Baby with rare disease given world-first personal CRISPR gene therapy

An infant with a severe genetic condition has shown signs of improvement after receiving a gene-editing treatment tailored to his specific mutation

Originally published  by Michael Le Page at newscientist.com on 15 May 2025

Baby KJ after a gene-editing infusion with researchers Rebecca Ahrens-Nicklas and Kiran Musunuru

Children’s Hospital of Philadelphia

A baby boy with a life-threatening genetic condition has become the first person to receive a bespoke CRISPR gene-editing treatment, giving a glimpse into what the future of medicine might look like.

It’s the first time anyone has been given a gene-editing treatment designed to correct a disease-causing mutation found only in that individual, Rebecca Ahrens-Nicklas at the Children’s Hospital of Philadelphia, Pennsylvania, told a press briefing. “He’s showing some early signs of benefit,” she says, but it is too soon to tell how well the treatment worked.

The researchers published the details as soon as possible in the hope that it will inspire others, says team member Kiran Musunuru at the University of Pennsylvania. “We very much hope that showing that it’s possible to make a personalised gene-editing therapy for a single patient in several months will inspire others to do the same,” he says.

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