Luxturna and Zolgensma are adeno-associated virus (AAV) gene therapies for RPE65 mutation-associated retinal dystrophy and spinal muscular atrophy (SMA), replacing the dysfunctional alleles of the RPE65 or SMN1 gene, respectively, with their functional copies [74, 75]. This evidence concerns the gene SMN1 and proximal spinal muscular atrophy.