A six-year-old girl died just seven days after receiving an experimental gene-editing treatment. Her death was not publicly disclosed by the researchers for more than a year.
In March 2025, a girl identified by the pseudonym “Mei” underwent a pioneering brain-directed gene-editing procedure at Xinhua Hospital in Shanghai, China.
Mei had Snijders Blok-Campeau syndrome, a rare genetic disorder affecting brain development. Her parents contributed approximately $860,000 toward developing a personalized treatment they hoped would improve her life.
The procedure involved injecting trillions of modified viruses carrying gene-editing instructions into her spinal fluid. But instead of helping, the treatment triggered a severe immune reaction. Mei developed kidney failure and dangerously low platelet levels before dying seven days later.
A hospital ethics committee subsequently determined that her death was definitely related to the treatment.
An investigation by Science and Retraction Watch revealed that the researchers later published related preclinical findings in Nature without disclosing the child’s death or her family’s financial contributions. Animal testing had also revealed serious liver damage.
The revelations prompted investigations by Shanghai Jiao Tong University School of Medicine and the journal Nature, raising major concerns about informed consent, clinical trial oversight, and scientific transparency.
Gene editing holds extraordinary promise for treating genetic diseases. But this tragedy demonstrates why experimental treatments must undergo rigorous safety evaluations, particularly when children are involved.
Sources: Science & Retraction Watch (July 23, 2026); CNN (July 30, 2026).