Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species
Key findings
MyoAAV-1A through 4A evolved through M-CREATE (muscle-tropic CREATE) selection in mice. Up to 100-fold enhanced skeletal and cardiac muscle transduction versus AAV9. Cross-species validation in mouse, pig, and non-human primate. Reduced liver and immune activation. Breakthrough for muscular dystrophy gene therapy.