Furthermore, we generated hCBOs from patients with FRDA, the most common inherited ataxia caused by abnormal GAA triplet-repeats in the FXN gene that affects 1 in 50,000 people globally.16, 17 The use of patient-derived iPSC lines with short and long GAA repeats, which differentially impact frataxin expression levels and correlate with disease severity in the clinic, enabled the identification of disease-phenotypes that were reversed by chemical and genetic strategies. This evidence concerns the gene FXN and Friedreich ataxia.