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.
Formáid: Artigo
Teanga:Inglês
Foilsithe: 2010
Ábhair:
Article
Rochtain Ar Líne: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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