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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
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. She and Musunuru have been developing treatments for this kind of condition that target the liver, allowing them to rapidly create a base-editing therapy – a form of CRISPR – that corrects one of KJ’s two copies of the CPS1 gene.
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