Food and Drug Administration (FDA) approved the first gene transfer treatment for the ocular disease in 2017, where a subretinal delivery of adeno-associated virus serotype 2 (AAV2)-mediated voretigene neparvovecrzyl (Luxturna®,Spark Therapeutics, Inc., Philadephia, PA, USA) is administered for treating inherited biallelic RPE65 mutation-associated retinal dystrophy [8]. The gene discussed is RPE65; the disease is inherited retinal dystrophy.