A 6-year-old Chinese girl with a rare genetic disorder died within about a week after receiving experimental gene-editing treatment in 2025. The case was recently revealed by the international academic journal Science and academic integrity platform Retraction Watch, sparking widespread concern among the medical community and the public.
Reports said the girl suffered from the rare Snijders Blok-Campeau syndrome. The research team used base-editing technology and injected a large amount of viral vectors into her spinal cord in an attempt to repair the disease-causing gene. However, days after the treatment, she developed serious complications including fever, a sharp drop in platelet levels and kidney dysfunction. She eventually died from thrombotic microangiopathy and an immune response. The hospital classified the incident as treatment-related but had not proactively disclosed the case.
The controversy surrounding the incident is not only due to the participant’s death but also involves several research ethics concerns. According to Science, the research team had already identified safety warning signs, including liver and kidney damage, during earlier primate studies, but the relevant information was reportedly not submitted for ethical review. The informed consent documents signed by the family also allegedly failed to clearly state the potentially fatal risks.
In addition, when the research team later published animal study results in Nature, they did not disclose that human trials had already been conducted or that a participant had died. They also failed to reveal that the family had provided around US$860,000 in research funding, raising concerns over conflicts of interest and insufficient information disclosure.
After the case came to light, Shanghai Jiao Tong University School of Medicine announced that it had established a special investigation team to conduct a comprehensive review. The Yangpu District Health Commission in Shanghai has also become involved in the follow-up investigation.
Commentary:
The entire research process has raised questions over whether a child was effectively used as a subject in a high-risk experiment. Safety concerns had already emerged during animal testing, yet the research proceeded to human trials. The family was also reportedly not fully informed of the potentially fatal risks, suggesting that participant safety may not have been given the highest priority.
The purpose of medical research is to advance science and find new treatments for patients. However, all breakthroughs must be built on strict research ethics and informed consent. This is especially important when participants are children who cannot make decisions independently, requiring the highest standards of protection for their rights and safety.
Only through more transparent information disclosure, stricter ethical reviews, and stronger accountability systems can medical innovation progress while maintaining the fundamental principles of protecting life and respecting human rights.