Gene Therapy Rescues Cone Structure and Function in the 3-Month-Old rd12 Mouse: A Model for Midcourse RPE65 Leber Congenital Amaurosis

PURPOSE. RPE65 function is necessary in the retinal pigment epithelium (RPE) to generate chromophore for all opsins. Its absence results in vision loss and rapid cone degeneration. Recent Leber congenital amaurosis type 2 (LCA with RPE65 mutations) phase I clinical trials demonstrated restoration of...

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Main Authors: Li, Xia, Li, Wensheng, Dai, Xufeng, Kong, Fansheng, Zheng, Qinxiang, Zhou, Xiangtian, Lü, Fan, Chang, Bo, Rohrer, Bärbel, Hauswirth, William. W., Qu, Jia, Pang, Ji-jing
Format: Artigo
Jezik:Inglês
Izdano: Association for Research in Vision and Ophthalmology, Inc. 2011
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Articles
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC3053305/
https://ncbi.nlm.nih.gov/pubmed/21169527
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1167/iovs.10-6138
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