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Saved October 29, 2025
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K.J. Muldoon, a premature infant from Philadelphia, has been treated with a personalized Crispr gene-editing therapy for a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. This breakthrough treatment, tailored to address his specific genetic mutation, marks a significant advancement in the application of gene-editing technology.
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