pAAV.IVT18
AAV packaging plasmid expressing AAV2 Rep protein and AAV.IVT18 capsid protein
AAV.IVT18-scRS/CMV-RS1, comprising a rationally designed novel capsid (AAV.IVT18) and an optimized RS1 gene expression cassette, was then constructed and delivered via intravitreal injection into mutant and wild-type (WT) mice at 3 to 4 weeks of age.
Wei, X., et al., A Novel AAV Capsid-Mediated RS1 Gene Therapy Restored Retinal Function to Wild-Type Levels in Rs1R213W Mouse Model. Investigative Ophthalmology & Visual Science, 2025. 66(4).
>pAAV9.HR
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