The tragic death of a six-year-old Chinese girl who succumbed rapidly from an experimental treatment has come under fire after an investigation found that authorities in China allegedly concealed the case from the public while allowing research to continue.
According to a joint investigation by Science and Retraction Watch, the girl, identified by the pseudonym “Mei,” died in March 2025 after undergoing experimental “gene-editing therapy” at Shanghai’s Xinhua Hospital. The report says the fatality was never publicly disclosed, despite the treatment being seen as groundbreaking research that scientists hoped would become the world’s first gene-editing therapy targeting the human brain.
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The case has renewed debate over the ethical boundaries of experimental gene-editing research, particularly when conducted on children with non-life-threatening conditions.
A high-risk experimental treatment
According to the investigation, Mei suffered from Snijders-Blok-Campeau syndrome, an extremely rare genetic disorder caused by a mutation in the CHD3 gene. The condition primarily affects cognitive development and speech but is generally not considered life-threatening, with most patients expected to have a normal life expectancy.
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Hoping to improve their daughter’s quality of life, Mei’s parents reportedly spent more than $860,000 by drawing on personal savings and financial support from relatives to help fund the experimental treatment after learning about research led by Chinese neuroscientist Zilong Qiu.
The treatment harnessed base editing, a more precise form of CRISPR gene editing designed to correct single DNA mutations. Unlike conventional CRISPR systems, which cut both strands of DNA, base editors chemically convert one DNA base into another, offering the potential to repair disease-causing mutations without introducing double-strand breaks.
Researchers hoped the therapy would correct Mei’s genetic mutation by delivering trillions of engineered viruses into her spinal fluid, thereby allowing the gene editor to reach neurons throughout her brain. But according to hospital records reviewed during the investigation, Mei developed a severe immune reaction and died just seven days later after beginning treatment.
Concerns over oversight and accountability
The investigation raises broader concerns about whether the experimental therapy should have proceeded at all. Seven independent experts in genetics, virology, bioethics, and gene therapy who reviewed the case for Science and Retraction Watch concluded that researchers appeared to underestimate the treatment’s risks, failed to adequately account for safety concerns identified during animal testing, and proceeded despite what they viewed as a low likelihood of clinical success.
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“This shouldn’t have gone to trial,” Steven Gray, a gene therapy researcher at the University of Texas Southwestern Medical Center, told the investigators. The report also alleges that Mei’s parents were not fully informed about the potential severity of the risks before consenting to the treatment.
According to the investigation, the trial proceeded under a Chinese regulatory pathway that did not require approval from national regulators, prompting questions and concerns involving oversight and accountability of highly experimental, investigator-initiated therapies.
Death omitted from published research
The controversy deepened after investigators found that Qiu and colleagues later published related preclinical animal research in Nature without disclosing that the clinical trial had resulted in the death of its only participant. The paper stated only that “bridging the gap between preclinical research and clinical translation remains a significant challenge,” making no reference to Mei, her family, or their financial contributions to the project, Science and Retraction Watch noted.
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Several experts interviewed by the journal noted that the circumstances surrounding Mei’s case warrant a comprehensive review of the published research and underlying data. Some suggested the issues could ultimately justify retraction of the paper, though no such action has yet been taken.
According to the investigation, Mei’s parents have also requested that the paper be withdrawn. However, neither Qiu nor Shanghai’s Xinhua Hospital responded to repeated requests for comment from Science and Retraction Watch before publication. Nature also told investigators it had not been aware of the clinical events surrounding the trial before publishing the preclinical findings.
Echoes of past medical controversies
For researchers, Mei’s case is reminiscent of the 2018 scandal involving Chinese scientist He Jiankui, whose creation of the world’s first gene-edited babies exposed major gaps in China’s oversight of cutting-edge biotechnology, leading to widespread condemnation and calls for stronger ethical and regulatory safeguards. He was ultimately convicted of illegally practicing medicine and sentenced to three years in prison, along with a fine of 3 million yuan (approximately USD$430,000 at the time).
The case has also revived memories of Jesse Gelsinger, an 18-year-old American who died during an experimental gene therapy trial in 1999. His death prompted sweeping reforms to clinical trial oversight and significantly slowed the development of gene therapy for years.
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