Journal article

Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6

MJ Blankinship, P Gregorevic, JM Allen, SQ Harper, H Harper, CL Halbert, AD Miller, JS Chamberlain

Molecular Therapy | CELL PRESS | Published : 2004

Abstract

Vectors based on recombinant adeno-associated viruses (rAAV) have emerged as tools of choice for gene transfer to skeletal muscle. rAAV vectors demonstrate efficient, safe, and stable transduction. Multiple serotypes of AAV exist, but vectors based on serotype 2 (rAAV2) are the most thoroughly characterized and frequently employed. Here, we characterize transduction of the skeletal musculature using rAAV vectors pseudotyped with serotype 6 capsid proteins (rAAV6). We demonstrate that rAAV6 vectors can efficiently transduce the skeletal musculature of mice at levels >500-fold higher than is achievable with rAAV2 vectors and can readily saturate individual muscles following direct injection. F..

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University of Melbourne Researchers