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N Engl J Med. 2015 May 14;372(20):1920-6. doi: 10.1056/NEJMoa1412965. Epub 2015 May 3.

Improvement and decline in vision with gene therapy in childhood blindness.

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From the Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia (S.G.J., A.V.C., A.J.R., A.S., S.B.S.); the Departments of Ophthalmology and Vision Sciences, the Hospital for Sick Children, University of Toronto, Toronto (E.H.); and the Department of Ophthalmology, University of Florida, Gainesville (W.W.H.).


Retinal gene therapy for Leber's congenital amaurosis, an autosomal recessive childhood blindness, has been widely considered to be safe and efficacious. Three years after therapy, improvement in vision was maintained, but the rate of loss of photoreceptors in the treated retina was the same as that in the untreated retina. Here we describe long-term follow-up data from three treated patients. Topographic maps of visual sensitivity in treated regions, nearly 6 years after therapy for two of the patients and 4.5 years after therapy for the third patient, indicate progressive diminution of the areas of improved vision. (Funded by the National Eye Institute; number, NCT00481546.).

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