Replacement Gene Therapy with a Human RPGRIP1 Sequence Slows Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis

RPGR-interacting protein-1 (RPGRIP1) is localized in the photoreceptor-connecting cilium, where it anchors the RPGR (retinitis pigmentosa GTPase regulator) protein, and its function is essential for photoreceptor maintenance. Genetic defect in RPGRIP1 is a known cause of Leber congenital amaurosis (...

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Hlavní autoři: Pawlyk, Basil S., Bulgakov, Oleg V., Liu, Xiaoqing, Xu, Xiaoyun, Adamian, Michael, Sun, Xun, Khani, Shahrokh C., Berson, Eliot L., Sandberg, Michael A., Li, Tiansen
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2010
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Research Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2928706/
https://ncbi.nlm.nih.gov/pubmed/20384479
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2009.218
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