Newspaper reports indicate that a 6-year-old girl died last year after undergoing experimental gene-editing treatment, in an incident that sparked widespread controversy over the ethics of scientific research.
The girl, who published her story under the pseudonym "May," suffered from the rare "Snyders-Block-Kambu syndrome," a genetic disorder caused by a mutation in the CHD3 gene, which leads to intellectual disability and developmental abnormalities.
Although her condition was relatively average, she was facing academic difficulties, and her family feared that she would not be able to live independently in the future.
After joining a private WeChat group for parents of children with developmental disorders, the girl's father contacted Chinese scientist Qilong Qiu, a researcher at Shanghai Jiao Tong University's School of Medicine, who is working on developing gene therapies using CRISPR technology. Qiu offered to help the family, who contributed over $800,000 to fund the trial.
The treatment relied on an advanced technique known as "base editing," which aimed to correct the genetic mutation responsible for the disease by injecting hundreds of trillions of modified viruses into the girl's spinal fluid, in an attempt to directly target her brain cells—a step that had never been done on humans before.
But the girl died just one week after undergoing the treatment in March 2025, and experts believe the cause of death was a severe immune reaction to the viruses used.
Although the hospital where the operation was performed was fined only $3,600 by local health authorities for inadequate supervision, Chiu did not initially face any administrative or legal penalties.
The case sparked further controversy when Chiu and his team proceeded to publish a scientific study on the treatment in the prestigious journal Nature last February, without any mention of the human trial or the girl's death. They also removed the girl's and her family's genetic sequences from the initial draft of the study, as well as a sentence acknowledging their financial contribution to the research.
The study contained only a general statement indicating that "bridging the gap between preclinical research and clinical application remains a major challenge."
After Science magazine and Retraction Watch revealed details of the case last week, Shanghai Jiao Tong University School of Medicine announced last Monday that it had opened an official investigation into the incident, stressing in a statement that it "attaches great importance to scientific integrity and strongly opposes any research that violates the ethics of science."
Researchers and bioethicists expressed their dismay at what they described as a "minimization of the risks" to the girl's family, and called on Nature magazine to re-examine the study, citing several concerns about the quality of the animal data the team relied on to justify the experiment, and their concealment of the research funding source.
Stephen Gray, a gene therapy researcher at the University of Texas, said, "This experiment should never have reached the stage of being applied to humans." Nature stated that it was unaware of these issues prior to publication and has not yet responded to inquiries about its plans to review the paper.
This incident comes five years after the scandal involving Chinese scientist He Jiankui, who was sentenced to prison for illegally editing the genes of human embryos, prompting China to tighten its laws on human research. The ongoing investigations are expected to yield further developments in this sensitive case.
