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首页|期刊导航|中华医学杂志（英文版）|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

Xiuwei Ma Yingqian Zhang Fang He Zhiming Zhu Shaopeng Du Juan Xu Ruijie Gu Yanping Zhang Shan Zhang Ting Li Xiao Yang Lu Zhuang Sheng Zhang Lina Zhu Qiuping Li Xiaoyan Dong Xiaobing Wu Zhichun Feng Wenhao Ma Jun Li Xiaodong Wang Zhongqiu Li Xinyang Jiang Yongxia Wang Ying Du

中华医学杂志（英文版）2024，Vol.137Issue(16)：1976-1978,3.
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中华医学杂志（英文版）2024，Vol.137Issue(16)：1976-1978,3.DOI:10.1097/CM9.0000000000003210

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

Xiuwei Ma 1Yingqian Zhang 2Fang He 1Zhiming Zhu 2Shaopeng Du 1Juan Xu 3Ruijie Gu 1Yanping Zhang 4Shan Zhang 5Ting Li 1Xiao Yang 4Lu Zhuang 6Sheng Zhang 4Lina Zhu 4Qiuping Li 5Xiaoyan Dong 2Xiaobing Wu 7Zhichun Feng 8Wenhao Ma 7Jun Li 4Xiaodong Wang 2Zhongqiu Li 4Xinyang Jiang 4Yongxia Wang 1Ying Du2

作者信息

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

摘要

引用本文复制引用

Xiuwei Ma,Yingqian Zhang,Fang He,Zhiming Zhu,Shaopeng Du,Juan Xu,Ruijie Gu,Yanping Zhang,Shan Zhang,Ting Li,Xiao Yang,Lu Zhuang,Sheng Zhang,Lina Zhu,Qiuping Li,Xiaoyan Dong,Xiaobing Wu,Zhichun Feng,Wenhao Ma,Jun Li,Xiaodong Wang,Zhongqiu Li,Xinyang Jiang,Yongxia Wang,Ying Du..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[J].中华医学杂志（英文版）,2024,137(16):1976-1978,3.

基金项目

This project was supported by the E-Town Cooperation& Development Foundation(No.YCXJ-JZ-2022-007). （No.YCXJ-JZ-2022-007）

中华医学杂志（英文版）

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