China investigates the death of a 6-year-old girl after a gene editing experiment

On July 26, the school confirmed AFP is investigating reports of this death and has established a specialized committee to fully review the incident. The announcement emphasized that the school always values ​​scientific integrity, resolutely opposes any medical research that violates ethics and will strictly handle relevant individuals based on the investigation results.

According to the results of a general investigation by the magazine Science and Retraction Watch – a non-profit organization specializing in monitoring withdrawn scientific works, showed that the child patient (known as Mei) died last March, just about a week after being injected into the spinal cord with a dose containing trillions of viruses. This is not a disease-causing virus, but a deactivated type used as a “transportation vehicle” to deliver the error-correcting gene into the brain to treat the child’s cognitive retardation.

Mei has Snijders Blok-Campeau syndrome, a rare genetic disorder caused by a mutation in a structural unit (base) in the DNA chain, with only about 237 cases recorded in the world. At the age of 6, he could only speak simple sentences and had difficulty moving, needing assistive devices when eating, but his condition is gradually improving thanks to speech and occupational therapy. This is a non-life-threatening syndrome, most patients have a normal lifespan, although they may have a larger head circumference and many degrees of intellectual impairment. The report does not specify why the family accepted the risks of an experimental therapy for a disease that is not life-threatening.

The project leader is neuroscientist Zilong Qiu. His goal is to create the world’s first gene editing therapy that directly affects the human brain, by correcting mutated genes right in nerve cells so that the patient’s body can produce an essential protein. It is unclear Mr. Qiu’s opinion on the incident.

Even though baby Mei died, in February this year, Mr. Qiu still published an article in a famous magazine Nature. This work only updates animal tests and does not mention incidents, only makes a general statement that shortening the gap between preclinical research (the testing phase before application on humans) and clinical application is still a big challenge.

Science and Retraction Watch reports also said that Mei’s parents spent more than 800,000 USD (about 20 billion VND) to fund the experiment, but the research team did not fully explain to them about the dangerous risks.

Theo AFPthe death was not made public, recalling the country’s most infamous gene editing scandal. In 2018, scientist He Jiankui shocked the global scientific community when he created the world’s first gene-edited babies, then received a 3-year prison sentence for practicing medicine illegally. Mr. Qiu was among more than 100 Chinese scientists who signed an open letter condemning He Jiankui at that time. “This project completely ignored the principles of medical bioethics, performing human trials without proving safety,” Mr. Qiu said in an interview at the time.