, 350

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 103

Author Indexrocchi, Anna ...............................................................53

rocchi, Martina .........................................................752

rochat, Cylia.............................................................606

roda, elvira ........................................................99, 370

rodino-Klapac, louise .................................379, 480, 622

rodino-Klapac, louise r. ......................................386, 497

rodino-Klapaca, louise r. ............................................625

rodo, Jordi ..................................................................50

rodriguez, Cristina e. .................................................730

rodriguez, Juli ............................................................242

rodriguez, Tania ........................................................746

rodriguez-Aguirre, Maria .............................................205

rodríguez-Garcia, estefania .................................310, 700

rodríguez-rivera, e. ....................................................453

roederer, Jan-Peter ........................................................59

roelvink, Peter ...................................................145, 260

roesch, lauren .....................................................75, 685

rogers, Christopher ....................................................623

rogers, Geoffrey l. .......................................................78

rogozin, igor b. .........................................................541

rohde, Michelle .................................................484, 485

romagnoli, noemi .....................................................724

romain, Gabrielle ......................................................278

romano, Guillermo .......................................................44

romano ibarra, Guillermo S. ................................398, 761

román-rodriguez, Francisco J. ......................................558

romberg, neil ...........................................................280

romero, Zulema .........................................................118

rommelfanger, Diana ..................................................749

roncarolo, Maria Grazia ...............................51, 123, 637

ronzitti, Giuseppe ............................49, 53, 77, 158, 595

rook, Martha ............................................................466

rooney, Cliona M. .....................................190, 451, 705

rooney, Cliona r. ......................................................751

rosa, luca ..................................................................23

rosales, Cecilia .........................................................296

rosen, benjamin ..........................................................65

rosenberg, Jonathan .............................................36, 755

rosenberg, Jonathan b. ...............................365, 471, 474

rosenberg, Steven A. ..........................................272, 391

rosenblum, Shira ........................................388, 633, 726

rosenblum, Shira T......................................................552

rosewell Shaw, Amanda .............................................513

rossi, Alice ...............................................................226

rossi, Derrick J. ..........................................................568

rossi, John ................................................................671

rossi, John J. ..............................................................586

rossi, John M. ...................................................287, 745

rossi, Settimio ....................................................320, 736

rothe, Michael ..........................................................690

roudaut, Cécile .................................225, 425, 689, 754

rouhani, Farshid n. ......................................................24

round, Jeff ................................................................452

rousseau, Joël............................................139, 321, 561

routkevitch, Denis .......................................................494

rowland, Christopher r. ..............................................502

roy, Debashish ..........................................................170

roychoudhury, Pavitra .................................................765

rozenberg, Alejandra .................................................604

rozenberg, Alejandra J. ...............................................362

r. Townsend, Alain .....................................................431

ruan, Guoxiang .........................................................329

ruberte, Jesús ............................................................348

rudert, William A. ......................................................239

rudilosso, laura .................................................529, 681

ruella, Marco ............................................................273

ruggiero, eliana ................................................121, 232

ruiz, Autumn ...............................................................67

ruiz, Pedro ...............................................................753

russell, Angela.............................................................19

russell, David W. .......................................................285

russell, Shayla ...........................................................301

russell, Stephen ...........................................................67

russell, Stephen J. ...............................................411, 416

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 104

Author Indexrussell, Stephen r. ......................................................185

russo, Anthony r. .......................................................755

russo, Carlo ......................................................471, 755

russo, Carol ..............................................................474

russo, Fabio ........................................................51, 637

rusten, Myra .............................................................439

ryu, byoung ......................................................229, 458

ryvlin, Jessica ............................................................413

Saayman, Sheena ......................................................532

Sabatino, Denise e. ......................................................52

Sabatino, Marianna ...................................................650

Sacchetti, lucia ..........................................................179

Sachidanandam, ravi ...................................................70

Sadek, hanem ..........................................................425

Sadelain, Michel ................................................274, 459

Saga, yasushi ....................................................223, 400

Sagara, Miyako .........................................................409

Sagartz, John e. ...........................................................16

Saha, Dipongkor ........................................................405

Saha, Dipongor .........................................................194

Saha, Krishanu ..................................................335, 575

Sahel, José ................................................................268

Sahel, José A. ......................................................83, 267

Sahenk, Zarife ...........................................................497

Sahni, Jaya .......................................................398, 761

Saito, hiroki ......................................................117, 395

Sajib, Abdul Mohin ....................................................677

Sakata, yoichi ...........................................................223

Sakellari, ioanna ........................................................426

Salas, David .............................................300, 310, 700

Salem, bässem ..........................................................599

Sallese, Anthony ....................................................21, 35

Salmon, ruth .............................................................572

Salva, emine .............................................................152

Salzwedel, Amanda o. ..............................................639

Samara-Kuko, ermira ...........................................120, 628

Samaranch, lluis ........................................................618

Sampaolesi, Maurilio ..................................................628

Sampson, Carlos ........................................................712

Samulski, r. J. ....................................................251, 544

Samulski, richard Jude ..................................................93

Sánchez, rebeca .......................................................753

Sánchez, Victor ..........................................................348

Sánchez, xavier .........................................................348

Sanchez-Dominguez, rebeca .......................................228

Sandey, Maninder ......................................................677

Sandhu, Arbans .........................................................699

Sandler, laura ............................................................279

Sands, Mark S. ....................................................27, 430

Sanghez, Valentina .....................................................430

Sankar, raman...........................................................250

Sankar, uma .............................................................713

San Sebastian, Waldy ........................................618, 621

Santoni de Sio, Francesca ...........................................123

Santoni de Sio, Francesca r. ........................................681

Santostefano, Katherine e. ...........................................360

Santry, lisa A. ......................................................66, 422

Sanvito, Francesca .....................................................226

Sarathy, Apurva .........................................................150

Sarcar, Shilpita ..........................................................120

Sardesai, n. .............................................................401

Sardesai, niranjan .....264, 428, 435, 440, 479, 505, 643

Sardesai, niranjan y. .........................................433, 436

Sargent, r. G. ...........................................................332

Sarkissian, Aniee ........................................198, 204, 275

Sather, blythe D. .........................................................572

Sato, Shoki ...............................................................673

Sato-Dahlman, Mizuho ................................................655

Saudemont, Aurore .....................................................512

S’Aulis, Dana ............................................................166

Saulquin, xavier .........................................................697

Savoia, Dalila ...........................................................179

Savoldo, barbara ...............................................507, 748

Saxena, nivedita .........................................................43

Page 105: Abstract Directory, Wednesday May 4, 2016 · ivana Trapani, Alberto Auricchio 29 11:30 AM ... Giovanni Sitia, Angelo lombardo, luigi naldini 43 5:00 PM CRISPR/Cas9 and rAAV6-Mediated

ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 105

Author IndexSaxena, nivi ...............................................................39

Scala, Serena ............................................................531

Scalabrino, Miranda l. ................................................620

Scaramuzza, Samantha .......................................235, 280

Scaria, Abraham ........................................249, 301, 329

Scavullo, Cinzia .................................................130, 703

Schaffer, David V. .......................................119, 125, 617

Schambach, Axel ...............................234, 522, 690, 766

Schappet, James ........................................................148

Scharenberg, Andrew .................................................284

Scharenberg, Andrew M. ..............................40, 398, 761

Scharenberg, Andy .....................................316, 562, 683

Schefter, lauren ..........................................................283

Scheiflinger, Friedrich ..................................................286

Scheinberg, David ......................................................393

Schena, Francesca .....................................................631

Scherer, Tanja ............................................................355

Schestova, olga ........................................................273

Schickel, Jean-nicolas .................................................280

Schiffer, Joshua T. .......................................................765

Schiroli, Giulia .......................................37, 42, 558, 752

Schiwon, Maren ..............................................14, 15, 18

Schleef, Martin ....................73, 306, 355, 465, 592, 743

Schlegel, Andrea ...............................................169, 355

Schmeer, Marco ...........................................73, 306, 592

Schmidt, Manfred ...............121, 134, 232, 279, 296, 470

Schmidt, Manuel ........................................................740

Schmidtko, Achim .........................................................17

Schmitt, Tom ..............................................................288

Schneider, Andreas .....................................................476

Schneider, bernard .....................................................463

Schneider, bernard l. ..................................................606

Schneider, Dina .................................................648, 649

Schneider, irene .........................................................506

Schneider, Jay ............................................................730

Schneider, Joel S. .......................................................499

Schneider, Marc.........................................................254

Schneider, Mark ...........................................................82

Schneller, Jessica l. .....................................................359

Schnitzler, Aletta .........................................................466

Schnödt, Maria ..........................................................306

Scholl, Claudia ..........................................................134

Scholler, John .............................................................515

Schreeder, Daniel .......................................................273

Schreiber, Claire A. ................................8, 172, 297, 307

Schroff, Matthias ........................................................740

Schuelke, Matthew .....................................................197

Schultheis, Katherine ...........................................264, 435

Schulze, Érica A. ........................................................292

Schwake, Gerlinde .....................................................599

Schwartz, Steven D. ...................................................248

Scibetta, Alex ....................................................449, 635

Scibetta, Alex C. ................................................446, 636

Scott, Thomas ............................................................518

Seclen, eduardo ........................................................330

Segal, David J. ............................................................26

Segovia, José C. ................................................228, 753

Segura, Mercedes ..............................................473, 709

Seira, oscar .............................................................614

Sekine, ikuo ..............................................................211

Selby, Peter .........................................................63, 749

Sell, Jordan .......................................................413, 421

Selleck, Will ..............................................................572

Selleck, William .........................................................732

Sellers, Stephanie .......................................................523

Selvaraj, Sridhar ........................................................312

Semeraro, Michaela ...................................................279

Sen, Sabyasachi ........................................................354

Sena-esteves, Miguel ..................................361, 373, 605

Senís, elena ..............................................................470

Senthivel, nisha .........................................................364

Sentman, Charles .........................................................71

Sepulveda, Fernando ..................................................759

Serada, Satoshi .........................................................117

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 106

Author IndexSergi Sergi, lucia .......................................................631

Sermet-Gaudelus, isabelle ............................................175

Servais, laurent ..........................................................503

Seth, Prem ................................................................417

Seto, yasuyuki ............................................................412

Sette, Paola ...............................................................520

Sevilla, Julián .....................................................228, 753

Sewald, Katherina ......................................................476

Seye, Ababacar K. .....................................................536

Sgouramalli, eleni.......................................................291

Shad, y. ...................................................................445

Shah, Ami J. ..............................................................250

Shah, Divya ..............................................................505

Shah, Seema .....................................................221, 229

Shamir, esther ............................................................250

Shankar, Premlata .......................................................574

Shankar, ram ............................................306, 465, 592

Shapir, nir ................................................................598

Sharei, Armon ...........................................................573

Sharer, Wendy ..........................................138, 200, 579

Sharma, Alok K. ...........................................................88

Sharma, Anurag ...........................................................22

Sharma, Jay ........................................................32, 336

Sharma, Priyanka ...............................................176, 554

Sharma, Sandhya.........................................................69

Shaw, robert .....................................................461, 706

Shayakhmetov, Dmitry M. ..............................................13

Shedlock, Devon ........................................................479

Shelke, rajani ..............................................................34

Shell, richard ............................................................480

Shelton, John M. ........................................................730

Shen, Shen ...............................................................124

Shen, xuefei ..............................................................505

Sheng, nengquan ......................................................660

Sherman, Marika .......................................................745

Shern, Jack ................................................................649

Shestova, olga ..........................................................399

Shi, bizhi ..................................................................202

Shi, Victoria.................................................................74

Shiao, Kenneth T. .......................................................318

Shichinohe, Toshiaki ....................................................673

Shihabuddin, lamya S. ........................................301, 621

Shiku, hiroshi ............................................................193

Shim, Kevin G. ..............................................63, 64, 749

Shima, Midori ...........................................................161

Shimada, hideaki ......................................................211

Shimada, Takashi ...............................................160, 308

Shimizu, hiroyuki .......................................................409

Shirakawa, Toshiro .............................................117, 395

Shirley, Jamie l. ............................................................78

Shivak, David A. ........................................................133

Shivakumar, nishkala K. ..............................................385

Shivaprasad, nityashree .............................................649

Shoaebargh, Shabnam ...............................................713

Shpall, elizabeth ........................................................763

Shrager, Joseph ..........................................................722

Shvygin, Anna ...............................................................3

Shyng, Charles ............................................................27

Sibiril, yann ...............................................................597

Siddiqi, Tanya ...........................................................745

Siefker-radtke, Arlene ....................................................72

Sier-Ferreira, Valerie ....................................................300

Sigrist, nelly..............................................................560

Silva, Diogo ........................................................69, 210

Sim, Chou h. ............................................................374

Simmons, Tabatha r. ...........................................328, 624

Simões, Ana ..............................................................582

Simon, David K. .........................................................376

Simonelli, Francesca ...........................................320, 736

Simon Sola, Marcelo ..................................................158

Simpson, bryan P........................................................252

Singh, harjeet ...........................................................278

Singh, Kshitiz .............................................................120

Singh, Moganavelli ....................................................142

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 107

Author IndexSingh, Swati ..............................................132, 316, 569

Sinn, Patrick l. ...........................................................438

Sitia, Giovanni ............................................................42

Siurala, Mikko ...........................194, 405, 410, 515, 652

Sivyer, benjamin ...........................................................96

Six, emmanuelle .........................................231, 560, 754

Sizov, Katherine .........................................................150

Skala, luke ....................................................................3

Skillings, Jamey ............................................................61

Skipper, Kristian A. .....................................................533

Skor, Maxwell ............................................................124

Skuba, elizabeth ........................................................399

Slager, Anna .............................................................440

Slauson, Sarah ..........................................................458

Slivac, igor ...............................................................341

Sloan, Jennifer l. ..................................................46, 353

Slukvin, igor ..................................................................4

Smith, bruce ..............................................................677

Smith, bruce F. ...........................................................517

Smith, bryn M. ...........................................................492

Smith, C. i. edvard .....................................................581

Smith, Drake J. ...........................................................199

Smith, James K. S. ......................................................294

Smith, Jeff .........................................138, 200, 394, 579

Smith, Julianne ...........................................................397

Smith, leia M. .....................................................88, 299

Smith, nicholas J. C. ..................................................250

Smith, richard h. ...............................................285, 541

Smith, Trevor ..............................................264, 433, 435

Smurnyy, yegor ..........................................................229

Smyth, Christine M. ....................................................298

Smyth, Sarah .............................................................353

Snowden, Adam ..........................................................95

Snyder, Jessica ...........................................................498

Snyder, richard o. .....................................................390

Snyder, Scott .................................................................4

Snykers, Sarah .............................................................71

Sobacchi, Cristina ......................................................631

Sofia, Martina ...................................................320, 736

Soheili, Tayebeh S. .....................................................225

Soheili, Tayebeh-Shabi ........................................682, 759

Soiffer, robert ..............................................................71

Solanki, Manish .....................................................14, 17

Sommer, Karen ..........................................316, 398, 761

Sommer, Karen M. .......................................................40

Sondhi, Dolan .....................36, 309, 365, 471, 474, 755

Song, bo ..................................................................202

Song, Fei ..........................................................137, 202

Song, Shuxian ...........................................................492

Song, Sihong ......................................................84, 632

Song, yafeng ..............................85, 383, 388, 633, 726

Song, yumei ........................................................40, 316

Soni, Sandeep ...........................................................279

Sonnentag, Peter ..................................................88, 299

Sonntag, Kai C. .........................................................742

Sookdeo, Cathleen .......................................................92

Sookdeo, Cathy .........................................................387

Sorafine, Josh ............................................................473

Sorenson, Paul ...........................................................649

Sorrentino, brian P. .............................................281, 571

Sorrentino, nicolina C. ...............................................724

Soufleros, loudovikos ..................................................291

Souza, David ..............................................................92

Speck, Tobias ..............................................................68

Spector, David l. ........................................................563

Spector, laura P. .........................................................129

Spencer, David M. .....................................................746

Spencer, h. T. ............................................................728

Spencer, h. Trent ........................................207, 402, 456

Spencer, Jacqueline F. ...................................................16

Spencer, Jeffrey M. .....................................................141

Spencer, Melissa J. .....................................................735

Spencer, Trent ............................................................670

Spinelli, Antonello .......................................................752

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 108

Author IndexSpinler, Kyle ..............................................................511

Spinozzi, Giulio .............................2, 212, 529, 537, 674

Spivak, ekaterina .......................................................151

Sprague, leslee .........................................................666

Sproul, Scott ......................................................484, 485

Srinivasan, Madhuwanti ........................................69, 210

Srivastava, Akhil .........................................................489

Srivastava, Arun .................10, 11, 91, 95, 215, 220, 548

Stagg, Scott ..............................................................478

Stamatoyannopoulos, George ..........................................2

Stan, rodica .............................................................734

Stanek, lisa .......................................................301, 621

Stankov, Sylvia G. ......................................................382

Stankovic, Konstantina M. ............................................269

Starr, renate ......................................198, 204, 247, 275

Stedman, hansell .........................................................85

Stedman, hansell h. ...383, 388, 389, 552, 627, 633, 726

Stefanski, Jolanta ........................................................459

Steinhoff, Gustav ........................................................338

Stephanou, Coralea ...................................................692

Stephenson, Sarah e. M. .............................................374

Steppan, Diana .........................................................237

Stevano, Alessio .........................................................691

Stewart, Francis .....................................................14, 17

Stewart, Phoebe ...........................................................13

Steyer, benjamin ................................................335, 575

Stieger, Knut ..............................................................137

Stiff, Patrick ...............................................................276

Stity, Ghazal .............................................................699

Stocia, lorelei ............................................................605

Stockholm, Daniel ...............................................536, 693

Stoica, lorelei ............................................................373

Stoker, Andrew W. .....................................................588

Stone, Daniel .............................................................765

Stone, richard .............................................................71

Stricker, hans ............................................................514

Stripecke, renata ...............................................469, 476

Strollo, Sandra ...........................................................724

Su, Chuang ...............................................................507

Su, leonard .........................................................85, 726

Su, Qin ............................. 7, 9, 58, 81, 157, 349, 351, 366, 570, 669, 733, 739

Su, Shihuang .............................................................278

Suarez, Felipe ...........................................................279

Sucunza, Diego ...................................................99, 370

Suff, natalie ......................................................305, 437

Sugimoto, Mitsuhiko ....................................................161

Sugiyama, Takashi ......................................................420

Suh, Junghae ...............................................................89

Suhy, David ...............................143, 145, 258, 260, 500

Suksanpaisan, lukkana ................................................411

Sukumaran, Sujita ...............................................190, 751

Sullivan, Jennifer .........................................................301

Sumner-Jones, Stephanie G. .........................................534

Sun, beicheng ...........................................................668

Sun, Chengcao ..........................................................585

Sun, Chenglong .........................................................127

Sun, Giun .........................................................156, 591

Sun, Kai ...................................................................524

Sun, xun ...................................................................266

Sun, ying ..................................................................521

Sundarasetty, bala Sai .........................................469, 476

Sundback, Cathryn A. .................................................598

Sung, biin ...................................................................22

Surace, enrico M. ......................................................724

Surace, enrico Maria ..........................................320, 736

Surosky, richard .........................................................684

Surrallés, Jordi ............................................................753

Suryaprakash, Smruthi .................................................242

Susani, lucia .............................................................631

Sutton, bryan .............................................................630

Sutton, Kathryn ...........................................................402

Suwan, Keittisak .........................................................672

Suzuki, Akiko .............................................................214

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 109

Author IndexSuzuki, Masataka .......................................................513

Suzuki, Shingo ...................................................122, 332

Suzuki, Takuji .................................................21, 35, 522

Svinartchouk, Fedor ....................................................377

Swain, Gary .............................................................611

Swain, Gary P. ............................................................56

Swamy, Manjunath .....................................................574

Swaroop, Anand .......................................................266

Sweeney, Colin .........................................................527

Sweeney, Colin l. ......................................................557

Sylester, Albert ...........................................................505

Szarejko, John M. ......................................................646

Szoor, Arpad .............................................................645

Tabata, yasuhiko ........................................................340

Tabebordbar, Mohammadsharif ....................................483

Tachibana, Katsuhiko ..........................................238, 378

Tada, yuji .................................................................211

Tagalakis, Aristides .......................................................23

Tagalakis, Aristides D. .................................................588

Tagawa, Masatoshi ....................................................211

Taggart, Kathryn.........................................................180

Taheri, Maryam ...........................................................60

Tähtinen, Siri .....................................194, 405, 515, 652

Tai, Phillip .................................................................739

Tai, Phillip W. ....................................................290, 733

Tai, Phillip W. l. .........................................................7, 9

Taipale, Kristian .........................................................418

Tajima, Asako ............................................................239

Takagi, Kazuko ..........................................................420

Takahashi, Mayumi .....................................................586

Takahashi, Mizuna .....................................................673

Takahashi, yoshiyuki ....................................................705

Takanashi, Masakatsu .................................................144

Takano, hironobu .......................................................673

Takaoka, yuki ............................................................340

Takeda, Kaori M. .......................................................593

Takeda, Shin’ichi ................................238, 337, 378, 741

Takeda, Shuko ...................................................567, 616

Takesako, Kazuto .......................................................400

Takesako, Kazutoh ......................................................193

Takishima, yuto ..........................................................409

Talbot, Kevin .............................................................741

Talbot, leeAnne .........................................................399

Tam, lawrence .............................................................19

Tamaoka, Akira ..........................................................308

Tambussi, Giuseppe ....................................................681

Tan, eddie h. P. .........................................................675

Tan, Gewen ..............................................................660

Tan, lay-Geok ............................................................296

Tan, Mengqun .............................................................10

Tan, Ming Jie .............................................................216

Tan, nguan Soon .......................................216, 477, 675

Tan, Tiong Kit ............................................................431

Tan, yvonne y. W. ......................................................296

Tanaka, Maki ............................................................660

Tanaka, Miyuki ..........................................................705

Tang, Jonathan K. .......................................................347

Tang, Qiushi ..............................................................183

Tang, ruhang ............................................................721

Tang, yanqing ...........................................................174

Tang, ying ........................................................240, 524

Tang, yuxiao .............................................................220

Tangney, Mark ...........................................................437

Tani, Kenzaburo .................................................236, 409

Tanoue, Kiyonori ........................................................513

Tao, lihua .................................................................126

Tap, Willian D. ..........................................................192

Tarantal, Alice ............................................................472

Tarantal, Alice F. .........................................................764

Tatsumi, Koichiro ........................................................211

Tayapiwatsna, Chatchai ..............................................333

Taylor, Amanda ..........................................................605

Taylor, James r. ..........................................................614

Taylor, lauren .............................................................508

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 110

Author IndexTaylor, William .............................................................96

Tebbs, Susan .............................................................452

Tedesco, Francesco S. .................................................628

Tejvir Khurana, Tejvir .....................................................85

Teknos, Theodoros ......................................................415

Telaar, Maurice ..........................................................465

Tenderini, erika ......................................2, 212, 537, 674

Tenenbaum, liliane .....................................................610

Terada, nao .............................................................360

Terada, naohiro ........................................................180

Teruya, Takeshi ...........................................................400

Tetzlaff, Wolfram ........................................................614

Tewari, Manu ............................................................511

Thakore, Pratiksha i. ............................................385, 482

Tharmarajah, Grace ...................................................614

Theobald, narda .........................................................41

Thiel, William h. ........................................148, 263, 744

Thiriez, Claire ............................................................367

Thomas, Alexandra .....................................................263

Thomas, Amelia .........................................................458

Thomas, Anitha ..........................................................614

Thomas, Calvin A. ......................................................591

Thomas, Sandra .........................................................640

Thomasson, nitza ........................................83, 267, 268

Thompson, Jill ..............................................63, 197, 749

Thompson, Patrick ......................................363, 551, 725

Thöny, beat .......................................................169, 355

Thrasher, Adrian .........................................140, 455, 690

Thrasher, Adrian J. ......................................................250

Tierney, Kevin ............................................................479

Tiffany, Matthew ................................................253, 722

Tilakawardane, Dileshni.......................................204, 406

Timberlake, nina ...........................................................4

Tingey, Colleen ..........................................................436

Tipper, Christopher .....................................................221

Tirughana, revathiswari ...............................................209

Tisdale, John ..................................................................3

Tiwari, neha .............................................................712

Tockary, Theofilus A. ...................................................593

Todd, levi .................................................................612

Todeasa, Sophia ........................................................373

Todessa, Sophia ........................................................605

Todo, Tomoki .............................................412, 664, 665

Tolar, Jakub ...............................................................121

Tollefson, Ann e............................................................16

Tolosa, ezequiel .........................................................413

Tom, Susan .......................................................484, 485

Tomberlin, Ginger .......................................138, 200, 579

Tomono, Taro .............................................................308

Tonne, Jason ..............................................................543

Toppins, Caleigh ........................................................604

Torbett, bruce e. .............................................................4

Tordo, Julie ................................................................256

Torgerson, Troy ..........................................284, 316, 683

Torgerson, Troy r. .........................................................40

Toriello, elisabetta .......................................................187

Tornabene, Patrizia .......................................................28

Toro Cabrera, Gabriela ...............................................738

Torres, David .............................................................746

Toso, raffaella .............................................................52

Toth, Karoly .................................................................16

Tournaire, benoît ........................................................390

Touzot, Fabien ...........................225, 231, 279, 689, 754

Towne, Chris .............................................................186

Traggiai, elisabetta .....................................................631

Tran, eric ..................................................................391

Tran, hung ................................................................684

Tran, Karen .......................................................570, 733

Tran, Thuy An ............................................................369

Trang nguyen, Jennifer ..................................................26

Trapani, ivana .....................................................28, 187

Trapnell, bruce ...........................................................522

Trapnell, bruce C....................................................21, 35

Trebska-McGowan, Katarzyna ......................................272

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 111

Author IndexTremblay, Jacques P. ....................................139, 321, 561

Tremblay, Jacques-P. ....................................................565

Tretiakova, Anna ....................................60, 82, 189, 699

Trieu, nhu .................................................................623

Trimble, Connie ..........................................................505

Trimborn, lennart ........................................................137

Trobridge, Grant ........................................284, 288, 683

Trobridge, Grant D. ............................................1, 6, 687

Trollet, Capucine ........................................................500

Trucco, Massimo ........................................................239

Trump, lisa ................................................................444

Truong, brian .............................................................347

Truong, lynn ..............................................................641

Truong, nhi ...............................................................616

Trusolino, livio ...........................................................674

Trychta, Kathleen A. ....................................................218

Tsafa, effrosyni ...........................................................672

Tsai, Shengdar Q. ......................................................731

Tsang, Ashley ............................................................262

Tsaturyan, lusine.........................................................443

Tse, herman ..............................................................598

Tseng, yu-Shan ...........................................................541

Tsokolaeva, Zoya .......................................................447

Tsuchikawa, Takahiro ..................................................673

Tsuchiya, Motohiro .....................................................146

Tsugumine, Shu ..........................................................534

Tuan, rocky ...............................................................524

Tunc, ilker..................................................................527

Tung, Ching-hsuan .....................................................147

Turner, Caitlin .............................................138, 200, 579

Turner, Caitlin A. ........................................................394

Turunen, heikki ............................................................34

Turunen, heikki T. .......................................................467

Turunen, Janne J. .........................................................581

Tycko, Josh ................................................................317

Tylawsky, Daniel .........................................................207

uchibori, ryosuke .......................................223, 243, 400

uchida, hirokuni ........................................................593

uchida, naoya .............................................................3

uchida, nobuko ..........................................................39

uchida, Satoshi .........................................................593

uchiyama, Toru ..........................................................224

uckert, Wolfgang .......................................106, 506, 757

ugen, K. e. ...............................................................401

umansky, Viktor ..........................................................405

ungerechts, Guy ..................................................68, 423

ur, Sarah ....................................................................32

urabe, Masashi .........................................223, 243, 400

urak, Kevin T. ............................................................263

urak, ryan Z. ............................................................640

urbinati, Fabrizia .......................................................230

uretsky, Scott .......................................................83, 267

utikal, Jochen ............................................................405

utoh, rie ..................................................................161

uzel, Gulbu ..............................................................285

Vachey, Gabriel .........................................................327

Vaelk, Kristjan ............................................................475

Vago, luca ................................................................752

Vähä-Koskela, Markus .................................194, 405, 652

Valdmanis, Paul n. .....................................................737

Vales, Africa ........................................................99, 370

Valogne, yannick ........................................................651

Valonen, Piia .............................................................706

Valsecchi, Maria Grazia ..............................................468

Valton, Julien ......................................................651, 676

Vanbelzen, Daniel ........................................................85

Vanbelzen, Daniel h. ..................................................627

Vance, Melisa .....................................................93, 544

Vandamme, Celine .....................................................697

Van de Graaf, benjamin ..............................................471

Vandenberg, Tracy .....................................................277

Vandenberghe, luk .....................................................483

Vandenberghe, luk h. .......29, 34, 80, 269, 467, 594, 608

VandenDriessche, Thierry .............................120, 628, 756

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 112

Author IndexVan den haute, Chris ..................................................175

van der burg, Mirjam ..................................................280

van der loo, Johannes C. M. ........................................285

Van Der Smissen, Patrick ..............................................163

Vander Wall, roshana ................................................188

van Deventer, Sander J. ...............................................424

van haasteren, Joost .....................................................23

van lieshout, laura .............................................108, 181

van logtenstein, richard ..............................................546

Vanrell, lucia ...............................................................99

van Til, niek P. ..........................................................404

van Vloten, Jacob .......................................................181

van Vloten, Jacob P. ......................................66, 108, 422

van Wittenberghe, laetitia ...........................................390

Varadarajan, navin ....................................................278

Vasireddy, Vidyullatha .........................................553, 556

Vassilev, lotta .............................................................661

Vasudevan, Shobha ....................................................146

Vaucamps, nadège......................................................54

Vega-Crespo, Agustin ..................................................347

Vehar, Gordon ...........................................................171

Veinalde, rşta ............................................................423

Velazquez, Victoria .....................................................698

Venditti, Charles P. ..................46, 48, 168, 353, 359, 613

Venditti, Chuck ...........................................................357

Vendomele, Julie M. ....................................................694

Ventrella, Domenico ....................................................724

Vera, Juan F. ..............................................190, 451, 751

Vera, Moin u.............................................................430

Veres, Gabor .............................................221, 229, 458

Verhoeyen, els ...........................................................682

Verma, Anjali .............................................................466

Verma, Karan ............................................................237

Vermeer, louic ............................................................536

Vernant, Jean Paul .......................................................231

Veron, Philippe ...........................................................498

Vicenzi, elisa .............................................................681

Vidal, Patrice .............................................................158

Vidovic, Dragana .......................................................175

Viecelli, hiu Man .......................................................169

Viecelli, hiu-Man ........................................................355

Vieira, Mathieu ..........................................................578

Vignal, Catherine .................................................83, 267

Vignaud, Alban ..........................................390, 629, 690

Vila, laia ....................................................................50

Vila, Maria C. ...........................................................584

Vilches-Moure, Jose .....................................................722

Vile, richard ..........................................63, 64, 197, 749

Viley, Angelia ..............................................................41

Villa, Anna ..................................................42, 280, 631

Villarreal, Daniel .........................................................643

Vincent, Karen .............................................................92

Vine, David J. ............................................................356

Vink, Conrad A. .........................................................609

Virag, Tamas .............................................................541

Visel, Meike ..............................................................617

Vitale, Andrea ...........................................................659

Vitaliano, Gordana .....................................................742

Vite, Charles h. ...................................................56, 611

Vives-Corrons, Joan l. ..................................................228

Vogelbaum, Michael .....................................................61

Voit, Thomas ..............................................................503

Volpin, Monica ..............................................................2

Vondrak, Kristen .........................................................430

Vong, Queenie ..........................................................562

von Kalle, Christof ........68, 121, 134, 232, 279, 423, 470

Voronina, natalia .......................................................338

Voytas, Daniel............................................................312

Vulchanova, lucy ............................................25, 86, 369

Wada, Masayuki .......................................................647

Waddington, Simon ...................................................256

Waddington, Simon n. ...............................305, 437, 609

Wade-Martins, richard .................................................19

Wadsworth, Samuel ...................................227, 249, 760

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AbSTrACT DireCTory AnD AuThor inDex — 113

Author IndexWadsworth, Samuel C. ...............................304, 460, 710

Wagers, Amy ............................................................483

Wagner, ernst ............................................................493

Wagner, Jamie r. .......................................................247

Wagner, Thor A. ........................................................761

Wahli, Walter A. .......................................................675

Wahlin, Karl J. ...........................................................494

Waite, Melissa ..........................................................715

Wakimoto, hiroaki .....................................................107

Waldheim, Matt .........................................................170

Walia, Jagdeep .................................................363, 551

Walia, Jagdeep S. .....................................................725

Waligura, Kathryn ......................................................162

Walker, Christopher M. ...............................................698

Wallace, lindsay M. ..........................................386, 625

Wang, bing ......................................................240, 524

Wang, blake y. .................................................570, 733

Wang, Cathy x. ........................................................330

Wang, Chi-hsiung .....................................................417

Wang, Chunmei ......................................................7, 81

Wang, Cindy ............................................................357

Wang, Dan ......................................................570, 733

Wang, Dongrui .........................................................198

Wang, Gang ....................................................331, 542

Wang, Gensheng ......................................................472

Wang, hailong ...........................................................14

Wang, hua ..............................................................417

Wang, Jianbin .....................................38, 133, 427, 734

Wang, li ..................................................................608

Wang, li-juan ............................................................678

Wang, lili .................................................227, 481, 760

Wang, lisheng ..........................................................417

Wang, Pei-rong .........................................................345

Wang, Pin-yi .....................................................518, 666

Wang, Qiang ...................................................695, 760

Wang, Qizhao ..........................................................545

Wang, xiaohong .......................................................521

Wang, xin ................................................................220

Wang, xiuli ...............................................................640

Wang, xiuyan ...........................................................459

Wang, xuefeng .....................................................5, 434

Wang, xujing ............................................................527

Wang, yang .............................................................428

Wang, yong-Dong .............................................281, 571

Wang, yu Tian ..........................................................614

Wang, yupeng ..................................................132, 569

Wang, Zhigang .........................................................660

Wang, Zhimin ...........................................................334

Wang, Zhongde ..........................................................16

Warischalk, Jayme K. ..................................................251

Warren, James ...................................................473, 709

Wasala, nalinda .......................................................499

Wasielewska, Teresa ..................................................459

Wasowicz, Marguerite W. ..........................................534

Wassmer, Sarah.............................29, 80, 269, 594, 608

Watanabe, Keisuke ....................................................515

Watanabe, norihiro ...................................190, 513, 751

Watanabe, Toshiaki ....................................................664

Watson-levings, rachael S. ..........................................723

Watts, Colin ..............................................................367

Weber, leslie .............................................................559

Wechsler, Thomas ..............................................484, 485

Wechsler-reya, robert ................................................443

Wechuck, James ........................................................184

Weed, Peter ..............................................................375

Wegner, Julia ..............................................................73

Wehbe, Mohammed ..................................................151

Wei, Jun ...................................................................182

Wei, Qiang ..............................................................669

Wei, yuquan ...........................................................7, 81

Wein, nicolas ...................................................328, 624

Weinberg, Kenneth i. ..............................................39, 43

Weinberg, Marc S. ....................................................532

Weiner, David ...................................428, 440, 505, 643

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ASGCT 19Th AnnuAl MeeTinG MAy 4-7, 2016WAShinGTon, DC

AbSTrACT DireCTory AnD AuThor inDex — 114

Author IndexWeiner, David b. ...............264, 401, 433, 436, 479, 644

Weinstein, David A. ...................................................350

Weisell, Janne ...........................................................706

Weiss, Alexander .......................................................656

Welch, Dan ..............................................................451

Welstead, Grant ........................................................572

Wendel, Dominik .......................................................493

Weng, lihong ....................................................247, 275

Wengel, Jesper ..........................................................581

Wenger, David A. ........................................................56

Wenthe, Jessica .................................................516, 748

Wertz, Julie S. ...........................................................148

Westerman, Michael ..................................................541

Wetzel, Keith J. ..........................................................394

Weyer, Sascha W. .......................................................59

Whaley, Adele S........................................................755

White, John .......................................................227, 481

White, Margaret e. ....................................................723

Whitley, Chester b. .....................................439, 484, 485

Widrick, Jeffrey ..........................................................483

Wiekhorst, Frank ........................................................338

Wielechowski, erik .............................................189, 699

Wielgosz, Matthew ....................................................530

Wiezorek, Jeff ...........................................................745

Wikstrom, Kristina i. ....................................................748

Wilcox, David A. .......................................................668

Wild, Andre ..............................................................614

Wilkening, Stefan .......................................................470

Wilkens, Alec b. ....................................................39, 43

Wilkinson, Fiona ........................................................364

Willard, Vincent P. ......................................................318

Willett, Keirnan ..........................................................270

Williams, Daniel D. ....................................................192

Williams, David A. .....................................250, 282, 314

Williams, elizabeth ...............................................75, 685

Williams, ian M. ........................................................613

Williams, Jim .............................................................712

Williams, Kirsten ........................................................685

Williams, lee ............................................................600

Williams, Theodore ....................................................687

Wilmes, Gwen ..........................................................227

Wilmes, Gwendolyn M. ..............................................304

Wilson, Chris ............................................................317

Wilson, David r. ........................................................494

Wilson, Gabrielle r. ...................................................374

Wilson, James M. ................. 24, 60, 82, 189, 227, 346, 481, 695, 696, 699, 760

Wilson, John .............................................................451

Wilson, Matthew h. ...................................................705

Wilson, Sophie ..........................................................452

Wimalawansa, Sunishka M. ........................................554

Winkler, Thomas ........................................................527

Winsauer, Peter J. .......................................................375

Winter, brandy ..........................................................180

Wires, emily S. ..........................................................218

Wirth, edward ...........................................................276

Wise, Megan ....................................................440, 643

Wise, Megan C. .......................................264, 433, 644

Wittig, burghardt .......................................................740

Witting, Scott ....................................................457, 707

Witzmann, Frank ........................................................457

Wohlfahrt, Martin .......................................................288

Wold, William ............................................................16

Wolf, Justine ..............................................................536

Wolfe, Scot ...............................................................326

Won, haejung ..........................................................261

Wong, Chinglam W. .................................................640

Wong, D. .................................................................445

Wood, Matthew ........................................................741

Wood, Michael S. .....................................................191

Woodard, Kenton T. ...................................................544

Woodley, evan ..................................................363, 725

Woodley, evan J. r. ....................................................551

Woods, Wendy S. .....................................................136

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AbSTrACT DireCTory AnD AuThor inDex — 115

Author IndexWootton, Sarah K. ...............................66, 108, 181, 422

Wopat, Mitchell J. ......................................................550

Worgall, Stefan ...........................................................22

Wosen, Jonathan .......................................................478

Wosen, Jonathan e. ....................................................293

Wright, Kaylor ...........................................................763

Wright, Sarah ...................................................204, 406

Wu, Chuanfeng .........................................................523

Wu, Darong ..............................................................648

Wu, David M. .......................................................29, 80

Wu, haoquan ...........................................................574

Wu, Jerry ..................................................................319

Wu, lusha ................................................................220

Wu, rl .....................................................................445

Wu, Tong .................................................................195

Wu, Wei-li ...............................................................252

Wu, Wenzhu ..............................................................22

Wu, xiaolin .................................................................41

Wu, yinghua .............................................................754

Wu, Zhijian...............................................................266

Wu, Zhiwen ..............................................................660

Wu, Zhongren ...........................................................545

Wurm, Florian M. .......................................................463

Wyles, David l. .........................................................258

Wynn, rob ...............................................................364

Wynne, Graham ..........................................................19

xiao, ru..........................................29, 34, 80, 483, 608

xiao, Weidong ..........................................................545

xiao, xiao .................................................................721

xiao, yuanyuan ........................................................7, 81

xie, Fei .....................................................................332

xie, Jun .........................................................................9

xie, Qing ............................................................12, 478

xie, Wei ...................................................................178

xiong, ying .......................................................648, 649

xu, Chenyu ...............................................................481

xu, Gaungchao ...........................................................81

xu, Guangchao .....................................................7, 570

xu, hongxia ................................58, 157, 349, 351, 366

xu, Jing ...............................................................20, 577

xu, li ..................................................................20, 577

xu, ruian ..................................................................545

xu, Weidong .............................................................417

xu, xin .....................................................................191

xu, xun .....................................................................739

xue, Jingwen .............................................................524

xue, Wen .................................................................733

yabe, idalia ..............................................................523

yada, ravi Chandra ...................................................527

yagita, Mari ..............................................................224

yagiz, Kader .............................................................205

yajima, Shoh .............................................................412

yakaboski, elizabeth ...................................................647

yamagami, Momona ....................................................89

yamamoto, Masato ............................421, 519, 639, 655

yamamoto, Motoko ....................................................160

yan, Jian ...........................264, 433, 440, 479, 505, 643

yan, Winston .....................................................482, 483

yan, xiaokang ...........................................................218

yan, Ziying ...............................................................254

yañez, rosa ..............................................................753

yang, bin ..................................................................252

yang, hsiao T. ...........................................................499

yang, Julia ................................................................562

yang, li ......................................................................81

yang, liu...................................................................271

yang, Su J. ................................................................330

yang, yang ...............................................................481

yang, yuefeng ...........................................................417

yanik, Mert ...............................................................137

yankee, Thomas M. ....................................................646

yannaki, evangelia .............................................291, 426

yao, Gary.................................................................499

yao, Jinjing................................................................371

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AbSTrACT DireCTory AnD AuThor inDex — 116

Author Indexyao, Ming ................................................................668

yao, xiaorui ..............................................................399

yasukawa, Masaki .....................................................193

ye, Guo-jie ..........................................................88, 299

ye, Guojie ................................................................101

ye, lin ......................................................................332

yeganeh Kazemi, nazanin ............................................64

yerden, randy ...........................................................714

yezzi, Michael ...........................................................122

yi, lan ......................................................................262

yilmazer, Acelya ................................................384, 450

yin, hao ...................................................................487

yin, Weifeng .............................................................524

yin, Zifei .....................................................10, 215, 548

ying, baoling ..............................................................16

ylä-herttuala, Seppo ...........................................461, 706

yliperttula, Marjo .......................................................659

yoder, Mervin ..............................................................10

yohe, Marielle ...........................................................649

yokota, Toshifumi ........................................................623

yoo, Ji young .............................................................658

yoon, A-rum ......................................................109, 116

yoon, hyun Joo ..........................................................454

yoon, Sorah ..............................................................671

yoshimoto, yu ............................................................340

you, young Myoung............................................155, 590

younan, Patrick ............................................................44

younesi, Shahab ........................................................735

young, Courtney S. .....................................................735

young, Gloria ............................................................265

young, regina ...........................................................399

ystaas, lars r. ...........................................................475

yu, Abigail ..................................................................26

yu, hongwei .............................................346, 481, 695

yu, Jun-Ge .................................................................415

yu, Kyung-rok ............................................................564

yu, Wenhan ..............................................................266

yu, xin .............................................................262, 462

yuan, ye .....................................................................84

yue, yongping ...........................................................499

yumul, roma .................................................14, 17, 757

yun, Chae-ok ....109, 110, 112, 113, 115, 116, 177, 417

yurkoski, Jacqueline ....................................................624

Zabaleta, nerea ........................................300, 310, 700

Zack, Don J. ..............................................................494

Zaia, John A. .............................................................734

Zaidi, Shane ...............................................................63

Zakas, Philip M. .........................................................456

Zakas, Phillip M. ........................................................728

Zamay, Anna S. .........................................................151

Zamay, Galina S........................................................151

Zamay, Tatiana n. .....................................................151

Zambidis, elias ..........................................................237

Zamore, Phillip D. ..............................................570, 739

Zanella, eugenia ........................................................674

Zanolini, Diego ..........................................................691

Zaraspe, Kimberly ......................................................352

Zein, Wadih .............................................................715

Zemmour, David .........................................................560

Zerfas, Pat ................................................................357

Zerfas, Patricia M. ......................................................356

Zeumer, leilani .............................................................84

Zhai, yubo ................................................................275

Zhan, hong ..............................................................455

Zhan, Jun ..................................................................686

Zhang, Daniel ...............................11, 95, 196, 215, 548

Zhang, Dong C. ........................................................374

Zhang, Fang .............................................................690

Zhang, Feijie .....................................................289, 737

Zhang, Feng .....................................................482, 483

Zhang, hui ...............................................................265

Zhang, Jian-Ping .........................................................195

Zhang, Jilu ................................................................508

Zhang, Keqing ..........................................................499

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AbSTrACT DireCTory AnD AuThor inDex — 117

Author IndexZhang, Kermit ............................................................260

Zhang, lei ........................................................641, 684

Zhang, lianwen .........................................................411

Zhang, ling ...............................................................278

Zhang, Mingdi ..........................................................520

Zhang, Wen .............................................................229

Zhang, Wenli ................................................14, 17, 105

Zhang, xiaoliu ...................................................126, 141

Zhang, xin-qiao ...........................................................72

Zhang, yajun .............................................................218

Zhang, yue .......................................................722, 737

Zhang, Zhengyu ........................................................371

Zhao, lichao .............................................................489

Zhao, lixia ..........................................................20, 577

Zhao, Ming ..............................................................411

Zhao, na .................................................................331

Zhao, Qianru ............................................................371

Zhao, xingli ..............................................................261

Zhao, yan D. .............................................................489

Zheng, hui ...............................................................721

Zheng, Min .......................................................339, 342

Zheng, Qiang ...........................................................739

Zhong, boya .............................................................211

Zhou, huayu .............................................................730

Zhou, Junfang ............................................................547

Zhou, Qi ..................................................................506

Zhou, Sheng .....................................................281, 571

Zhou, yan .................................................................127

Zhu, D. ....................................................................445

Zhu, Kexian ...............................................................483

Zhu, lihua J. ..............................................................326

Zhu, yaxi ..................................................................543

Zhu, yongzhao ..........................................................182

Ziegler, Mona-larissa ..................................................541

Zimmer, Virginie .........................................................327

Zimmerlin, ludovic ......................................................237

Zinn, eric ..................................................................467

Znidar, Katarina .........................................................495

Zolotukhin, irene .........................................................688

Zolotukhin, Sergei.......................................159, 549, 723

Zonari, erika .............................................................235

Zou, Jizhong ..............................................527, 557, 566

Zou, yaqun ...............................................................150

Zubkova, ekaterina .....................................................447

Zubkova, olga ..........................................................151

Zuliani, Vincent ..........................................................468