换一批
换一批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
作者：
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]
期刊：
《中华医学杂志英文版》2024年137卷16期 1976-1978页
SCIMEDLINEISTICCSCDBP
栏目名称：
Rapid Report
DOI：
10.1097/CM9.0000000000003210
发布时间：
2024-09-10
基金项目：
E-Town Cooperation & Development Foundation
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