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Girl’s death in China reveals human cost of unregulated science
The case has prompted debate about ‘first in human’ gene-editing trials, particularly for rare but serious disorders<br>Updated - July 26, 2026 03:53 pm IST
Vasudevan Mukunth
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The investigation has raised important questions about the oversight of an experimental personalised gene-editing trial and the way the researchers and their institutions communicated their choices |Image used for representational purpose only |Image used for representational purpose only<br>| Photo Credit: REUTERS
A six-year-old girl in China died in March 2025 after receiving what researchers said they believed was the world’s first base-editing therapy directed at the brain, according to an investigation by Science and Retraction Watch.<br>The investigation has raised important questions about the oversight of an experimental personalised gene-editing trial and the way the researchers and their institutions communicated their choices.<br>The girl had Snijders Blok-Campeau syndrome, a rare neurodevelopmental disorder caused by a mutation in the CHD3 gene. Although the condition affects intellectual development and its severity varies widely, most people with the condition live full lives.<br>But that such a case could still clear the bar for an unprecedented, high-risk brain intervention says something about a society uneasy with disability and enamoured of techno-scientific interventions.<br>The girl’s parents sought the experimental treatment after learning of research led by neuroscientist Zilong Qiu at the Shanghai Jiao Tong University.<br>Base-editing is a more recent invention than CRISPR-Cas9. The American biochemist David Liu developed it at the Broad Institute in 2016 after finding a way to convert one DNA letter directly into another — say, C to T — without cutting both strands of the DNA double helix, as Cas9-based editing does.<br>As a result, base-editing is, in principle, more ‘gentle’ and can cause fewer unintended mutations. Its development thus renewed hopes that gene-editing could graduate from being a proof of concept and to treating rare disorders caused by defects in single genes.<br>That said, the memory of He Jiankui, the Chinese scientist who announced in 2018 that he had edited the genomes of twin girls at the embryonic stage without adequate ethical review, scientific justification, and even disclosure to his own university, has weighed heavily on the field.<br>He was later sentenced to three years in prison for his actions, and the episode forced China to tighten its rules on human gene-editing research. But as the new investigation by Science and Retraction Watch has found, the rifts between what the law says and how it is enforced have remained wide enough for a similar failure to recur.<br>Dr. Qiu’s team developed a personalised base-editing therapy designed to correct the genetic mutation in the child’s brain cells.<br>However, delivering the ‘editor’ to the brain required the researchers to inject large doses of adeno-associated viruses (AAVs), which are known to carry risks of severe immune reactions.<br>According to the investigation report, the child’s parents helped finance much of the therapy’s development, contributing around $860,000 (Rs 8.3 crore) from their personal savings and money borrowed from relatives.<br>The treatment proceeded as a clinical trial initiated by the investigator at the Xinhua Hospital in Shanghai. This process did not require the National Medical Products Administration, China’s national drug regulator, to review it first.<br>However, the report has quoted independent experts expressing concerns with several aspects of the process — including whether preclinical studies (in animals) had adequately proved the treatment’s safety for human use.<br>Toxicology studies in monkeys reportedly found that all the treated animals had developed liver injury and one monkey’s kidneys were damaged as well.<br>However, the researchers proceeded to inject the therapy into the girl’s cerebrospinal fluid on March 24, 2025. Within days, she developed a fever and kidney damage, and died a week later.<br>An internal review at the hospital concluded that the treatment was certainly linked to her death, and listed the cause of death as thrombotic microangiopathy, a complication that has previously been associated with genetic...