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Gene therapy: light is finally in the tunnel

After two decades of ups and downs, gene therapy has recently achieved a milestone in treating patients with Leber’s congenital amaurosis (LCA). LCA is a group of inherited blinding diseases with retinal degeneration and severe vision loss in early infancy. Mutations in several genes, including RPE6...

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Detalhes bibliográficos
Publicado no:Protein Cell
Main Authors: Cao, Huibi, Molday, Robert S., Hu, Jim
Formato: Artigo
Idioma:Inglês
Publicado em: Higher Education Press 2012
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4728158/
https://ncbi.nlm.nih.gov/pubmed/22231356
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s13238-011-1126-y
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