Leber’s congenital amaurosis (LCA), a much earlier onset and faster progressing form of RP, has only recently received effective treatments in the form of gene therapy to replace the defective gene associated with the disease; however, these therapies do not work for all the genes associated with the diseases (LCA has an FDA-approved gene therapy for supplementing the gene for retinal pigment epithelium 65 kD protein, or RPE65) [35,36]. The gene discussed is RPE65; the disease is Leber congenital amaurosis.