A phase 1/2 clinical trial and a subsequent randomized controlled phase 3 trial showed the safety and efficacy of a unilateral injection of the recombinant adenoviral vector voretigene neparvovec (VN) for the treatment of biallelic RPE65 mutation-related inherited retinal dystrophy (RPE65-IRD)4,5. Here, RPE65 is linked to inherited retinal dystrophy.