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2018年美国眼科学会年会：GenSight生物技术公司展示GS010的积极数据

2018-10-14 MedSci MedSci原创

GS010线粒体视神经病变
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GenSight是一家专注于发现和开发视网膜神经退行性疾病和中枢神经系统疾病创新基因疗法的生物制药公司。GenSight生物技术公司将于2018年10月27日至30日在伊利诺伊州芝加哥举行的2018年美国眼科学会年会上，公布GS010的REVERSE III期临床试验的积极数据。

GenSight是一家专注于发现和开发视网膜神经退行性疾病和中枢神经系统疾病创新基因疗法的生物制药公司。GenSight生物技术公司将于2018年10月27日至30日在伊利诺伊州芝加哥举行的2018年美国眼科学会年会上，公布GS010的REVERSE III期临床试验的积极数据。

GS010是利用线粒体靶向序列（MTS）专有技术平台针对Leber遗传性视神经病变（LHON）的疗法，该平台使用AAV载体（腺相关病毒）特异性地解决线粒体内的缺陷，将目的基因转移到待表达的细胞中并产生功能性蛋白质，然后通过特定的核苷酸序列将其转运至线粒体，以恢复缺失或缺乏的线粒体功能。RESCUE和REVERSE是两个独立的随机、双盲的关键性III期试验，旨在评估单次玻璃体内注射GS010（rAAV2 / 2-ND4）对受LHON影响的受试者的有效性。


原始出处：

http://www.firstwordpharma.com/node/1597162#axzz5Trc0A3L7

本文系梅斯医学（MedSci）原创编译整理，转载需授权！

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    2018-11-13 江川靖瑶

    #积极数据#

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    2018-12-05 lilili1104

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    2019-07-01 xlysu

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