To date, only an inherited retinal disease, namely Leber’s congenital amaurosis, can be treated effectively by gene augmentation with voretigene neparvovec (Luxturna®, Spark Therapeutics, Pennsylvania, USA) via adenovirus-associated virus vectors transporting the RPE65 transgene into target cells [94]. The gene discussed is RPE65; the disease is Leber congenital amaurosis.