上海交通大学医学院附属新华医院一名6岁女童在接受体细胞碱基编辑治疗后于一周内死亡[1]。该患儿患有Snijders Blok–Campeau综合征[1]，这是一种全球仅有数百名确诊患者的罕见疾病[1]。事件发生于2025年3月24日[1]，后经国际期刊《Science》的调查而在2026年7月26日公开[1]。
这一死亡事件引发了关于实验性基因治疗的广泛讨论，涉及研究设计、临床前证据、伦理审查和不良事件报告等多个环节[1]。上海交通大学医学院已成立专项工作组对此进行全面调查[1]。该事件也唤起了对1999年Jesse Gelsinger在腺病毒基因治疗临床试验中死亡的历史回顾[1]。
A six-year-old girl died within one week after receiving experimental somatic cell base editing therapy at Xinhua Hospital affiliated with Shanghai Jiao Tong University School of Medicine [1]. The child was being treated for Snijders Blok–Campeau syndrome, a rare genetic disorder affecting only several hundred confirmed patients globally [1].
The fatal incident occurred on March 24, 2025, but remained undisclosed until an investigation by the international journal Science brought the case to public attention on July 26, 2026 [1]. The incident has sparked widespread debate regarding research design, preclinical evidence, ethical review procedures, and adverse event reporting protocols [1]. Shanghai Jiao Tong University School of Medicine has established a special task force to conduct a comprehensive investigation into the matter [1].
This case draws comparison to the 1999 death of Jesse Gelsinger, who died during an adenoviral gene therapy clinical trial, highlighting ongoing concerns about safety in gene therapy research [1].
