Luxturna (voretigene neparvovec-rzyl) is an AAV2-based vector that delivers the retinoid isomerohydrolase RPE65 [6], the mutated gene in Leber’s congenital amaurosis, and was the first gene therapy treatment approved by the U.S. Food and Drug Administration (FDA) following improved functional vision in a previously untreatable retinal dystrophy in human clinical trials. Here, RPE65 is linked to inherited retinal dystrophy.