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First Infant Receives Life-Changing CRISPR Therapy for Rare Disorder

5/22/2025

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Summary

Story summary

In a groundbreaking moment for medical science, 9-month-old KJ Muldoon became the first infant to receive a personalized CRISPR gene-editing therapy for CPS1 deficiency, a rare metabolic disorder. Developed by the Children's Hospital of Philadelphia, this innovative treatment has significantly improved KJ's metabolic control, reducing the need for medication and igniting hope for future therapies targeting rare diseases.

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Story summary

In a groundbreaking moment for medical science, 9-month-old KJ Muldoon became the first infant to receive a personalized CRISPR gene-editing therapy for CPS1 deficiency, a rare metabolic disorder. Developed by the Children's Hospital of Philadelphia, this innovative treatment has significantly improved KJ's metabolic control, reducing the need for medication and igniting hope for future therapies targeting rare diseases.