PhysiologybioRxivPreprint
Axonopathy in Duchenne Muscular Dystrophy limits microdystrophin gene therapy efficacy
Aly Bourguiba, Maxime Gelin, Aurélie Fail…·France Piétri‐Rouxel
This study shows that axonopathy in DMD limits microdystrophin gene therapy efficacy by demonstrating that dystrophin is expressed in peripheral nerves and its absence causes nerve structural collapse and impaired nerve-muscle connectivity.