In 2008, three independent research groups reported a successful subretinal injection of an AAV-based expression vector carrying the retinoid isomerohydrolase RPE65 (retinal pigment epithelium-specific 65 KDa protein) gene to improve vision in individuals with inherited blindness (Leber’s congenital amaurosis) [9,10,11]. Here, RPE65 is linked to Leber congenital amaurosis.