Fatal Experiment: Girl Dies from Experimental Gene Therapy in the Brain

Parents in China paid millions of yuan for an experimental gene therapy in the brain, hoping to cure their daughter of a rare developmental disorder. The treatment, never tested on humans, led to the tragic death of the child. The lead researcher published a paper in Nature without mentioning the case.

Parents in China paid millions of yuan (about $860,000) to leading researchers for an experimental gene therapy in the brain, never tested on humans, to cure their daughter Mei, who suffered from a rare genetic developmental disorder (CHD3 gene mutation). The treatment, injected directly into the spinal canal, contained hundreds of trillions of viruses. Within days, Mei developed fever and kidney failure, and died the next day. The ethics committee determined the cause of death was thrombotic microangiopathy – a complication also seen in previously treated monkeys. The lead researcher, Professor Zilong Qiu, published a paper in Nature months after her death without mentioning the case. Shanghai Jiao Tong University announced an investigation. The report in Science raises serious questions about research oversight and exploitation of desperate families.