L. Osborne

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

Friedreich's ataxia (FRDA) is a triplet repeat expansion disorder with multisystem, progressive symptoms caused by reduced frataxin protein levels and compromised mitochondrial function. Restoration of frataxin protein is a focus of clinical trials involving adeno-associated viral vectors, but substantial barriers exis...