AAV9.61 Serotype

Engineered AAV9 variant with 10-fold improved cardiac-to-liver transduction ratio for heart-targeted gene therapy.

Length: 1 bp

Tropism: Cardiac muscle (highly selective), reduced liver and skeletal muscle

Characteristics

10-fold improved cardiac:liver transduction ratio. Capsid mutations reducing hepatic tropism. Enhanced cardiac specificity. Reduced off-target biodistribution.

Applications

Heart failure gene therapy. Cardiomyopathies. Cardiac-specific transgene expression with minimal hepatic exposure.

Literature References

  1. 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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