Death of girl during gene-editing clinical trial raises ethical concerns

The death of a 6-year-old girl allegedly linked to an experimental gene-editing clinical trial in Shanghai has sparked widespread concern over the safety and ethical oversight of medical research involving human beings.

The case came to light on July 23, when the journal Science and academic watchdog Retraction Watch jointly published an investigative report revealing that the child received an experimental brain-directed gene-editing therapy at the Xinhua Hospital Affiliated To Shanghai Jiao Tong University School of Medicine on March 24, 2025, and died within a week from thrombotic microangiopathy — a rare, potentially life-threatening condition in which tiny blood clots form in small blood vessels, causing damage to organs such as the kidneys and brain.

The girl had Snijders Blok-Campeau syndrome, a rare neurodevelopmental disorder caused by mutations in the CHD3 gene that lead to developmental delays, reduced muscle tone and speech difficulties.

However, her condition was not life-threatening. She attended kindergarten, received speech and occupational therapy, and had special education support. Seeking better treatment options, her parents approached a research team led by Qiu Zilong, a neuroscientist at the Shanghai Jiao Tong University School of Medicine.

Researchers claimed the trial was the world's first human test of its kind, with the girl as its only participant. According to the investigative report, the published research failed to disclose liver and kidney damage observed during prior safety studies in nonhuman primates.

The report also said an internal ethics review at Xinhua Hospital concluded that the girl's death was "definitely related" to the treatment. However, the trial leaders did not publicly disclose her death or update the clinical trial registry.

In February, the research team published preclinical findings in the journal Nature without disclosing either the human trial or the girl's death. According to the investigative report, the paper also omitted acknowledgments to the girl's family and funding sources that had appeared in earlier drafts.

On July 26, the Shanghai Jiao Tong University School of Medicine announced that it had established a special task force to investigate the case.

"The medical school has always attached great importance to research integrity and scientific standards, and firmly opposes any medical research that violates scientific ethics," the statement said. "We will handle this matter with seriousness based on the investigation findings, and appreciate the concern and oversight from the public."

The institution also said it would conduct a comprehensive review of research papers published by Qiu as well as the clinical study carried out at Xinhua Hospital.

On July 29, Nature added an editorial note to the paper's webpage, saying concerns had been raised about the publication and its data and that "further editorial action will be taken if appropriate as soon as the investigation into the concerns is complete".

On July 30, the trial was marked as terminated on ClinicalTrials.gov, with a notation stating: "The single participant died on 31 March 2025 from thrombotic microangiopathy, a serious adverse event assessed as definitely related to the study drug."

Shi Jiayou, a professor at Renmin University of China Law School, told The Beijing News that subjecting a child with a non-life-threatening condition to known lethal risks violates the principles of minimizing harm and ensuring controllable risk under China's Measures for Ethical Review of Life Science and Medical Research Involving Human Beings.

"The goal of regulation is not to impede scientific research, but to eliminate reckless human experimentation and prevent the entire industry from stagnating because of individual malicious incidents," Shi said. "The lives and safety of research participants must always take priority over world's firsts, journal publications and academic breakthroughs."