Disease Modeling and Drug Discovery Using Human Pluripotent Stem Cells

Cellular reprogramming and genome editing have enabled the generation of disease-specific human pluripotent stem cells for disease modeling and drug discovery. Although these cells have been heralded as a novel and invaluable tool for these application and numerous proof-of-principle studies have demonstrated their potential, there are still several challenges that have to be addressed in order for them to efficiently translate into drug discovery programs. In this chapter, we review some of the recent progress made in the field and also highlight the challenges that must be overcome in the use of human pluripotent stem cells to recapitulate human disease phenotypes and to screen for therapeutic agents in vitro.