Systemic AAV vectors for widespread and targeted gene delivery in rodents

Challis et al. (2019). Nat Protoc DOI: 10.1038/s41596-018-0097-3 Citations: 453

Key findings

  • PHP.C2 evolved through third-generation CREATE for enhanced cardiac tropism. PHP.Cs designed for skeletal muscle targeting. Both maintain some BBB penetration capability. Expanded PHP toolkit beyond CNS applications.