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The Status of RPE65 Gene Therapy Trials: Safety and Efficacy
Several groups have reported the results of clinical trials of gene augmentation therapy for Leber congenital amaurosis (LCA) because of mutations in the RPE65 gene. These studies have used subretinal injection of adeno-associated virus (AAV) vectors to deliver the human RPE65 cDNA to the retinal pi...
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| Publicado no: | Cold Spring Harb Perspect Med |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Cold Spring Harbor Laboratory Press
2015
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4561397/ https://ncbi.nlm.nih.gov/pubmed/25635059 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1101/cshperspect.a017285 |
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