The AQP-1-positive area (Fig. 8i, j), mitochondrial morphology and function (Fig. 8k–n), and the expression of mitochondrial marker genes such as ATP synthase (Supplementary Fig. 12b) and Nd1 (Supplementary Fig. 12c) were restored in FA mice treated with the EPRS1 inhibitor. Here, MT-ND1 is linked to Friedreich ataxia.