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Updated: Jun 5, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
Systemic gene transfer to skeletal muscle using reengineered AAV vectors
Jana L Phillips1, Julia Hegge, Jon A Wolff
1Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Abstract:
Gene therapy of musculoskeletal disorders warrants efficient gene transfer to a wide range of muscle groups. Reengineered adeno-associated viral (AAV) vectors that selectively transduce muscle tissue following systemic administration are attractive candidates for such applications. Here we provide examples of several lab-derived AAV vectors that display systemic tissue tropism in mice. Methods to evaluate the efficiency of gene transfer to skeletal muscle following intravenous or isolated limb infusion of AAV -vectors in mice are discussed in detail.

