Luxturna® uses adeno-associated virus serotype 2 (AAV2) as a delivery vehicle to carry the wild-type Retinal Pigment Epithelium 65 (RPE65) gene into the retinal cells with RPE65 mutation for treating patients with Leber congenital amaurosis (LCA), a rare form of inherited vision loss [9]. The gene discussed is RPE65; the disease is Leber congenital amaurosis.