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CBS News – Infant becomes world’s first patient to undergo personalized gene-editing treatment
May 16, 2025/
Soon after KJ Muldoon was born in the summer of 2024, he was diagnosed with a rare genetic disorder that is fatal for about half the infants who are born with it.
Until now, the only effective long-term treatment for the rare metabolic disease known as severe Carbamoyl Phosphate Synthetase 1 deficiency, or CPS1, had been a liver transplant.
Instead, doctors at the Children’s Hospital of Philadelphia told KJ’s family they could try something never done before. They would use a technology known as CRISPR, a personalized gene-editing therapy, to find the one uniquely mutated gene out of 20,000 in his little body, and fix it.
Jon LaPook
A Baby Received a Custom Crispr Treatment in Record Time
Scientists were able to create a bespoke treatment for KJ Muldoon’s rare genetic disorder within six months. It could be a blueprint for potentially life-saving, gene-editing Crispr therapies.
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