Improvement and decline in vision with gene therapy in childhood blindness.
N Engl J Med
; 372(20): 1920-6, 2015 May 14.
Article
em En
| MEDLINE
| ID: mdl-25936984
ABSTRACT
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; ClinicalTrials.gov number, NCT00481546.).
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Retina
/
Terapia Genética
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Células Fotorreceptoras de Vertebrados
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Amaurose Congênita de Leber
Tipo de estudo:
Clinical_trials
/
Observational_studies
/
Prognostic_studies
Limite:
Adolescent
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Adult
/
Humans
Idioma:
En
Revista:
N Engl J Med
Ano de publicação:
2015
Tipo de documento:
Article