RPE65 and retinitis pigmentosa 1: Patients with RPE65-associated RP can now be treated with the first commercially available gene augmentation therapy LUXTURNATM (voretigene neparvovec-rzyl; Spark Therapeutics, Inc., Philadelphia, PA, USA), which delivers a healthy copy of the RPE65 cDNA to the retinal cells using an adeno-associated virus (AAV) [34,35].