MyoAAV-2A Serotype
Muscle-tropic AAV capsid from M-CREATE directed evolution with high skeletal and cardiac muscle transduction efficiency and a distinct biodistribution profile from MyoAAV-1A. Cross-species validated in mouse, pig, and non-human primate, enabling capsid selection tailored to specific disease indications. Alternative or comparator to MyoAAV-1A for muscular dystrophy and cardiomyopathy gene therapy.
Characteristics
Alternative capsid from M-CREATE selection. High muscle transduction efficiency. Cross-species validation in multiple models. Slightly different biodistribution versus myoAAV-1A. Reduced immunogenicity.
Applications
Muscular dystrophy gene therapy. Cardiac muscle disorders. Alternative to myoAAV-1A for specific applications.
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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