Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset
Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset
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DOI：
10.1016/j.ophtha.2020.12.012
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发表时间：
2021-04-20
期刊：
影响因子：
13.7
通讯作者：
Sahel, Jose-Alain
中科院分区：
文献类型：
--
作者：
Newman, Nancy J.;Yu-Wai-Man, Patrick;Sahel, Jose-Alain
目的：评估单次玻璃体内注射 rAAV2/2-ND4 对 Leber 遗传性视神经病变 (LHON) 导致视力丧失的受试者的疗效。设计：RESCUE 是一项多中心、随机、双盲、假对照、3 期临床试验。参与者：患有 m.11778G>A 线粒体 DNA 突变和视力丧失的受试者
Purpose: To evaluate the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects with visual loss from Leber hereditary optic neuropathy (LHON).Design: RESCUE is a multicenter, randomized, double-masked, sham-controlled, phase 3 clinical trial.Participants: Subjects with the m.11778G>A mitochondrial DNA mutation and vision loss