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Baby KJ's Life Transformed by Groundbreaking CRISPR Therapy

6/4/2025

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Summary

Story summary

After 307 days in the hospital, baby KJ Muldoon received groundbreaking CRISPR gene-editing therapy for his severe CPS1 deficiency. Developed by the innovative team at Children’s Hospital of Philadelphia, this personalized treatment corrected his genetic mutation, giving him a new lease on life and showcasing the promise of tailored gene therapies for rare diseases.

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Story summary

After 307 days in the hospital, baby KJ Muldoon received groundbreaking CRISPR gene-editing therapy for his severe CPS1 deficiency. Developed by the innovative team at Children’s Hospital of Philadelphia, this personalized treatment corrected his genetic mutation, giving him a new lease on life and showcasing the promise of tailored gene therapies for rare diseases.