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AAV8(Y733F)-mediated gene therapy in a Spata7 knockout mouse model of Leber congenital amaurosis and retinitis pigmentosa

Loss of SPATA7 function causes the pathogenesis of Leber congenital amaurosis and retinitis pigmentosa. Spata7 knockout mice mimic human SPATA7–related retinal disease with apparent photoreceptor degeneration observed as early as postnatal day 15 (P15). To test the efficacy of adeno-associated virus...

詳細記述

保存先:
書誌詳細
出版年:Gene Ther
主要な著者: Zhong, Hua, Eblimit, Aiden, Moayedi, Yalda, Boye, Sanford L, Chiodo, Vince A, Chen, Yiyun, Li, Yumei, Nichols, Ralph M, Hauswirth, William W, Chen, Rui, Mardon, Graeme
フォーマット: Artigo
言語:Inglês
出版事項: 2015
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC5026120/
https://ncbi.nlm.nih.gov/pubmed/25965394
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2015.42
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