Scientists edit gene that could permanently lower high cholesterol

A groundbreaking pilot study published Saturday in the New England Journal of Medicine suggests that a single gene edit could one day permanently reduce dangerously high cholesterol levels, potentially eliminating the need for lifelong medication.

The study ‘Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3’, involving just 15 patients with severe cholesterol disorders was designed primarily to test the safety of a CRISPR-Cas9–based therapy. 

This technology, often described as a pair of biological “scissors”, precisely cuts targeted DNA to modify or switch off specific genes.

Early results were promising: participants experienced an average 50% reduction in low-density lipoprotein (LDL), commonly known as “bad” cholesterol. Elevated LDL is a major contributor to heart disease, the leading cause of death among adults in the United States and worldwide.

The study also found an average 55% reduction in triglycerides, a different type of fat in the blood that is also linked to an increased risk of cardiovascular disease.

Dr Steven Nissen, senior study author and chief academic officer of the Sydell and Arnold Miller Family Heart, Vascular & Thoracic Institute at Cleveland Clinic in Ohio said, “We hope this is a permanent solution, where younger people with severe disease can undergo a ‘one and done’ gene therapy and have reduced LDL and triglycerides for the rest of their lives."