title: Systemic gene transfer to skeletal muscle using reengineered AAV vectors.
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.
authors.affiliation: Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
authors.forename: Jana L
authors.initials: JL
authors.lastname: Phillips
authors.forename: Julia
authors.initials: J
authors.lastname: Hegge
authors.forename: Jon A
authors.initials: JA
authors.lastname: Wolff
authors.forename: R Jude
authors.initials: RJ
authors.lastname: Samulski
authors.forename: Aravind
authors.initials: A
authors.lastname: Asokan
mesh_terms: D000818:Animals; D036022:Capsid Proteins / Q000235:genetics*; D000229:Dependovirus / Q000235:genetics*; D004195:Disease Models, Animal; D018014:Gene Transfer Techniques; D005818:Genetic Engineering; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors; D051379:Mice; D018482:Muscle, Skeletal; D009136:Muscular Dystrophies / Q000235:genetics / Q000628:therapy; D009137:Muscular Dystrophy, Animal / Q000235:genetics / Q000628:therapy; D018523:Tropism
chemical_list: D036022:Capsid Proteins