Fig. 1

Cellular models derived from FRDA iPSCs. FRDA patient-derived fibroblasts are reprogrammed into iPSCs by cellular reprogramming through the overexpression of OCT4, SOX2, C-MYC and KLF4, transcription factors known to induce pluripotency. iPSC colonies are expanded, selected and confirmed to express pluripotency markers and to be karyotypically normal. At the iPSC stage, cells can be converted into almost any cell type. Particularly relevant for FRDA is the conversion to neurons, cardiomyocytes, pancreatic β cells and retinal pigment epithelial cells. Figure created with BioRender.com