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 t he future of medicine might look like. It’s the first time anyone has been given a gene-editing treatment designed t o 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...