基因编辑临床应用中接连发生的悲剧事件引发警惕。2025年8月，辉大基因开展的杜氏肌营养不良症（DMD）基因编辑临床试验中，一名男童在接受高剂量AAV载体全身给药后死亡，被诊断为严重补体与细胞因子激活所致的急性呼吸窘迫综合征（ARDS）[1]。此外，2025年3月，一名6岁女童在上海新华医院接受脑靶向碱基编辑治疗后因血栓性微血管病（TMA）离世[1]。
这两起事件暴露了当前临床实践中的多个薄弱环节。辉大基因试验中的其余3名受试者未出现同等严重的病情，目前仍在长期随访阶段[1]。其中女童病例所反映的问题尤为突出——治疗针对非致命性疾病，患者需承担高额自费，而伦理审查形同虚设，信息披露也不充分[1]。这些流程漏洞的存在，使得受试者面临可避免的风险。
值得注意的是，这些悲剧并非基因编辑技术本身固有缺陷的证明[1]。2023年全球首个获批上市的基因编辑疗法Casgevy已在临床治疗和商业应用中取得成功[1]。当前的关键在于如何建立更严格的安全管理规范，特别是在高剂量体内递送和伦理审查领域，以确保这一前沿技术的安全推进。
Two tragic deaths in gene editing clinical trials have raised serious concerns about the safety protocols and oversight mechanisms governing experimental genetic therapies.[1] In August 2025, a young boy died following high-dose systemic administration of an AAV vector in a DMD gene editing trial conducted by Huida Gene, with autopsy findings pointing to acute respiratory distress syndrome triggered by severe complement and cytokine activation.[1] Separately, a six-year-old girl died from thrombotic microangiopathy in March 2025 after receiving brain-targeted base editing treatment at Shanghai Xinhua Hospital.[1]
While these incidents highlight critical vulnerabilities in current clinical practices, experts emphasize that the deaths do not represent fundamental flaws in gene editing technology itself.[1] The Huida Gene trial's three other remaining participants have not experienced similarly severe complications and continue under long-term observation.[1] The case involving the girl at Shanghai Xinhua Hospital exposed systemic gaps—including treatment of non-fatal diseases combined with high out-of-pocket costs, absent ethical review, and inadequate information disclosure.[1] By contrast, Casgevy, the first gene-editing therapy approved for commercial use globally in 2023, has demonstrated both clinical efficacy and commercial viability.[1] The field's trajectory since Theodore Friedman and Richard Roblin formally introduced the concept of "gene therapy" in Science magazine in 1972 underscores that responsible regulatory frameworks and rigorous ethical oversight are essential to realizing the technology's therapeutic potential.[1]
