AAV-PHP.Cs Serotype
Skeletal muscle-targeted PHP variant with reduced off-target CNS and hepatic transduction.
Characteristics
CREATE-evolved for skeletal muscle specificity. Reduced off-target transduction versus AAV9. Maintains some BBB penetration capability. Improved muscle:liver transduction ratio.
Applications
Muscular dystrophy research in mice. Skeletal muscle-directed gene therapy. Systemic delivery with muscle focus.
Literature References
- Challis et al. (2019). Systemic AAV vectors for widespread and targeted gene delivery in rodents. Nat Protoc - Challis 2019 PHP.C
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