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Jun 1, 2025  |  
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NextImg:For the first time, a CRISPR drug treats a child’s unique mutation
Science & technology | One of a kind

For the first time, a CRISPR drug treats a child’s unique mutation

Scientists hope more children will benefit

WITHIN DAYS after KJ was born in Philadelphia in August 2024 it was clear that something was wrong. He was not eating and slept too much. Blood tests revealed sky-high levels of ammonia, a toxic substance the body usually expels. Genome sequencing confirmed that he had a rare genetic disease called carbamoyl-phosphate synthetase 1 (CPS1) deficiency, which often kills in infancy, and for which no good neonatal treatment exists. Then one of his doctors suggested something radical: a gene-editing drug designed specifically for him.

This article appeared in the Science & technology section of the print edition under the headline “One of a kind”

A man stands under the wing of a model of Tempest, which will be the next-generation RAF fighter jet.

The race to build the fighter planes of the future

They can hold more fuel, carry more weaponry and boast more computing power

The English Channel as the sun rises above, seen from the port of Dover

Britain is now the biggest funder of solar-geoengineering research

It is supporting experiments to thicken sea ice and make clouds more reflective


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Are juice shots worth the price?

Fresh fruit is probably a cheaper alternative


Companies have plans to build robotic horses

One diminutive design is aimed at children

Compressed music might be harmful to the ears

In guinea pigs it can weaken muscles important for hearing

How to build strong magnets without rare-earth metals

China’s export restrictions may boost scientific innovation