Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency

To test whether fast-acting, self complimentary(sc), AAV vector-mediated RPE65 expression prevents cone degeneration and/or restores cone function, two mouse lines were studied: the Rpe65- deficient rd12 mouse and the Rpe65- deficient, rhodpsin null (‘i.e. cone function-only’) Rpe65(−/−)::Rho(−/−) m...

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Main Authors: Pang, Jijing, Boye, Shannon E., Lei, Bo, Boye, Sanford L., Everhart, Drew, Ryals, Renee, Umino, Yumiko, Rohrer, Bärbel, Alexander, John, Li, Jie, Dai, Xufeng, Li, Qiuhong, Chang, Bo, Barlow, Robert, Hauswirth, William W.
Format: Artigo
Sprog:Inglês
Udgivet: 2010
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3014850/
https://ncbi.nlm.nih.gov/pubmed/20237510
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2010.29
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