Voretigene neparvovec-rzyl (Luxturna, Spark Therapeutics) already is available for the treatment of biallelic RPE65-associated retinal dystrophy, with further trials underway to treat CHM-, RS1-, RPGR-, MERTK-, ABCA4-, USH2A-, MY07A-, CNGA3-, and CNGB3-associated retinal disease.10 The gene discussed is RPE65; the disease is Retinal dystrophy.