Washington: A six-year-old Chinese girl died last year after receiving experimental gene therapy to correct a non-life-threatening condition, according to an investigation by Science and Retraction Watch.Thursday’s report said the girl, who went by the pseudonym “Mei,” died a week after receiving a spinal infusion of trillions of viruses last March that were designed to penetrate the brain and repair the rare genetic disorder that caused her mild cognitive impairment. But her parents paid more than $800,000 for the experiment without fully understanding the risks, the report said.The lead researcher subsequently published a paper on related animal studies in Nature, which did not mention Mei, noting only that “bridging the gap between preclinical research and clinical translation remains a significant challenge.”May suffers from a rare genetic disorder called Snijders Blok-Campeau syndrome, which is known to affect only 237 people worldwide. Caused by a mutation in a single DNA base (T should be C), most affected individuals have a normal life expectancy, larger than normal heads, and intellectual disability, although the degree varies widely.The trial is led by neuroscientist Zilong Qiu, one of several researchers in a global race to turn base editors, a more precise form of the CRISPR gene editor, into customized therapies for children with rare genetic diseases.Seven experts who reviewed the details said Qiu and his team downplayed the risks and went ahead despite the low chance of success. They also called for a comprehensive review of the data submitted in the Nature paper and said some findings may need to be retracted. The case also raises questions about whether initial testing of such treatments should be conducted on patients with non-fatal illnesses.(This is a report from Agence France-Presse)
