Journal
NEW ENGLAND JOURNAL OF MEDICINE
Volume 372, Issue 20, Pages 1920-1926Publisher
MASSACHUSETTS MEDICAL SOC
DOI: 10.1056/NEJMoa1412965
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Funding
- National Eye Institute
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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.
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