Axonopathy in Duchenne Muscular Dystrophy limits microdystrophin gene therapy efficacy

Duchenne muscular dystrophy (DMD) is classically defined as a primary myopathy, and current AAV-mediated microdystrophin gene therapies are shown to successfully preserve muscle integrity.

Duchenne muscular dystrophy (DMD) is classically defined as a primary myopathy, and current AAV-mediated microdystrophin gene therapies are shown to successfully preserve muscle integrity. However, their efficacy in recovering functional outcomes remains to improve. We…