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.

SUBMITTER: Elitt MS 

PROVIDER: S-EPMC6061782 | biostudies-literature | 2018 Aug

REPOSITORIES: biostudies-literature