About 4–16% of patients with LCA subtype 2 (LCA2) are found to have mutations in RPE65. Preclinical models of RPE65-related degeneration have been produced in order to move onto clinical trials [93] and, among them, canine [157], murine (rd12) [158], and the genetically built Rpe65−/− knockout mouse [159] mimic the retinal degeneration seen in the disease. This evidence concerns the gene RPE65 and Leber congenital amaurosis 2.