Design of Tissue-specific Regulatory Cassettes for High-level rAAV-mediated Expression in Skeletal and Cardiac Muscle

Salva, Maja Z; Himeda, Charis L; Tai, Phillip WL; Nishiuchi, Eiko; Gregorevic, Paul; Allen, James M; Finn, Eric E; Nguyen, Quynh G; Blankinship, Michael J; Meuse, Leonard; Chamberlain, Jeffrey S; Hauschka, Stephen D

doi:10.1038/sj.mt.6300027

Design of Tissue-specific Regulatory Cassettes for High-level rAAV-mediated Expression in Skeletal and Cardiac Muscle

Systemic delivery of recombinant adeno-associated virus (rAAV) 6 vectors mediates efficient transduction of the entire striated musculature, making this an attractive strategy for muscle gene therapy. However, owing to widespread transduction of non-muscle tissues, optimization of this method wou…