MyoAAV-1A Serotype
Lead muscle-tropic AAV capsid from M-CREATE directed evolution with 100-fold greater skeletal and cardiac muscle transduction than AAV9. Cross-species validated in mouse, pig, and non-human primate with substantially reduced liver off-target uptake. Primary delivery vector for Duchenne muscular dystrophy, limb-girdle muscular dystrophy, and cardiomyopathy gene therapy.
Characteristics
100-fold enhanced muscle transduction versus AAV9. M-CREATE (muscle-tropic CREATE) selection in mice. Cross-species efficacy in mouse, pig, and NHP. Reduced liver uptake and immune activation. Peptide insertion conferring muscle-specific receptor binding.
Applications
Duchenne muscular dystrophy gene therapy. Limb-girdle muscular dystrophies. Cardiomyopathies. Translational muscle gene therapy (validated in large animals).
Literature References
- Tabebordbar et al. (2021). Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species. Cell - Tabebordbar 2021 MyoAAV
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