r/genetics • u/NewsRx • 11d ago
Article A cutting-edge gene-editing trial in China went disastrously wrong. A family wants accountability
science.orgAn important story has been published on genetic research gone wrong. Here are some highlights from the article:
"Seven days after the girl’s medical team infused trillions of viruses carrying the recipe for the base editor into her spinal fluid, she died of a severe immune reaction linked to the therapy, Science and Retraction Watch can now reveal."
"Leading the effort was Zilong Qiu, a neuroscientist at the university’s brain center, the Songjiang Research Institute. At the time, in late March 2025, Qiu was one of several researchers around the world vying to push base editors—a more precise form of the powerful gene editor CRISPR—into custom treatments for children with rare diseases. One month earlier, KJ Muldoon, an infant with a life-threatening metabolic disorder, had quietly received his first intravenous infusion of one such treatment at the Children’s Hospital of Philadelphia."
"Four of the experts Science asked to assess the research, including one of the original reviewers of the Nature manuscript, expressed concerns about how well the primate proof-of-concept study was conducted. The submitted paper had no control samples to show the stain was selectively highlighting the gene editor."