Treatment of SMA type 1 infants using a single-dose AAV9-mediated gene therapy via intrathecal injection of GC101: An open-label, single-arm study

二维码有效期 120s

作者 Ma Xiuwei ^[1] Zhuang Lu ^[1] Ma Wenhao ^[2] Li Jun ^[3] Wang Xiaodong ^[4] Li Zhongqiu ^[3] Jiang Xinyang ^[3] Wang Yongxia ^[1] Du Ying ^[4] Zhang Yingqian ^[4] He Fang ^[1] Zhu Zhiming ^[4] Du Shaopeng ^[1] Xu Juan ^[5] Gu Ruijie ^[1] Zhang Yanping ^[3] Zhang Shan ^[6] Li Ting ^[1] Yang Xiao ^[3] Zhang Sheng ^[3] Zhu Lina ^[3] Li Qiuping ^[6] Dong Xiaoyan ^[4] Wu Xiaobing ^[2] Feng Zhichun ^[1] 学术成果认领

作者单位 Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China ^[1] Research and Development Division, Genecradle Therapeutics Inc., Beijing 100176, China ^[2] National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China ^[3] Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China ^[4] Department of Pharmacy, Medical Supplies Center of Chinese PLA General Hospital, Beijing 100700, China ^[5] Beijing Key Laboratory of Pediatric Organ Failure, Beijing 100700, China ^[6]

栏目名称 Rapid Report

DOI 10.1097/CM9.0000000000003210

发布时间 2025-03-04

基金项目

E-Town Cooperation & Development Foundation

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中华医学杂志英文版

2024年137卷16期

1976-1978页

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Treatment of SMA type 1 infants using a single-dose AAV9-mediated gene therapy via intrathecal injection of GC101: An open-label, single-arm study

Treatment of SMA type 1 infants using a single-dose AAV9-mediated gene therapy via intrathecal injection of GC101: An open-label, single-arm study

中华医学杂志英文版

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中华医学杂志英文版

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Treatment of SMA type 1 infants using a single-dose AAV9-mediated gene therapy via intrathecal injection of GC101: An open-label, single-arm study

Treatment of SMA type 1 infants using a single-dose AAV9-mediated gene therapy via intrathecal injection of GC101: An open-label, single-arm study

中华医学杂志英文版

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中华医学杂志英文版

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