Cells heal themselves: gene-edited hematopoietic stem cells restore frataxin deficiency in multiple organs

Lucy R. Osborne. Cells heal themselves: gene-edited hematopoietic stem cells restore frataxin deficiency in multiple organs. J Clin Invest. 2026;136(20):e211630. doi:10.1172/JCI211630. 

 There is undoubtedly a lot of work to be done optimizing the editing efficiency of the repeat expansion, cell engraftment, and the timing of delivery, but the results reported by Sivakumar et al. are very encouraging and demonstrate that even with relatively limited editing and chimerism, there were concrete improvements in neurological, pancreatic, and cardiac function in the humanized mouse model of FRDA.

Recent developments in Friedreich's ataxia: a state-of-the-art review

Chapman LR, Mortiboys H, Shaw PJ. Recent developments in Friedreich's ataxia: a state-of-the-art review. Brain Commun. 2026 Apr 20;8(3):fcag143. doi: 10.1093/braincomms/fcag143. PMID: 42158846; PMCID: PMC13180647. 

We have conducted a critical review of all the studies published between 1999 and December 2024. The free text search terms were ((Friedreich's Ataxia) OR (FRDA) OR (FRDA) OR (inherited ataxias)) AND ((recent developments) OR (new developments) OR (new treatments) OR (phenotype)).