Systemic gene transfer to skeletal muscle using reengineered AAV vectors.
Journal Article (Journal Article)
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.
- Phillips, JL; Hegge, J; Wolff, JA; Samulski, RJ; Asokan, A
Volume / Issue
- 709 /
Start / End Page
- 141 - 151
Pubmed Central ID
Electronic International Standard Serial Number (EISSN)
Digital Object Identifier (DOI)
- United States