Recently, phase III clinical trials of gene augmentation therapy have shown promising and successful results in RPE65-associated inherited retinal dystrophy (RPE65-IRD) cases, and Luxturna (voretigene neparvovec-rzyl) was approved in 2017 by the U.S. Food and Drug Administration as the first gene therapy for IRD patients [14,15,16]. The gene discussed is RPE65; the disease is inherited retinal dystrophy.