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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.
Ma, Xiuwei; Zhuang, Lu; Ma, Wenhao; Li, Jun; Wang, Xiaodong; Li, Zhongqiu; Jiang, Xinyang; Wang, Yongxia; Du, Ying; Zhang, Yingqian; He, Fang; Zhu, Zhiming; Du, Shaopeng; Xu, Juan; Gu, Ruijie; Zhang, Yanping; Zhang, Shan; Li, Ting; Yang, Xiao; Zhang, Sheng; Zhu, Lina; Li, Qiuping; Dong, Xiaoyan; Wu, Xiaobing; Feng, Zhichun.
Afiliación
  • Ma X; Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China.
  • Zhuang L; Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China.
  • Ma W; National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China.
  • Li J; Research and Development Division, Genecradle Therapeutics Inc., Beijing 100176, China.
  • Wang X; National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China.
  • Li Z; Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China.
  • Jiang X; National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China.
  • Wang Y; National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China.
  • Du Y; Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China.
  • Zhang Y; Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China.
  • He F; Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China.
  • Zhu Z; Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China.
  • Du S; Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China.
  • Xu J; Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China.
  • Gu R; Department of Pharmacy, Medical Supplies Center of Chinese PLA General Hospital, Beijing 100700, China.
  • Zhang Y; Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China.
  • Zhang S; National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China.
  • Li T; Beijing Key Laboratory of Pediatric Organ Failure, Beijing 100700, China.
  • Yang X; Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China.
  • Zhang S; National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China.
  • Zhu L; National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China.
  • Li Q; National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China.
  • Dong X; Beijing Key Laboratory of Pediatric Organ Failure, Beijing 100700, China.
  • Wu X; Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China.
  • Feng Z; Research and Development Division, Genecradle Therapeutics Inc., Beijing 100176, China.
Chin Med J (Engl) ; 2024 Jul 09.
Article en En | MEDLINE | ID: mdl-39030070
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Texto completo: 1 Base de datos: MEDLINE Idioma: En Revista: Chin Med J (Engl) Año: 2024 Tipo del documento: Article
Texto completo
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XML
PubMed Links
Buscar en Google

Texto completo: 1 Base de datos: MEDLINE Idioma: En Revista: Chin Med J (Engl) Año: 2024 Tipo del documento: Article