AAV9.45 Serotype
Liver-detargeted AAV9 variant engineered to reduce hepatic uptake while maintaining cardiac and skeletal muscle transduction.
Characteristics
5-fold reduction in liver uptake versus AAV9. Maintained cardiac and skeletal muscle transduction efficiency. Rational mutagenesis approach. Improved therapeutic index for muscle-targeted applications.
Applications
Cardiac gene therapy with reduced liver exposure. Muscular dystrophies. Systemic muscle-directed gene delivery.
Literature References
- Pulicherla et al. (2011). Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer. Mol Ther - Pulicherla 2011 AAV9 Variants
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