The death of a 6-year-old girl following an experimental brain-directed gene-editing therapy in China has raised serious concerns about patient safety, research oversight, and ethical standards in first-in-human trials, according to a joint investigation by Science and Retraction Watch. The child, identified by the pseudonym "Mei," had Snijders Blok-Campeau syndrome, a rare neurodevelopmental disorder caused by a mutation in the CHD3 gene. The condition affects only about 237 people worldwide and is associated with intellectual disability of varying severity, although most patients have a normal life expectancy.
Mei had relatively mild disease and was receiving speech and occupational therapy before undergoing the experimental treatment. The investigational therapy, led by neuroscientist Zilong Qiu of Shanghai Jiao Tong University, aimed to become the world's first brain-directed…
