An experimental gene therapy trial involving trillions of engineered viruses delivered directly to the spine has resulted in the death of a six-year-old child known by the pseudonym “Mei,” according to an investigation published jointly by Science and Retraction Watch.
The Experimental Procedure and Clinical Trial Oversight
Mei suffered from the Snijders-Blok-Campeau syndrome, a rare genetic disorder impacting approximately 237 people globally. Most individuals diagnosed with the condition experience typical life expectancies alongside intellectual disabilities and macrocephaly. At six years old, Mei communicated in simple phrases and used training chopsticks, according to the investigative report. Neuroscientist Zilong Qiu led the research project, which aimed to execute the world’s first brain-directed gene editing therapy by altering mutated genes within neurons to restore vital protein production.
Parents of the child contributed more than 800 thousand dollars, equivalent to approximately 703 thousand euros, to finance the trial. However, the joint investigation by Science and Retraction Watch found that these families did not receive adequate information regarding the associated medical risks. Approximately one week following the spinal infusion of trillions of viruses engineered to penetrate the brain, Mei died.
Did you know? Snijders-Blok-Campeau syndrome is an extremely rare genetic condition.
Institutional Response and Investigation in Shanghai
Following the procedure and subsequent death, principal investigator Zilong Qiu published a related animal study in the journal Nature, omitting references to Mei. In response to the revelations published by Science and Retraction Watch, the Shanghai Jiao Tong University School of Medicine issued a public statement addressing the controversy.
The institution stated that it “attributes great importance to the matter and has created a special working group to conduct a comprehensive investigation into the incident.” The medical school added that it “always attached great importance to the integrity and norms of scientific research and opposes firmly the conduct of medical scientific research that violates scientific ethics.” University officials confirmed that serious measures would follow the conclusion of the inquiry.
Broader Implications for the Global Biotech Sector
The unreported death serves as a significant setback for China’s ambitions to compete directly with the United States as a leading biotechnology superpower. This event echoes the 2018 scientific scandal involving biofísico He Jiankui, who produced genetically modified babies in secret.
Frequently Asked Questions
What was the specific diagnosis of the patient in the trial?
The patient, referred to as Mei, was diagnosed with Snijders-Blok-Campeau syndrome, a rare genetic condition affecting roughly 237 people worldwide that causes intellectual disability and delayed cognitive development.
How was the experimental treatment administered?
According to the investigation, the treatment involved a spinal infusion of trillions of engineered viruses designed to penetrate the brain and rewrite mutated genes in neurons.
What actions have university officials taken regarding the incident?
The Shanghai Jiao Tong University School of Medicine established a special working group to execute a comprehensive investigation and promised serious measures based on the findings.
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