Viral particles retargeted to skeletal muscle

Abstract

Provided herein are compositions and methods for retargeting viral particles. e.g. adeno-associated virus (AAV) particles, to muscle cells using muscle-specific surface proteins. AAV adapted accordingly may be a viable gene therapy platform for the treatment of a skeletal muscle related disorder (e.g., X-linked myotubular myopathy (XLMTM). Duchenne muscular dystrophy (DMD), myotonic dystrophy (DM1), Facioscapulohumeral muscular dystrophy Type 1 (FSHD), congenital muscular dystrophy type 1A (MDC1A), Limb girdle muscular dystrophy, dystroglycanopathy, etc.) in a patient in need thereof.

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