NeurosciencebioRxivPreprint
Transcriptional profiling of isogenic Friedreich ataxia induced pluripotent stem cell-derived neurons
Jiun‐I Lai, Daniel C. Nachun, Lina Petrosyan…·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.
DOI 10.1101/457093