Investigation Launched After 6-Year-Old Dies Following Gene Editing Therapy
A six-year-old girl tragically lost her life last year after participating in an experimental gene-editing therapy. Questions are now being raised about the neuroscientist overseeing the research, Zilong Qiu, particularly regarding his choice not to make the death public.
Science Magazine, along with Retraction Watch, first reported the story last week. On Monday, AFP highlighted that the Shanghai Jiao Tong University School of Medicine, where Qiu is employed, has initiated a formal investigation into the situation. The girl’s family had reportedly invested over $800,000 to fund the trial, which aimed to address her rare genetic disorder.
The university emphasized its commitment to research integrity and ethical standards, underscoring that it opposes any medical research that breaches scientific ethics.
Hope Amid Experimental Failures
The girl, known by the pseudonym Mei, was born with Snijders Blok-Campeau syndrome, a condition identified in 2013 due to mutations in the CHD3 gene. Symptoms can vary but often involve intellectual challenges and a higher likelihood of seizures and heart issues. Although Mei’s case was relatively mild, she was beginning to struggle in school and faced the reality of possibly never being able to live independently.
The family learned about Qiu through a private WeChat group for parents of children with developmental disorders. After reaching out to him, Mei’s father quickly received a hopeful response from Qiu, suggesting that he could help.
The team sought to correct the specific mutation causing Mei’s condition, using two viruses to deliver a type of gene therapy called base editing. This method aimed to directly edit cell DNA within Mei’s brain, marking a first for human procedures. To maximize the therapy’s effectiveness, they injected hundreds of trillions of viruses into her spinal fluid.
Sadly, Mei passed away just a week after the treatment in late March 2025. Reports suggest that her death was linked to a severe immune reaction to the therapy. Despite the local health department imposing a minor fine on the hospital for inadequate oversight of the trial, Qiu has seemingly faced no penalties.
Following the incident, Qiu’s team published a study in Nature detailing their therapy, even after the family requested its withdrawal, to which Qiu had initially agreed. Notably, the published study omits any mention of the human trial, and it appears that gene sequences related to Mei and her family, previously included, were removed from the paper. The only reference to her death is a line about the challenges of bridging preclinical research and clinical applications.
Looking Ahead
Mei’s death comes several years after the He Jiankui case, in which embryos were illegally altered to resist HIV. Following that scandal, stricter regulations were established for human research in China.
Various researchers and bioethicists have expressed concerns that Qiu’s team may have downplayed risks to Mei’s family and overlooked safety issues. They’ve called on Nature to reevaluate the study due to worries regarding the credibility of the animal data used to justify advancing Mei’s treatment, along with the lack of transparency about the family’s financial support.
One researcher remarked that this trial should not have proceeded at all. Nature stated it was unaware of the issues surrounding the study prior to its release. An inquiry was made to Nature for comments on their intent to review the paper, but no response was received at the time of this report.
In response, Shanghai Jiao Tong University has established a working group to thoroughly investigate the situation.
