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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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Bibliografische gegevens
Gepubliceerd in:Hum Mol Genet
Hoofdauteurs: Datta, Poppy, Ruffcorn, Avri, Seo, Seongjin
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Oxford University Press 2020
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Online toegang: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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