First-in-human trial of CRISPR gene-editing therapy safely lowers cholesterol and triglycerides

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In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, according to a preliminary late-breaking science presentation Saturday at the American Heart Association's Scientific Sessions 2025. The meeting, held Nov. 7–10 in New Orleans, is a premier global exchange of the latest scientific advancements, research and evidence-based clinical practice updates in cardiovascular science. The paper was simultaneously published in the New England Journal of Medicine.