RPE65 and retinitis pigmentosa 1: Gene therapy for a single mutation has shown effectiveness in treating recessive RP.5,8 In 2017, the US FDA approved Luxturna, a novel RPE65 gene replacement therapy to treat patients with autosomal recessive RP caused by RPE65 mutation.9 But, to date, long-term efficacy from clinical reports has been controversial.5,10-12 In addition, the RPE65 mutation only accounts for a small percentage of RP cases.