Transcriptional profiling of isogenic Friedreich ataxia induced pluripotent stem cell-derived neurons
Jiun‐I Lai, Daniel C. Nachun, Lina Petrosyan, Benjamin T. Throesch, Erica Campau, Fuying Gao, Kristin K. Baldwin, Giovanni Coppola, Joel M. Gottesfeld, Elisabetta Soragni
This study uses isogenic iPSC-derived neurons to analyze the transcriptomic effects of GAA*TTC repeat expansion in Friedreich ataxia and shows partial restoration with HDACi treatment.
bioRxiv · Oct 30, 2018 · PreprintNeuroscience