RPE65 and inherited retinal dystrophy: One is AAV2-mediated delivery of the RPE65 gene in treating confirmed biallelic RPE65-mediated inherited retinal dystrophy in 2017, the other is AAV9-mediated therapy that functionally replaces the mutated survival motor neuron 1 gene in treating spinal muscular atrophy type 1 disease in children under 2 years of age.