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.

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