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Improvement and Decline in Vision with Gene Therapy in Childhood Blindness
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...
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| Publicado no: | N Engl J Med |
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| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2015
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4450362/ https://ncbi.nlm.nih.gov/pubmed/25936984 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1412965 |
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