Some examples for CNS disorders include an AAV2 gene therapy designed to express RPE65 in the eye to treat Leber Congenital Amaurosis (Luxturna®) and an AAV9 vector that expresses SMN1 in motor neurons to treat Spinal Muscular Atrophy (Spinaraza®). The gene discussed is SMN1; the disease is proximal spinal muscular atrophy.