RPE65 and inherited retinal dystrophy: There are five major FDA-approved AAV gene therapies: (i) Luxturna (voretigene neparvovec), the first FDA-approved AAV gene therapy, developed by Spark Therapeutics, approved in 2017 for the treatment of biallelic RPE65 mutation-associated retinal dystrophy, which uses AAV2 vector to deliver functional RPE65 gene to retinal cells [138,269,270,271].