PubReading [326] - Drug screening for human genetic diseases using iPSC models - M. Elitt, L. Barbar & P. Tesar

<strong>Induced pluripotent stem cells</strong> (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 <strong>drug screening</strong> efforts on these disorders, and has facilitated the identification of novel <strong>therapeutics</strong> for patients. Here we will review the foundational technologies that have enabled <strong>iPSC</strong>s, 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.<em>doi: 10.1093/hmg/ddy186 - 2018</em>