In the pivotal Phase III randomized controlled clinical trial, bilateral subretinal voretigene neparvovec-rzyl (Luxturna; AAV2-hRPE65v2; Spark Therapeutics [Philadelphia, PA]/Roche) administration was evaluated for safety and efficacy in patients with RPE65-mediated inherited retinal dystrophy [50]. Here, RPE65 is linked to inherited retinal dystrophy.