hRPGRstb

hRPGRstb is an investigational **gene therapy transgene construct** consisting of a stabilized, truncated version of the human **retinitis pigmentosa GTPase regulator** gene designed for treatment of **X-linked retinitis pigmentosa** caused by **RPGR** mutations. It has been developed by **AGTC** for delivery in recombinant AAV ocular gene therapy vectors, including vectors using the proprietary **AAV2tYF** capsid and a **GRK1** photoreceptor-directed promoter. The stabilization strategy removes repetitive regions that can cause sequence instability during vector engineering and manufacturing, while preserving therapeutic RPGR function. Its intended mechanism is **gene replacement**, restoring RPGR expression in retinal photoreceptors to slow or prevent degeneration of rods and cones and preserve vision.

Other names

stabilized truncated human RPGR transgenestabilized truncated human retinitis pigmentosa GTPase regulator gene