Axonopathy in Duchenne Muscular Dystrophy limits microdystrophin gene therapy efficacy
Aly Bourguiba, Maxime Gelin, Aurélie Fail, Lucile Saillard, Stéphanie Bauché, Cécile Peccate, Pierre Meunier, Zoheir Guesmia, Lou Ann Mirabile, Julianne Perronnet, Mégane Lemaître, Lorenzo Giordani, Sestina Falcone, Christel Gentil, 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.
bioRxiv · Aug 20, 2026 · PreprintPhysiology