Death of girl in Chinese gene-editing trial kept secret: Report - Medical Xpress

What the report says

Medical Xpress, citing an investigation by Science and Retraction Watch, reported that a 6-year-old girl in China died in 2025 after receiving an experimental gene-editing therapy for Snijders Blok-Campeau syndrome, a rare condition described as non-life-threatening. The child, identified by the pseudonym “Mei,” reportedly received a spinal infusion in March containing large quantities of viruses intended to carry a base-editing treatment into the brain and correct a single DNA-letter mutation.

According to the report, Mei died one week after the procedure from a severe immune reaction. Her parents had paid more than $800,000 to support the experiment, and the investigation said they were not properly warned about the risks. The work was led by neuroscientist Zilong Qiu, who was pursuing what the report described as a potential first gene-editing therapy aimed at the brain. Qiu later published related animal-study findings in Nature, but the article did not disclose Mei’s death, Medical Xpress reported.

The case has drawn scrutiny from outside experts. Seven specialists who reviewed details for Science and Retraction Watch reportedly said the research team minimized the dangers and proceeded despite limited chances of benefit. They also urged a fuller review of data connected to the Nature paper, with some concerns potentially serious enough to raise the question of retraction.

Medical Xpress framed the episode as another setback for China’s biotechnology ambitions after the 2018 scandal involving He Jiankui and gene-edited babies. More broadly, the report raises ethical questions about testing high-risk gene therapies first in children whose conditions are rare but not usually fatal, echoing concerns that followed the 1999 death of U.S. gene-therapy participant Jesse Gelsinger.