Indeed, genetic testing results containing a RPE65 VUS can prevent patients from obtaining treatment with an FDA-approved gene therapy, Voretigene neparvovec (Luxturna, Spark Therapeutics, Philadelphia, PA).9,10 Mutations in the RPE65 gene account for 0.6–6% of RP and 3–16% of LCA/EORD cases,11 and patients are eligible for treatment only if they have documented biallelic pathogenic or likely pathogenic mutations.12 The gene discussed is RPE65; the disease is Leber congenital amaurosis.