Recently, there has been significant progress in several gene therapies for RP and related diseases, in particular retinal pigment epithelium-specific 65 kDa protein (RPE65) gene therapy for treatment of Leber congenital amaurosis (LCA) in humans [6-11], two mouse models of aryl hydrocarbon receptor interacting protein-like 1 (AIPL1) gene therapy [12], and retinitis pigmentosa GTPase regulator (RPGR) gene therapy in canine models of X-linked RP [13]. Here, RPGR is linked to Leber congenital amaurosis.