Pluripotent stem cells in disease modelling and drug discovery

Yishai Avior, Ido Sagi & Nissim Benvenisty; Nature Reviews Molecular Cell Biology (2016) doi:10.1038/nrm.2015.27 Published online 28 January 2016

The ability to model human diseases using cultured PSCs has revolutionized the ways in which we study monogenic, complex and epigenetic disorders, as well as early- and late-onset diseases. Despite the promising future of PSC-based therapies, there are still substantial hurdles between its potential and its fulfilment. In the future, we expect to have repositories of PSCs that will enable the modelling of practically any genetic disease.
 Examples of drug screening in Friedreich's ataxia using patient-derived induced pluripotent stem cell models: Forskolin and RG2833

 Pluripotent stem cells in disease modelling and drug discovery