首页|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

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  • 国家科技期刊平台
  • NETL
  • NSTL
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Xiuwei Ma、Lu Zhuang、Wenhao Ma、Jun Li、Xiaodong Wang、Zhongqiu Li、Xinyang Jiang、Yongxia Wang、Ying Du、Yingqian Zhang、Fang He、Zhiming Zhu、Shaopeng Du、Juan Xu、Ruijie Gu、Yanping Zhang、Shan Zhang、Ting Li、Xiao Yang、Sheng Zhang、Lina Zhu、Qiuping Li、Xiaoyan Dong、Xiaobing Wu、Zhichun Feng

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Department of Neurodevelopment,Faculty of Pediatrics,The Seventh Medical Center of Chinese PLA General Hospital,Beijing 100700,China

National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology,Beijing 100700,China

Research and Development Division,Genecradle Therapeutics Inc.,Beijing 100176,China

Medical Division,Genecradle Therapeutics Inc.,Beijing 100176,China

Department of Pharmacy,Medical Supplies Center of Chinese PLA General Hospital,Beijing 100700,China

Beijing Key Laboratory of Pediatric Organ Failure,Beijing 100700,China

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E-Town Cooperation& Development Foundation

YCXJ-JZ-2022-007

2024

10.1097/CM9.0000000000003210

中华医学杂志(英文版)
中华医学会

中华医学杂志(英文版)

CSTPCD
影响因子：0.838
ISSN：0366-6999
年,卷(期)：2024.137(16)