一名6岁女童小美于2025年3月在上海交通大学医学院附属新华医院接受基因治疗后，在短短一周内因严重免疫反应不幸去世[1]。小美患有斯奈德斯·布洛克-坎波综合征，这是一种全球仅有236例确诊病例的罕见病[1]。治疗后第三天，她出现发热症状，随即伴随严重肾功能损伤[1]。
该基因治疗项目的牵头人仇子龙团队于2026年2月在《自然》杂志发表了相关动物研究论文，但论文中未披露人体试验情况及患者死亡事件[1]。在录音中，仇子龙曾表示采用AAV鞘内注射方式免疫原性较小、风险较低[1]。小美家属对此投诉并申请撤回论文[1]。此外，新华医院伦理委员会曾要求提交猴子病理学报告，但未见后续提交记录[1]。
上海交大医学院于2026年7月26日发布情况说明并成立专项工作组开展全面调查[1]。同日，《自然》杂志在论文页面标记编辑关注[1]。上海市卫健委也随后成立调查组介入[1]。小美于2023年3月确诊该罕见病，当时她仅4岁2个月[1]。
A six-year-old girl named Xiaomei underwent gene therapy at Shanghai Xinhua Hospital in March 2025 and died within a week due to severe immune complications [1]. Three days after receiving the treatment, Xiaomei developed fever accompanied by acute kidney dysfunction [1]. The child had been diagnosed with Snijders-Blok-Campeau Syndrome (SNIBCPS) in March 2023 at age four years and two months, a rare condition affecting approximately 236 known patients worldwide [1].
The research team led by Qiu Zilong published a paper in Nature magazine in February 2026 related to animal studies, but the publication did not disclose the human trial or the patient's death [1]. Following Xiaomei's family's complaint and request for retraction, Shanghai Jiao Tong University School of Medicine and Shanghai Municipal Health Commission each established investigation teams to conduct comprehensive inquiries [1]. On July 26, 2026, Shanghai Jiao Tong University School of Medicine released a statement establishing a special working group for investigation [1]. The journal Nature flagged the paper with an editorial notice on its page as of July 29, 2026 [1].
Prior to the treatment, the ethics committee at Xinhua Hospital had requested submission of pathology reports from monkey studies, though no subsequent submission records were documented [1]. In recorded audio, Qiu Zilong stated that intrathecal AAV injection carries low immunogenicity and presents lower risk [1].
