AAV5-GRK1-hRPGRco

AAV5-GRK1-hRPGRco is an **investigational ocular gene therapy** used in preclinical research for **X-linked retinitis pigmentosa** caused by **RPGR** mutations. It comprises an **AAV serotype 5 capsid** delivering a **codon-optimized full-length human RPGR transgene** under control of the **GRK1 photoreceptor-specific promoter**, with the goal of restoring RPGR expression in retinal photoreceptors after **subretinal administration**. In animal studies, this construct was designed to transduce rods and cones and to slow photoreceptor degeneration, thereby preserving retinal structure and visual function. The specific AAV5-based construct is primarily described as a **preclinical reference vector**, while later clinical development appears to have proceeded with a modified related construct using an AAV2tYF capsid rather than this exact AAV5 vector.

Other names

AAV2/5-GRK1-hRPGRcoAAV5 GRK1 hRPGRcoAAV-5 GRK1 hRPGRcoAAV 5 GRK1 hRPGRco