Science: Little girl dies in China as a result of experimental gene therapy

A little girl died in a Chinese hospital last spring as a result of experimental gene therapy, reports the journal Science.

The child’s death was hushed up and not mentioned in a research article published in the journal Nature this year, in which the developers of the treatment present the favorable results of previous animal experiments.

Science has investigated the course of events together with the maintainers of the Retraction Watch database.

According to experts interviewed by the journal, the animal study was incomplete and showed red flags that should not have been given to humans.

The six-year-old girl had Snijders Blok-Campeau syndrome, a very rare brain development disorder caused by a genetic defect.

Her parents funded neuroscientist Zilong Qiu’s research into gene therapy using a new precise base editing technique with more than 700,000 euros in order to help their mildly mentally retarded daughter develop normally.

After animal experiments, the treatment was started, and the renowned Xinhua University Hospital in Shanghai gave permission for it.

Billions of AAV viruses were injected into the girl’s spinal fluid, which were tasked with carrying the gene editors packaged in them to the girl’s brain to correct the genetic defect.

Viruses are commonly used as carriers because they seek out cells. Usually, viruses are injected into the bloodstream rather than the spinal cord. However, in the blood, they do not want to pass through the blood-brain barrier that protects the brain.

It was known that a large dose of viruses can produce a dangerously strong immune response in the body. It can cause, for example, excessive blood clotting. In previous animal experiments, the treatment caused liver and kidney damage in monkeys.

These risks were explained in the consent form signed by the parents, but according to the parents, they were never clearly told that the child could die as a result.

The parents recorded their conversations with the researchers and doctors, so the magazine was able to verify the content of the conversations from the recordings.

Three days after the treatment, the girl developed a fever. She did not urinate once after the treatment, which indicated kidney damage. Seven days after the treatment, the child died despite intensive care.

The hospital’s ethics committee stated that the child’s death was definitely related to the treatment she received. The cause of death was determined to be thrombotic microangiopathy, which means blockage and damage to small blood vessels.

The parents demanded that Nature retract the research article published earlier this year so that other children do not suffer the same fate as their daughter.

The research article does not include information about their daughter’s case. It only presents the positive results of animal experiments.

The results were enthusiastically received in the research community, and according to the parents, some families also began planning to contact Qiu on a social media group.

Nature has said that the journal’s editorial board was unaware of the circumstances surrounding the death before publishing the article.

Scientists and ethicists interviewed by the journal criticize China’s system for lax regulation of experimental treatments and a lack of transparency.

Shanghai Jiao Tong University has announced that it will launch an investigation into the case and will take seriously the information presented by the journal about the activities of its researcher Zilong Qiu and the Xinhua Hospital affiliated with the university.

China tightened its research ethics rules after He Jiankui edited the genomes of embryos in 2018, implanted them in the womb, and produced two genetically modified children without permission and despite the risks. They were sentenced to three years in prison.

The edits made to the genes of the embryos are still passed on to the next generation.

Correction 29.7. 2026 at 3:30 PM: The treatment did not use adenoviruses but AAV viruses (adeno-associated virus).