Scientists have unveiled a groundbreaking gene-editing treatment that could reduce harmful cholesterol levels by half after just one dose. In a small trial involving 15 participants with high cholesterol, despite taking conventional lifelong medications, the experimental drug produced significant reductions in both LDL ("bad" cholesterol) and triglycerides, two major risk factors for heart disease.
The therapy uses CRISPR technology to target the ANGPTL3 gene in the liver. Lead researcher Dr. Steven Nissen, a cardiologist at the Cleveland Clinic, stated: "We've never had anything that could lower both LDL and triglycerides by around 50 percent." The results were presented at the American Heart Association’s annual meeting and published in the New England Journal of Medicine.
Participants' cholesterol and triglyceride levels began to drop within two weeks of receiving the treatment, with effects sustained for at least 60 days. The trial included individuals from Australia, New Zealand and the UK, all in their 50s and 60s. Thirteen were men, and all had uncontrolled high cholesterol, triglycerides or both.
Dr. Luke Laffin, co-leader of the study, highlighted the potential: "Rather than a once-daily pill or monthly injection, this therapy would potentially offer a one-time infusion that is safe and durable." Three participants experienced temporary reactions, such as nausea and elevated liver enzymes, which quickly resolved. Future larger trials are needed to confirm safety and efficacy.
