World’s First Personalized Gene-Editing Treatment Cures Baby’s Condition

Student summary by GL

Original Source:Published May 15, 2025 in The New York Times by Gina Kolata

Abstract

Background: KJ was a baby born with a rare and serious condition called CPS1 deficiency, which made it hard for his body to remove ammonia. This could cause brain damage or death. Most babies with this disease do not survive their first week.

Objective: Doctors wanted to help KJ by fixing the exact genetic mistake that caused his illness, using a new type of treatment that had never been tried before in a person.

Method: Scientists designed a custom gene-editing treatment using CRISPR. They packaged it in fat droplets to safely travel through the blood to KJ’s liver, where it could correct the faulty DNA.

Results: KJ received three doses of the treatment. After that, he was able to eat more protein, needed less medicine, and got stronger. He began to grow normally and is now well enough to prepare to go home from the hospital.

Conclusion: This was the first time a personalized gene-editing treatment was used in a human. It shows that this method could be used to help other people with rare genetic diseases in the future.

Original Source: The New York TimesAuthor: Gina KolataPublished: May 15, 2025