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Limited time window for retinal gene therapy in a preclinical model of ciliopathy

Retinal degeneration is a common clinical feature of ciliopathies, a group of genetic diseases linked to ciliary dysfunction, and gene therapy is an attractive treatment option to prevent vision loss. Although the efficacy of retinal gene therapy is well established by multiple proof-of-concept prec...

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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Datta, Poppy, Ruffcorn, Avri, Seo, Seongjin
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7424757/
https://ncbi.nlm.nih.gov/pubmed/32568387
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddaa124
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