The confirmation of both pathogenic mutations in the three tested geFA-iPSCs, together with our observations showing that all stable iPSC clones contained the AAVS1-targeted FANCA gene (Supplementary Table S1) and had a functional FA pathway, demonstrates that the disease-free nature of gene-edited FA-iPSCs is a consequence of the functional insertion of FANCA within the AAVS1 safe harbor site of these reprogrammed FA cells. Here, FANCA is linked to Friedreich ataxia.