Improvement and decline in vision with gene therapy in childhood blindness.
Ontology highlight
ABSTRACT:
SUBMITTER: Jacobson SG
PROVIDER: S-EPMC4450362 | biostudies-literature | 2015 May
REPOSITORIES: biostudies-literature
Publications
Improvement and decline in vision with gene therapy in childhood blindness.
Jacobson Samuel G SG Cideciyan Artur V AV Roman Alejandro J AJ Sumaroka Alexander A Schwartz Sharon B SB Heon Elise E Hauswirth William W WW
The New England journal of medicine 20150503 20
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 ...[more]
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