Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

Mei Hong Tan; Alexander J. Smith; Basil Pawlyk; Xiaoyun Xu; Xiaoqing Liu; James B. Bainbridge; Mark Basche; Jenny McIntosh; Hoai Viet Tran; Amit Nathwani; Tiansen Li

首页> 外文期刊>Human Molecular Genetics >Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

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Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

机译：因AIPL1缺陷引起的色素性视网膜炎和Leber先天性黑桃病的基因治疗：使用AAV2 / 2和AAV2 / 8载体有效挽救部分和完全Aipl1缺乏症的小鼠模型

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《Human Molecular Genetics》 |2010年第4期|p.735|共1页
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Mei Hong Tan; Alexander J. Smith; Basil Pawlyk; Xiaoyun Xu; Xiaoqing Liu; James B. Bainbridge; Mark Basche; Jenny McIntosh; Hoai Viet Tran; Amit Nathwani; Tiansen Li;
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1. Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors. [J] . Tan MH, Smith AJ, Pawlyk B Human Molecular Genetics . 2009,第12期

机译：由AIPL1缺陷引起的色素性视网膜炎和Leber先天性黑桃病的基因治疗：使用AAV2 / 2和AAV2 / 8载体有效挽救部分和完全Aipl1缺乏症的小鼠模型。
2. Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors [J] . Mei Hong Tan1 Alexander J. Smith1 Basil Pawlyk2 Xiaoyun Xu2 Xiaoqing Liu2 James B. Bainbridge1 Mark Basche1 Jenny McIntosh3 Hoai Viet Tran1 Amit Nathwani3 Tiansen Li2* and Robin R. Ali1* Human Molecular Genetics . 2009,第12期

机译：因AIPL1缺陷引起的色素性视网膜炎和Leber先天性黑桃病的基因治疗：使用AAV2 / 2和AAV2 / 8载体有效挽救部分和完全Aipl1缺乏症的小鼠模型
3. Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65 (vol 23, pg 857, 2016) [J] . Georgiadis A., Duran Y., Ribeiro J., Gene therapy . 2018,第6期

机译：由于RPE65（Vol 23，PG 857,2016）的缺陷，莱伯先天性阿颈病的优化AAV2 / 5基因治疗载体的开发
4. Analysis of Retinal Degeneration in a Leber Congenital Amaurosis Mouse Model Using High Spatial Resolution MALDI-Imaging Mass Spectrometry [C] . David M. G. Anderson, Zsolt Ablonczy, Yiannis Koutalos, ASMS Conference on Mass Spectrometry and Allied Topics . 2016

机译：利用高空间分辨率MATDI成像质谱法分析LEBER先天性生物疲劳小鼠模型的视网膜变性
5. Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors [O] . Mei Hong Tan, Alexander J. Smith, Basil Pawlyk, -1

机译：因AIPL1缺陷引起的色素性视网膜炎和Leber先天性黑桃病的基因治疗：使用AAV2 / 2和AAV2 / 8载体有效挽救部分和完全Aipl1缺乏症的小鼠模型
6. Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors [O] . Tan, Mei Hong, Smith, Alexander J., Pawlyk, Basil, 2009

机译：因AIPL1缺陷引起的色素性视网膜炎和Leber先天性黑桃病的基因治疗：使用AAV2 / 2和AAV2 / 8载体有效挽救部分和完全Aipl1缺乏症的小鼠模型

Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

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