Oxidative stress and loss of Fe-S proteins in Friedreich ataxia induced pluripotent stem cell-derived PSNs can be reversed by restoring FXN expression with a benzamide HDAC inhibitor.
Amélie Hu, Myriam Rai, Simona Donatello, Massimo Pandolfo
This study shows that FXN deficiency in FRDA patient-derived PSNs can be partially reversed by a benzamide HDAC inhibitor, restoring Fe-S proteins and reducing oxidative stress.
bioRxiv · Nov 17, 2017 · PreprintNeuroscience