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Gene Editing Saves Baby from Rare Disease
9 Apr
Summary
- A rare genetic disease nearly claimed KJ Muldoon's life at six months old.
- CRISPR gene editing and mRNA technology created a personalized therapy.
- KJ is now walking, talking, and thriving after receiving the treatment.
KJ Muldoon, an infant diagnosed with a rare enzyme deficiency, was saved by a revolutionary personalized therapy. This treatment, developed by a team at Children's Hospital of Philadelphia and Penn Medicine, employed CRISPR gene editing and mRNA technology, similar to that used in COVID-19 vaccines, to correct a genetic defect.
KJ received his first dose at six months old and now, one year later, is thriving at home with his family, walking and talking. This success story highlights a significant advancement in treating rare genetic diseases, which affect millions, particularly children, with limited treatment options.
Recent breakthroughs in mRNA science and CRISPR gene editing offer hope for developing such targeted therapies more rapidly and affordably. While heroic efforts were required to save KJ, establishing a systematic pathway for these treatments is crucial to benefit the thousands of other children who could similarly be helped.
