SMN1 and Leber congenital amaurosis 2: For example, Luxturna (AAV2.hRPE65v2) for the treatment of Leber’s congenital amaurosis type 2 (LCA2), a retinal dystrophy that affects children [23] and Zolgensma, which prevents the early death (usually before the 2nd birthday) of patients with spinal muscular atrophy (SMA) by delivering SMN1 through an AAV9 [44].