RHO1-2 meganuclease

**RHO1-2 meganuclease** is an engineered allele-specific gene-editing therapy being developed for **autosomal dominant retinitis pigmentosa** caused by the **P23H mutation in the rhodopsin gene**. The active agent is an engineered **I-CreI-derived meganuclease** delivered by a **self-complementary AAV5 vector** under a **GRK1 photoreceptor-specific promoter** and administered by **subretinal injection**. It is designed to recognize a 22-base-pair sequence spanning the mutant **P23H RHO** allele, induce a targeted double-strand break, and inactivate the toxic mutant allele through error-prone end joining while sparing the wild-type allele. Preclinical work in transgenic P23H pig models has shown restoration of rod structure and function, correction of rhodopsin mislocalization, preservation of cones, and durable activity through at least one year. The program has been associated with **Precision BioSciences** and remains a preclinical ophthalmic genome-editing candidate.

Other names

RHO1-2 meganucleaseRHO-1-2 meganucleaseRHO 1-2 meganuclease