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gene editing

Shanghai Jiao Tong University School of Medicine over the weekend said it would launch a “comprehensive investigation” into the previously undisclosed death last year of a 6-year-old girl who had received an experimental gene therapy from one of its scientists. The medical school promised “serious action” based on its findings.

Science and Retraction Watch reported the March 2025 fatality in an investigative news story last week that raised questions about the efficacy and safety of the so-called base-editing treatment, meant to fix a genetic mutation that impaired the girl’s brain development. The parents of the girl, given the pseudonym Mei, provided documents related to the trial, including a report on a primate study that showed the treatment had caused serious organ damage in four monkeys. The parents also shared financial information and recordings of conversations with her biomedical team indicating they had paid more than $860,000 for the development of the potential therapy.

The genetic recipe for the base editor was delivered into Mei’s brain cells by trillions of viruses infused into her spinal fluid. The viruses sparked a fatal...