bioRxiv / medRxiv
open access
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. However, their efficacy in recovering functional outcomes remains to improve. We hypothesized that this limitat
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