![Page 1: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/1.jpg)
Cicle de Conferències en Malalties Rares, VHIR, Barcelona, 16 Des 2014
Rare diseases as platform to develop
novel therapeutic strategies: the
example of Fanconi anemia
Dr. Jordi Surrallés
Catedràtic de Genética
Professor ICREA-Acadèmia
Universitat Autònoma de Barcelona
Genome Instability Group: http://gig.uab.cat
![Page 2: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/2.jpg)
Fanconi anemia
![Page 3: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/3.jpg)
http://www.malaltiaamagada.com
A tale: Arnau has a hidden disease
![Page 4: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/4.jpg)
Garaycoechea and Patel (2014) Blood 123:26-34
Bone marrow failure in Fanconi anemia
![Page 5: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/5.jpg)
Disease evolution in Fanconi Anemia
Kutler et al., Blood, 2003
![Page 6: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/6.jpg)
Chromosome fragility in Fanconi anemia
![Page 7: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/7.jpg)
C. Group Gene Reference
FA-A FANCA Lo Ten Foe et al. Nature Genetics 1996
FABC Consortium. Nature Genetics 1996
FA-B FANCB Meetei et al Nature Genetics 2004
FA-C FANCC Strathdee et al. Nature, 1992
FA-D1 FANCD1/BRCA2 Howlett et al. Science, 2002
FA-D2 FANCD2 Timmers et al. Mol Cell, 2001
FA-E FANCE De Winter et al. Am. J. Hum Genet 2000
FA-F FANCF De Winter et al. Nature Genet. 2000
FA-G FANCG De Winter et al. Nature Genet. 1998
FA-I FANCI Smogorzewska et al. Cell 2007
FA-J FANCJ/BRIP1 Levran et al. Nature Genet. 2005; Levitus et al. Nature Genet. 2005;
FA-L FANCL Ruhikanta et al. Nature Genet. 2003
FA-M FANCM Meetei et al Nature Genetics 2005
FA-N FANCN/PALB2 Reid et al Nature Genetics 2007
Xia et al Nature Genetics 2007
FA-O FANCO/RAD51C Vaz et al Nature Genetics 2010
FA-P FANCP/SLX4 Stoepker et al Nature Genetics 2011
Kim et al Nature Genetics 2011
15+1 complementation Groups in Fanconi Anemia
![Page 8: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/8.jpg)
Interstrand crosslink repair (ICLR)
Fork stalling
Unhooking
Translesion sythesis
Recombination
![Page 9: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/9.jpg)
1 10 100 1000
100
80
60
40
20
0
FANC-C
FANC-G
FANC-F
FANC-A
MMC (nM)
Re
lati
ve
su
rviv
al
%
Empty
Retroviral subtyping of FA
![Page 10: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/10.jpg)
Genetic subtyping of 143 Spanish FA patients
Callen et al., Blood 2005; Casado et al., J Med Genet 2007; Kalb et al Am J Hum Genet
2007; Castella et al Blood 2011
Unassigned
![Page 11: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/11.jpg)
whole exome sequencing
501500
498 499
FA 287
![Page 12: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/12.jpg)
Mutations in the XPF nuclease lead to three
rare disorders: XP, XFE-progeria and FA
Xeroderma pigmentosum XFE-progeria Fanconi anemia
FANCQ alias for XPF in HUGO
Bogliolo et al., 2013, Am J Hum Genet
![Page 13: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/13.jpg)
Interstrand crosslink
repair (ICLR)
Fork stalling
Unhooking
Homologous recombination
Nucleotide excision
repair (NER)
recognition
dual incision
excision
DNA synthesis
ligation
UV
![Page 14: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/14.jpg)
NER
ICLR
Xeroderma pigmentosum
NER
ICLR
Xeroderma pigmentosum
NER
ICLR
Fanconi anemia
NER
ICLR
Fanconi anemia
NER ICLR
Progeria
NER ICLR
Progeria
1 gene (XPF), 2 repair pathways, 3 syndromes
Bogliolo et al., Am J Hum Genet (2013)
UV skin sensitivity anemia Anemia + UV skin
sensitivity
![Page 15: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/15.jpg)
C. Group Gene Reference
FA-A FANCA Lo Ten Foe et al. Nature Genetics 1996
FABC Consortium. Nature Genetics 1996
FA-B FANCB Meetei et al Nature Genetics 2004
FA-C FANCC Strathdee et al. Nature, 1992
FA-D1 FANCD1/BRCA2 Howlett et al. Science, 2002
FA-D2 FANCD2 Timmers et al. Mol Cell, 2001
FA-E FANCE De Winter et al. Am. J. Hum Genet 2000
FA-F FANCF De Winter et al. Nature Genet. 2000
FA-G FANCG De Winter et al. Nature Genet. 1998
FA-I FANCI Smogorzewska et al. Cell 2007
FA-J FANCJ/BRIP1 Levran et al. Nature Genet. 2005;Levitus et al. Nature Genet. 2005;
FA-L FANCL Ruhikanta et al. Nature Genet. 2003
FA-M FANCM Meetei et al Nature Genetics 2005
FA-N FANCN/PALB2 Reid et al Nature Genetics 2007
Xia et al Nature Genetics 2007
FA-O FANCO/RAD51C Vaz et al Nature Genetics 2010
FA-P FANCP/SLX4 Stoepker et al Nature Genetics 2011
Kim et al Nature Genetics 2011
FA-Q FANCQ/ERCC4 Bogliolo et al Am J Hum Genet 2013
16 complementation Groups in Fanconi Anemia
![Page 16: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/16.jpg)
![Page 17: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/17.jpg)
DNA damage response
Ataxia Telang.
Nijmegen
Fanconi
Bloom
Seckle
![Page 18: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/18.jpg)
Breast cancer risk in BRCA1/2 mutation carriers
![Page 19: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/19.jpg)
April 2005
Synthetic lethality in cancer therapeutics
![Page 20: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/20.jpg)
Synthetic lethality in cancer treatment
Lord and Ashworth BMC Biology 2010 8:38 doi:10.1186/1741-7007-8-38
![Page 21: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/21.jpg)
![Page 22: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/22.jpg)
![Page 23: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/23.jpg)
1 10 100 1000
100
80
60
40
20
0
FA disrupted
tumor cell
Tumor cell
Cisplatin concentration
Re
lati
ve
su
rviv
al
%
Chemo-sensitizing tumor cells through
disruption of the FA/BRCA pathway
Lyakhovich and Surralles, 2009
![Page 24: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/24.jpg)
From genes to therapy
-Bone marrow or umbilicar cord blood
transplant
-Preimplantational embryo selection
-Gene therapy?
-Stem cell based regenerative medicine???
![Page 25: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/25.jpg)
Dr. Eliane Gluckman and Matthew Farrow
1988
![Page 26: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/26.jpg)
1993
Dr. Pablo Rubinstein and Mitch Santa
Leukemia
Metabolic
syndromes
![Page 27: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/27.jpg)
Saviour babies to cure FA
Molly Nash
![Page 28: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/28.jpg)
Barcelona, August 15th 2006
![Page 29: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/29.jpg)
Trujillo and Surralles, in press
Savior babies and Fanconi anemia: low success
rates after 38 trials in 7 families
![Page 30: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/30.jpg)
![Page 31: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/31.jpg)
Gene therapy of severe immunodeficiencies
![Page 32: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/32.jpg)
![Page 33: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/33.jpg)
Fancostem/Fancolen team
C. Díaz de Heredia
Vall Hebron
J. Surrallés
UAB
J. Bueren
CIEMAT
J. Sevilla
Niño Jesús
![Page 34: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/34.jpg)
Phase I/II Gene therapy trial of Fanconi anemia
patients with a new Orphan Drug consisting of a
lentiviral vector carrying the FANCA gene
Coordinator: Juan Bueren (Madrid)
http://www.eurofancolen.eu
EuroFancoLen Project
WP1 WP2 WP3 WP4
J. Surralles A. Thrasher M. CavazzanaA. Galy and F. Mavilio
![Page 35: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/35.jpg)
Transduction with therapeutic
lentiviral vectors
blood mobilization
Plerixafor+filgrastim
CD34+ cells
Selection
Infusion of transduced graft
Gene therapy: Genetic Correction of
Hematopoietic Stem Cells (CD34+)
![Page 36: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/36.jpg)
cPPT Wpre*
GA
U3 R U5
PGK
SD SA
RRE
yFANCA
CMV R U5
PGK-FANCA.Wpre* LV
Orphan Medicinal Product Designation: EU 3/10/822
Lentiviral vector containing the Fanconi anemia A (FANCA) gene
Lentiviral vector: better and safer
![Page 37: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/37.jpg)
Figure 3
Navarro et al., 2008, Blood; Rio et al., 2009, Blood
It works in mice
Gene therapy of FANCD1 KO mice (BRCA2-/-)
![Page 38: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/38.jpg)
“Natural” vs “medical” gene therapy
Mutation Reverse mutation (mosaics)
After gene therapy
![Page 39: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/39.jpg)
Mosaicism often results in fast clinical improvement
hemoglobin
platelets
Des 2008
T=G
Oct 2013
T>>>G
Feb 2011
T>G
c.3335T>G
![Page 40: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/40.jpg)
Future
![Page 41: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/41.jpg)
![Page 42: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/42.jpg)
![Page 43: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/43.jpg)
FA fibro
IPS
cytokines Blood
Haematopoietic
progenitors
3-4 transcription
factors* (retro)
*KLF4, cMYC, OCT4, SOX2
FA gene (lenti)
Raya et al. Nature, 2009
Disease-corrected haematopoietic progenitors from
Fanconi anemia induced pluripotent stem (iPS) cells
![Page 44: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/44.jpg)
Disease-free cardiomyocytes from Fanconi anemia skin
Nature 460:53-59
![Page 45: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/45.jpg)
Making blood cells from IPS cells
Liu et al., Nature Communications, 2014
![Page 46: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/46.jpg)
Modeling Fanconi Anemia
therapeutics using patients iPSCs
Liu et al., Nature Communications, 2014
![Page 47: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/47.jpg)
DSB
DSB
mutation repair (genome editing or
homologous recombination)
save integration (save harbour)
Nucleases
(ZFN, TALEN, CRISPR)
Fixing rare diseases by genome editing
![Page 48: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/48.jpg)
Spanish FANCA mutational spectrum
Point mutationSplicing mutation
Microdeletion
MicroinsertionDeletion
Callén et al., 2005; Blood; Castella et al., Blood 2011
C>T missense mutation
![Page 49: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/49.jpg)
Genome editing to correct a FANCA mutation
by homologous recombination
(helper dependent adenoviral vectors targeting FANCA)
FA Crt FA-CFA Crt FA-C
Liu et al., Nature Communications, 2014
![Page 50: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/50.jpg)
Save harbour: the AAVS1 locus and ZFN
Rio et al., EMBO Mol Med, 2014
![Page 51: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/51.jpg)
Harvest of
CD34+ cells
Infusion of
genetically-
corrected Cells
PATIENT
Gene therapy in the future
Freezing of
CD34+ cells
Genome
editing
![Page 52: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/52.jpg)
Why research on rare genetic diseases?
• First umbilical cord blood transplant
• First savior baby
• Among the first gene therapy trials
• First generated disease-free IPS
• Model to understand fundamental biological processes (genome stability, cancer, ageing, pluripotency…)
• Tool to find new cancer drugs and new cancer genes
![Page 53: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/53.jpg)
Surrallés’ lab members, BarcelonaDr. M. Bogliolo
Dr. L. Mina
Dr. J. Minguillón
Dr. R. Pujol
Dr. MJ Ramírez
Dr. J. Surrallés
Dr. G. Hernández
Dr. M. Aza-Carmona
A. Molina
M. Marin
H. Montanuy
M. Peiró
Genome Instability Group: www.gig.uab.cat
![Page 54: "Rare diseases as platform to develop novel therapeutic strategies: the example of Fanconi anemia" by Prof. Jordi Surrallés Calonge](https://reader033.vdocuments.net/reader033/viewer/2022060205/55a19e661a28ab3e5c8b467d/html5/thumbnails/54.jpg)