Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

To the Editor: Leber's congenital amaurosis, a common cause of blindness in infants and children, 1 recently became the first human genetic retinal disease to show improved vision in response to treatment. Patients with mutations in the gene encoding retinal pigment epithelium–specific 65-kD protein ( RPE65 ) had gains in vision within weeks after subretinal injection of a vector containing the gene in one eye. 2 – 5 At 1-year follow-up after gene therapy, the three young adult patients in our trial 4 , 5 remained without serious adverse events. A noteworthy observation in one patient at 1 year after treatment prompted further studies. For . . .