Published on: 2025-05-18 17:10:56
A pivotal medical milestone has been reached. For the first time ever, researchers have used a personalized CRISPR-based gene therapy to treat an infant’s rare and life-threatening illness. Doctors at the Children’s Hospital of Philadelphia (CHOP) and Penn Medicine detailed their achievement in a study published Thursday in the New England Journal of Medicine. The treated child, named KJ, was born with a metabolic disorder known to kill up to 50% of children in their infancy. Now, three months
Keywords: cps1 gene kj medicine therapy
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