An Experimental One-Time Treatment for High Cholesterol Shows Promising Results

Heart disease remains the leading killer of people worldwide, and one of the key risk factors driving deaths is elevated levels of cholesterol. In a new article published in the New England Journal of Medicine, researchers report that a gene-editing therapy helped people with increased levels of cholesterol and triglycerides maintain lower levels of these lipids for at least a year. The findings build on earlier results of the therapy, reported last November. The gene-editing treatment, developed by the biotech company CRISPR Therapeutics, uses CRISPR technology to precisely edit a gene involved in cholesterol regulation. The gene, called ANGPTL3, makes an enzyme that blocks the breakdown of bad cholesterol (LDL) and triglycerides. But scientists discovered a long-lived population in Italy in which people born with a mutation in ANGPTL3—rendering it defective—had low levels of LDL and triglycerides and very little heart disease. The CRISPR therapy mimics this naturally occurring mutation; people who have received it now have this mutation in their ANGPTL3, which means they can now break down cholesterol and triglycerides properly.In the small study, 15 people with severe forms of h...