Despite remarkable progress made in the development of therapies for these diseases over the past decade, which have culminated in approved gene therapy for the inherited retinal dystrophy, Leber congenital amaurosis (LCA) associated with biallelic mutations in RPE65 (Luxturna, Hoffmann-La Roche, Basel, Switzerland),3 and implantable retinal prostheses for end-stage retinal degeneration (Argus II; Second Sight Medical Products, Inc., Sylmar, CA; and Retinal Implant AG, Reutlingen, Germany), the majority of IRDs remain untreatable. This evidence concerns the gene RPE65 and Leber congenital amaurosis.