A six-year-old Chinese girl died in March 2025 after receiving an experimental CRISPR-based gene therapy for a rare neurological disorder, an investigation by Science and Retraction Watch has revealed. Her death was allegedly concealed by the researchers and the hospital for over a year. Source: abc.es
The girl, identified as Mei, suffered from Snijders-Blok-Campeau syndrome, a condition caused by a mutation in the CHD3 gene. Her parents raised approximately $860,000 to fund a personalized therapy developed by neuroscientist Zilong Qiu at Shanghai Jiao Tong University. The therapy used base editors delivered via adeno-associated viruses (AAVs) directly into her cerebrospinal fluid. Source: infobae, elconfidencial
Seven days after the infusion, Mei died from a severe immune reaction (thrombotic microangiopathy) directly linked to the viral vector. Source: abc.es Instead of reporting the death, the research team:
“Los comités internos del propio centro hospitalario dictaminaron que el deceso estaba «definitivamente relacionado» con la infusión.”
“Este caso sobrepasa la desgracia humana o la mala suerte, ya que existen numerosos indicios de mala praxis científica y bioética.”
“Los investigadores chinos habrían ignorado estas alertas preclínicas y decidido continuar con el experimento.”
Experts condemned the lack of transparency and informed consent. Gemma Marfany, a genetics professor, noted "numerous signs of scientific and bioethical malpractice." Source: abc.es The case highlights the risks of n-of-1 therapies and the need for oversight in cutting-edge treatments. Source: emolcl
“La menor, identificada con el nombre ficticio de Mei para proteger su intimidad, se convirtió en la primera persona del mundo en recibir una terapia de edición genética dirigida al cerebro.”