Drug screening for human genetic diseases using iPSC models

Matthew S. Elitt; Lilianne Barbar; Paul J. Tesar

doi:10.1093/hmg/ddy186

Drug screening for human genetic diseases using iPSC models

Matthew S. Elitt, Lilianne Barbar, Paul J. Tesar

Research output: Contribution to journal › Review article › peer-review

110 Scopus citations

Abstract

Induced pluripotent stem cells (iPSCs) enable the generation of previously unattainable, scalable quantities of disease-relevant tissues from patients suffering from essentially any genetic disorder. This cellular material has proven instrumental for drug screening efforts on these disorders, and has facilitated the identification of novel therapeutics for patients. Here we will review the foundational technologies that have enabled iPSCs, the power and limitations of iPSC-based compound screens along with screening guidelines, and recent examples of screening efforts. Additionally we will provide a brief commentary on the future scientific roadmap using pluripotent- and 3D organoid-based, combinatorial approaches.

Original language	English
Pages (from-to)	R89-R98
Journal	Human molecular genetics
Volume	27
Issue number	2
DOIs	https://doi.org/10.1093/hmg/ddy186
State	Published - Aug 1 2018

Access to Document

10.1093/hmg/ddy186

Cite this

@article{f8fc47dccc4e4cafb59b07c3a2c59b2f,

title = "Drug screening for human genetic diseases using iPSC models",

abstract = "Induced pluripotent stem cells (iPSCs) enable the generation of previously unattainable, scalable quantities of disease-relevant tissues from patients suffering from essentially any genetic disorder. This cellular material has proven instrumental for drug screening efforts on these disorders, and has facilitated the identification of novel therapeutics for patients. Here we will review the foundational technologies that have enabled iPSCs, the power and limitations of iPSC-based compound screens along with screening guidelines, and recent examples of screening efforts. Additionally we will provide a brief commentary on the future scientific roadmap using pluripotent- and 3D organoid-based, combinatorial approaches.",

author = "Elitt, {Matthew S.} and Lilianne Barbar and Tesar, {Paul J.}",

year = "2018",

month = aug,

day = "1",

doi = "10.1093/hmg/ddy186",

language = "English",

volume = "27",

pages = "R89--R98",

journal = "Human molecular genetics",

issn = "0964-6906",

number = "2",

}

TY - JOUR

T1 - Drug screening for human genetic diseases using iPSC models

AU - Elitt, Matthew S.

AU - Barbar, Lilianne

AU - Tesar, Paul J.

PY - 2018/8/1

Y1 - 2018/8/1

N2 - Induced pluripotent stem cells (iPSCs) enable the generation of previously unattainable, scalable quantities of disease-relevant tissues from patients suffering from essentially any genetic disorder. This cellular material has proven instrumental for drug screening efforts on these disorders, and has facilitated the identification of novel therapeutics for patients. Here we will review the foundational technologies that have enabled iPSCs, the power and limitations of iPSC-based compound screens along with screening guidelines, and recent examples of screening efforts. Additionally we will provide a brief commentary on the future scientific roadmap using pluripotent- and 3D organoid-based, combinatorial approaches.

AB - Induced pluripotent stem cells (iPSCs) enable the generation of previously unattainable, scalable quantities of disease-relevant tissues from patients suffering from essentially any genetic disorder. This cellular material has proven instrumental for drug screening efforts on these disorders, and has facilitated the identification of novel therapeutics for patients. Here we will review the foundational technologies that have enabled iPSCs, the power and limitations of iPSC-based compound screens along with screening guidelines, and recent examples of screening efforts. Additionally we will provide a brief commentary on the future scientific roadmap using pluripotent- and 3D organoid-based, combinatorial approaches.

UR - http://www.scopus.com/inward/record.url?scp=85059244548&partnerID=8YFLogxK

U2 - 10.1093/hmg/ddy186

DO - 10.1093/hmg/ddy186

M3 - Review article

C2 - 29771306

AN - SCOPUS:85059244548

SN - 0964-6906

VL - 27

SP - R89-R98

JO - Human molecular genetics

JF - Human molecular genetics

IS - 2

ER -

Drug screening for human genetic diseases using iPSC models

Abstract

Access to Document

Other files and links

Fingerprint

Cite this