A six-year-old Chinese girl, identified by the pseudonym "Mei," died roughly one week after receiving an experimental brain-targeted gene-editing therapy at Xinhua Hospital, affiliated with Shanghai Jiao Tong University School of Medicine, in March 2025 [1, 2, 3, 4, 5, 6, 7, 8]. The treatment aimed to repair a mutation in the CHD3 gene responsible for Snijders Blok-Campeau syndrome, a rare condition causing mild cognitive and developmental delays but generally not life-threatening [3, 5, 6, 7, 8].
The therapy involved injecting trillions of viruses carrying base editing tools into the spinal canal to correct the genetic mutation and restore normal protein function in neurons [1, 3, 5, 6, 7, 8]. Shortly after the procedure, the girl developed fever, severe kidney damage, thrombocytopenia, and was admitted to the intensive care unit. She died about seven days later due to thrombotic microangiopathy and a severe immune reaction linked to the gene-editing treatment, as determined by an internal hospital investigation [1, 3, 5, 6, 7, 8].
The deceased child's family provided approximately $800,000 to $860,000 USD to fund the therapy. However, these financial details and potential risks were not adequately disclosed in published research papers [1, 3, 5, 6, 7, 8]. The lead scientist overseeing the trial was neuroscientist Zilong Qiu from Shanghai Jiao Tong University's Songjiang research institute [1, 3, 5, 6, 7, 8]. In February 2026, Qiu's team published an animal study in Nature related to the therapy but omitted any mention of the earlier human trial or the child's death [1, 2, 3, 4, 5, 6, 7, 8].
Seven independent experts reviewing the case strongly criticized the research team for underestimating risks and proceeding despite low chances of success and alarming toxicity findings in animal models [1, 3, 6, 7, 8]. Ethical concerns have been raised about administering high-risk gene-editing interventions to children with a non-lethal condition and the adequacy of informed consent [3, 6, 7, 8]. Steven Gray, a gene therapy expert at the University of Texas Southwestern Medical Center, said, "这项试验本来就不应该进行。" (translated: "This trial should never have been conducted.") [3].
Shanghai Jiao Tong University School of Medicine announced the formation of a special task force on July 26, 2026, to conduct a full investigation into the incident [2, 3, 4, 7]. The Shanghai Yangpu District Health Commission confirmed a parallel inquiry starting in late July 2026 [4]. Earlier, in September 2025, Shanghai authorities fined Xinhua Hospital approximately $3,600 USD (24,000 RMB) for regulatory violations related to the gene-editing trial [1, 3].
The therapy was administered on March 24, 2025, and the girl died on March 31, 2025, about seven days after treatment [3, 6, 8]. An investigative report jointly published by Science and Retraction Watch on July 23, 2026, exposed the undisclosed risks and the child's death [1, 3, 6, 7, 8]. The investigation continues with findings pending.
