CRISPR Takes on Cholesterol
An experimental gene-editing treatment lowered “bad” cholesterol and triglycerides in patients for at least a year after a single dose, according to new results released yesterday from an early-stage trial. Among those receiving the highest dose, bad cholesterol fell by an average of 53% and triglycerides by 48%.
The treatment uses CRISPR—a technology that allows scientists to make targeted changes to DNA—to switch off a gene in liver cells that helps regulate blood fats. Initial results released last November showed the approach worked for at least two months; the latest follow-up suggests the effect may be longer lasting. The trial involved 15 patients with severe cholesterol or triglyceride disorders. Researchers reported no serious treatment-related side effects, though larger trials are needed to assess long-term safety and effectiveness. Watch how CRISPR works.
Cholesterol is a waxy substance that comes in two forms, low- and high-density lipoproteins (LDL and HDL), while triglycerides are energy stored as fat in the bloodstream. Too much LDL, or “bad” cholesterol, can contribute to plaque buildup in blood vessels, increasing the risk of heart attack and stroke.
