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Vitreal delivery of AAV vectored Cnga3 restores cone function in CNGA3(−/−)/Nrl(−/−) mice, an all-cone model of CNGA3 achromatopsia(†)

The CNGA3(−/−)/Nrl(−/−) mouse is a cone-dominant model with Cnga3 channel deficiency, which partially mimics the all cone foveal structure of human achromatopsia 2 with CNGA3 mutations. Although subretinal (SR) AAV vector administration can transfect retinal cells efficiently, the injection-induced...

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Dettagli Bibliografici
Pubblicato in:Hum Mol Genet
Autori principali: Du, Wei, Tao, Ye, Deng, Wen-Tao, Zhu, Ping, Li, Jie, Dai, Xufeng, Zhang, Yuxin, Shi, Wei, Liu, Xuan, Chiodo, Vince A., Ding, Xi-Qin, Zhao, Chen, Michalakis, Stylianos, Biel, Martin, Zhang, Zuoming, Qu, Jia, Hauswirth, William W., Pang, Ji-jing
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2015
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4459390/
https://ncbi.nlm.nih.gov/pubmed/25855802
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv114
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