Systemic gene transfer to skeletal muscle using reengineered AAV vectors

Methods Mol Biol. 2011:709:141-51. doi: 10.1007/978-1-61737-982-6_9.

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.

MeSH terms

  • Animals
  • Capsid Proteins / genetics*
  • Dependovirus / genetics*
  • Disease Models, Animal
  • Gene Transfer Techniques*
  • Genetic Engineering*
  • Genetic Therapy / methods
  • Genetic Vectors*
  • Mice
  • Muscle, Skeletal*
  • Muscular Dystrophies / genetics
  • Muscular Dystrophies / therapy
  • Muscular Dystrophy, Animal / genetics
  • Muscular Dystrophy, Animal / therapy
  • Tropism

Substances

  • Capsid Proteins