cellular models for human disease
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Using cellularmodels to
study humandisease
Conrad Schools of Science
March 20, 2013
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Diane Wuest
4th Year PhD Student
Chemical & Biomolecular EngineeringDept.
Kelvin ee a!oratory
Be"ore #rad School$o B.S. Chemical & Biomolecular
Engineering "rom Cornello %ored ' years at a !iotechnology
company
(ntroduction
Used
cells tostudycancerdrugs
Used large-scale cell
experients!in
"ioreactors#to a$e
Using cellsto a$e aodel ofthe "rain
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BrainDisease is
)ard toStudy*
+he !rain is the most comple, organ o"the human !ody*
Detailed research is liited!ecause itis hardto investigate the !rain o" a li%inghuan "eing
o Unlie simpler organs- the !rain cannot!e removed and repaired outside o"the !ody
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BrainDisease is
)ard toStudy*
ong history o" !rain e,aminationo starting in the European enaissance
during the /011s 2.explains the Latin
names
Scientists have learned more a!out the!rain in the last 15 years than in allprevious centuries
o development o" sophisticated iagingtechni&ues
3any uestions a!out the !rain are
unans5eredo disease understandingo possi!le treatments
Degenerative diseases o"adult li"e 6Alzheimersdisease7
8eurogenetic diseases6Huntingtons
disease)
3eta!olic diseasesGauchers disease
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Cellular3odels
6aa :in
vitro7Can )elpesearch
*So +e need to study the ((( to a$e "raindrugs
Scientists can gro+ and study "rain cells in thela"oratoryto study the BBB
= in vitro experiental approach
o ;
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Cellular3odels
6aa :in
vitro7Can )elpesearch
Can compare in vitro conditions to in vivo6animal models7 studies
o then hope"ully dra5 conclusions "or humans
.
+esting a drug onhumans is limited!y$
/. Patient num!ers=. )aving enough
sample volume'. Ethical
considerations4. 3oney
Screening ne5 drugsis e>cient & cost?e
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Biotechnology &
Engineering in
@ction*
In vitro models are an e,ample o"(C45678$
In vitro models are an e,ample o"575/57$
he use of "iological systes to a$euseful products
issues, Cells Model Syste
Designing, ipro%ing, and anufacturinga product that can "e used for any
applications
Use
athe
atic
sand
econoic
s
6arge-scale9roduction
Coprehensi%e
9ro"leSol%ing
Sol%e a coplex pro"le "y
siplifying :copartentali;ing