上海交通大学医学院附属新华医院于2025年3月24日对6岁女童小美实施了全球首例脑部碱基编辑治疗试验[1]。仅七天后，患者因严重免疫反应——血栓性微血管病变——于3月31日死亡[1]。医院伦理委员会随后认定患者死亡"肯定与治疗有关"[1]。患者小美患有Snijders Blok-Campeau综合征，这是一种极其罕见的疾病，全球已知患者约237人[1]。为了这次试验，患者家属自筹近600万元承担治疗费用[1]。
该事件暴露出试验方案存在多项严重缺陷。灵长类动物毒理测试数据显示，4只参与试验的猴子均出现中度至重度肝损伤，其中一只还出现肾损伤[1]。多位专家指出，研究团队对试验风险过度乐观，临床前安全性评估不足，对灵长类动物的毒理数据存在忽视[1]。2026年2月，仇子龙团队在《自然》杂志发表了相关论文，但未在论文中披露患者死亡的情况[1]。
事件曝光后，相关论文遭到质疑。7月26日，上海交通大学医学院宣布成立专项工作组进行调查[1]。7月29日，《自然》杂志在论文页面增加编辑注记，提示论文数据受质疑[1]。7月30日，该项目在国际临床试验登记平台宣布终止招募新患者[1]。
Shanghai Jiao Tong University School of Medicine's affiliated Xinhua Hospital conducted the world's first base editing therapy trial on a six-year-old girl's brain in March 2025, but the experimental treatment resulted in fatal complications within days.[1] The child, identified by the pseudonym Xiaomei, underwent the procedure on March 24, 2025, and died on March 31, 2025, from thrombotic microangiopathy, a severe immune response.[1] The hospital's ethics committee determined that the death was "definitely related" to the treatment.[1]
The girl suffered from Snijders Blok-Campeau syndrome, a rare condition affecting approximately 237 known patients worldwide.[1] Her family self-funded nearly 6 million yuan for the treatment costs.[1] Preclinical animal testing revealed concerning results: all four primates tested experienced moderate to severe liver damage, with one also developing kidney injury.[1] Despite these findings, the trial proceeded with inadequate risk assessment and insufficient preliminary safety evaluation.[1]
The trial's credibility deteriorated significantly after disclosure of the death. A paper published in Nature in February 2026 by the research team led by Qiu Zilong failed to disclose the fatality, drawing criticism from the scientific community.[1] On July 29, the journal added an editorial note questioning the paper's data integrity.[1] The project announced recruitment termination on the international clinical trials registry on July 30, 2025.[1] Shanghai Jiao Tong University School of Medicine established a special task force on July 26 to investigate the incident.[1] Experts have identified multiple deficiencies in the trial protocol, including excessive optimism about safety, insufficient preclinical evaluation, and disregard for primate toxicology data.[1]
