In a phase III randomized controlled trial (RCT) (NCT00999609), 31 participants (21 in the intervention group and 10 in the control group) with confirmed RPE65-associated retinal dystrophy received bilateral sequential subretinal injections of AAV2-hRPE65v2 (voretigene neparvovec, Luxturna) [96,97]. The gene discussed is RPE65; the disease is inherited retinal dystrophy.