LCA is an early-onset retinal dystrophy leading to blindness or severe visual impairment in infants.82 LCA is a heterogenous retinal disease family with more than 400 mutations in 14 LCA-associated genes, several of which are also implicated in other retinal diseases, such as RP.83 Luxturna, the first FDA-approved in vivo gene therapy for the eye, targets autosomal recessive LCA2 that is caused by mutations in RPE65. This evidence concerns the gene RPE65 and Leber congenital amaurosis.