On Thursday, July 23, the international top academic journal “Science” jointly with the academic supervision website “Retraction Watch” released two blockbuster investigations, uncovering a case of a 6-year-old Chinese girl who died due to concealed gene editing therapy.
The report quickly shocked the global scientific community. Several foreign gene therapy experts and bioethicists who reviewed the case questioned whether the Chinese doctors fully informed the girl’s parents about the risks of the treatment.
In March 2025, at the Xinhua Hospital affiliated with the Shanghai Jiao Tong University School of Medicine, Chinese doctors and scientists attempted to help a 6-year-old girl with abnormal brain development caused by a gene mutation. They injected billions of viruses into her cerebrospinal fluid, carrying instructions to assemble a customized gene repair system called an “alkaline base editor” to correct the defective genes in her brain cells.
The joint investigation revealed that the girl, known as “Xiao Mei,” was the first known person globally to receive brain gene editing therapy, and she was the subject of a clinical trial with only one patient.
The hospital received 860,000 US dollars (approximately 5.82 million RMB) from the girl’s family as partial funding for the development of this therapy.
According to the report, Xiao Mei’s initial condition was unlikely to be fatal but could potentially lead to lifelong disabilities and severe health problems. Therefore, her parents sought help from the Institute for Pediatric Translational Medicine (IPTM) at the Shanghai Jiao Tong University School of Medicine in Songjiang. The team showed them evidence of the effectiveness of the developed alkaline base editor in the brains of mice and monkeys. After the doctors reassured the parents that the risks were minimal, they decided to proceed.
After receiving the treatment, Xiao Mei developed a fever and signs of kidney damage. Sadly, she passed away a week after receiving the treatment.
The hospital’s report concluded that the most likely cause of death was an immune reaction in the patient to the viruses used to deliver the gene editing vectors.
Almost a year after Xiao Mei’s passing, the IPTM team who developed her therapy published a paper in the journal “Nature” in February 2026, providing evidence of the therapy’s effectiveness in mice and monkeys. Although the draft manuscript mentioned the girl’s family genes and acknowledged her parents’ support for the research, these details were ultimately removed from the published paper.
Before the paper’s publication, Xiao Mei’s parents requested the research team to retract the paper and later wrote to the journal “Nature” to express their protest, mentioning their financial support for the trial and their daughter’s passing.
The journal replied that these ethical issues were “beyond the scope of our data integrity” and should be handled by the Chinese institutions conducting the research.
An editor at “Nature” told investigators from “Science” that during the manuscript review process, the journal was unaware of Xiao Mei’s death or related issues, such as the hospital’s penalties for inadequate supervision.
Now, Xiao Mei’s parents seek accountability from those involved.
“We didn’t realize how uncommon and dangerous many of the arrangements were at the time,” they told investigators from “Science.”
Seven foreign gene therapy experts and bioethicists who analyzed the case collectively questioned whether Xiao Mei’s parents were adequately informed of the therapy’s risks and why the trial progressed in a situation with very low success probability.
Steven Gray, an expert in viral vectors at the University of Texas Southwestern Medical Center, bluntly stated that this human trial “should not have been conducted at all.”
Furthermore, experts also questioned whether the therapy was as effective in animals as implied in the paper published in “Nature,” why the paper’s authors did not disclose Xiao Mei’s cause of death and the primate studies that raised safety concerns, and whether the publishing journal is responsible for updating or retracting the paper.
Xiao Mei’s parents expressed their desire to protect other gene therapy patients from similar harm.
After Xiao Mei’s passing, one of the Chinese scientists involved in the trial refunded the family over 100,000 US dollars they had received earlier.
Observers believe that this incident has exposed more than just an issue within a Chinese research team; it reflects a serious test of China’s scientific integrity, medical ethics, and patient rights.
The investigation by “Science” also revealed some shocking content. The report stated that throughout the treatment process, the child’s family not only bore significant costs but also had multiple communications with the research team on matters such as virus preparation and experiment progress.
The report also disclosed a recorded conversation where one of the paper’s authors, Kan Yang, explained to the girl’s father, “Without subsequent data analysis and monkey experiments, these virus injections would be meaningless.”
Simultaneously, there were lengthy discussions between both sides regarding the experimental costs. According to the investigation by “Science,” the child’s family felt severely misled throughout the process and raised strong doubts about cost arrangements, information disclosure, and the trial’s progression.
In this tragedy, was the patient receiving treatment or merely a “resource” in a research project? If patients are exposed to life-threatening risks and deliberately concealed in the final paper by researchers, where is the essence of medicine?
Following Xiao Mei’s death at the age of 6, the Xinhua Hospital paid a relatively small fine to the local health department, but Qiu Zilong did not face public punishment.
The investigative report by “Science” magazine stated that this disturbing death marks a new setback for Beijing in its efforts to compete with the United States for bioengineering dominance.
Three years ago, the Chinese government vowed to strengthen biomedical research oversight in response to the 2018 ethical scandal involving He Jiankui, a biophysicist who covertly created gene-edited babies.
For more information, please refer to the report titled “Shanghai Girl Dies After Gene Editing Experiment Exposed Over a Year Later.”