Bone marrow transplantation stimulates neural repair in Friedreich's ataxia mice

Kevin C Kemp, Kelly Hares, Juliana Redondo, Amelia J Cook, Harry R Haynes, Bronwen R Burton, Mark A Pook, Claire M Rice, Neil J Scolding and Alastair Wilkins; Annals of Neurology, Accepted manuscript online: 13 MAR 2018 DOI: 10.1002/ana.25207

Our data provide proof-of-concept of gene replacement therapy, via allogeneic bone marrow transplantation, that reverses neurological features of Friedreich's ataxia with the potential for rapid clinical translation.

 Bone marrow transplantation stimulates neural repair in Friedreich's ataxia mice