Six-Year-Old Dies After Experimental Brain Gene-Editing Trial in China
A six-year-old girl identified as May died a week after receiving an experimental brain gene-editing treatment at Xinhua Hospital in Shanghai. The first-in-human trial used a base-editing therapy intended to correct a CHD3 gene mutation linked to a rare developmental disorder that was not considered life-threatening. The treatment delivered extremely high doses of two modified viruses into her spinal fluid because both had to reach the same brain cells. May developed fever, kidney failure and a severe drop in platelets. The hospital’s ethics committee concluded that the therapy directly caused thrombotic microangiopathy, a condition involving dangerous small-vessel blood clots and organ damage. Investigators reported that animal tests had already shown liver and kidney injury, while the final toxicity report was not completed before the human trial was approved. May’s parents, who reportedly paid about $860,000 towards the research, say they were not clearly told that the treatment could be fatal. The case has renewed concerns about informed consent, trial transparency and the risks of gene editing for non-life-threatening conditions.
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