Chinese university probes girl’s death in gene-editing trial

A Chinese university has launched an investigation into the death of a six-year-old girl following an experimental gene-editing treatment, a fatality that was never publicly disclosed in the researchers' published work.

Shanghai Jiao Tong University School of Medicine said on Sunday that it "attaches great importance to the matter and has set up a special working group to conduct a comprehensive investigation into the incident," according to Channel News Asia.channelnewsasia

What Happened

The case, revealed through a joint investigation by Science magazine and Retraction Watch published on July 23, details how the girl — given the pseudonym "Mei" — received what is described as the world's first brain-directed CRISPR base-editing therapy at Shanghai Xinhua Hospital in March 2025.bloomberg+1

Mei had Snijders Blok-Campeau syndrome, a rare genetic disorder caused by mutations in the CHD3 gene that led to mild cognitive impairment. The treatment involved a spinal infusion of trillions of viruses designed to penetrate the brain and correct the underlying genetic defect.livescience+2

Seven days after the infusion, Mei died from a severe immune reaction. The hospital determined her death was caused by thrombotic microangiopathy — an immune response that causes blood clots and can arise from virus-based gene therapies. The condition she was being treated for was not life-threatening.science+3

Concealment Raises Alarm

The investigation found that the trial had not disclosed the fatality or animal safety problems in its published research. Mei's parents have called for accountability, according to Science.ground+3

The case has raised broader questions about regulatory oversight and transparency as China rapidly expands its biotechnology sector. It also evokes memories of the 2018 controversy surrounding He Jiankui, the Chinese scientist who created the world's first gene-edited babies and was subsequently imprisoned.straitstimes+1

Global Implications

The death carries implications for the broader gene therapy field, which has seen rapid advances in recent years. The use of viral vectors to deliver gene-editing tools to the brain remains an area of active research worldwide, and adverse immune reactions remain among the most serious risks associated with such approaches. The case underscores the stakes involved when experimental treatments are applied to non-life-threatening conditions, particularly in pediatric patients.