Induced pluripotent stem cells-derived neurons from patients with Friedreich ataxia exhibit differential sensitivity to resveratrol and nicotinamide

Pauline Georges, Maria-Gabriela Boza-Moran, Jacqueline Gide, Georges Arielle Pêche, Benjamin Forêt, Aurélien Bayot, Pierre Rustin, Marc Peschanski, Cécile Martinat & Laetitia Aubry ; Scientific Reports volume 9, Article number: 14568 (2019) doi:10.1038/s41598-019-49870-y

By comparing the effects of both molecules on different cell types that may be considered to be non-relevant for the disease, such as fibroblasts, or more relevant to the disease, such as neurons differentiated from iPSCs, a differential response was observed; this response suggests the importance of developing more predictive in vitro systems for drug discovery. Our results demonstrate the value of utilizing human iPSCs early in drug discovery to improve translational predictability.

Induced pluripotent stem cells-derived neurons from patients with Friedreich ataxia exhibit differential sensitivity to resveratrol and nicotinamide