Girl dies after experimental gene-editing treatment in China

A young girl in China has died following an experimental gene-editing treatment aimed at correcting a neurological syndrome. The child suffered from a mild developmental delay associated with Snijders-Blok-Campeau syndrome, which can cause cognitive impairment and autism.

The experimental therapy involved an infusion into the spinal cord using a large quantity of virus vectors carrying a CRISPR base editor. The goal was to correct a single letter in a gene crucial for brain expression. Tragically, the girl died seven days after the treatment due to a severe immune reaction.

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Experimental therapies for a single patient usually make big headlines in the media and open television news. When they work.

The costly therapy, priced at $860,000, was carried out at Shanghai's Xinhua Hospital under the direction of Zilong Qiu, a neuroscientist involved in developing CRISPR gene-editing techniques for rare childhood diseases. Pre-clinical trials on mice and non-human primates had indicated potential liver and kidney issues, suggesting a need for further review before human application.

Scientists emphasize the inherent risks of such experimental, single-patient procedures, which are often not formally approved. While these therapies can generate significant media attention when successful, adverse outcomes, especially fatal ones, can also emerge. The incident underscores the ethical and safety challenges in pioneering gene-editing treatments for rare conditions.

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However, if something goes wrong, we usually don't find out, unless the consequences are fatal for the patient and the news escalates again to headlines due to the harm caused.