The efficacy and safety of voretigene neparvovec, which is a new drug for RPE65-mediated inherited retinal dystrophy using AAV2 discovered in human cells, and onasemnogene abeparvovec-xioi, which is a prescription for spinal muscular atrophy packaged by AAV9, have been confirmed and used for treatment39,40. The gene discussed is RPE65; the disease is inherited retinal dystrophy.