Scientists Use Gene Editing to Dramatically Lower Cholesterol in Early Human Study

A pioneering gene-editing study has shown promising results in lowering dangerously high cholesterol — potentially offering a permanent, one-time solution for millions at risk of heart disease.

The small pilot trial, published in the New England Journal of Medicine, used CRISPR-Cas9 technology — a biological “scissor” that can cut and modify DNA — to target and deactivate a gene responsible for producing harmful cholesterol and triglycerides. Fifteen patients with severe genetic cholesterol disorders participated in the study, which found an average 50% drop in low-density lipoprotein (LDL), the “bad” cholesterol, and a 55% reduction in triglycerides.

“We hope this is a permanent solution — a ‘one and done’ therapy,” said Dr. Steven Nissen, senior author and chief academic officer at the Cleveland Clinic’s Heart, Vascular & Thoracic Institute. “Younger people with severe disease could have reduced cholesterol for the rest of their lives.”

The treatment works by turning off a gene called ANGPTL3, which regulates LDL and triglyceride levels. People naturally born with a nonfunctioning version of this gene have extremely low cholesterol and little risk of heart disease. The study’s CRISPR therapy mimics that protective mutation but targets only the liver — where cholesterol and triglycerides are produced — reducing the risk of unintended genetic effects elsewhere in the body.

Side effects were minimal, mostly minor infusion irritation. One participant later died from advanced heart disease unrelated to the therapy. The U.S. Food and Drug Administration has required researchers to monitor participants for 15 years to ensure long-term safety.

Phase 2 and 3 clinical trials are expected to begin within the next year. While current cholesterol medications can achieve similar results, experts say a permanent treatment would be revolutionary for patients who struggle with adherence or have lifelong high cholesterol.

“The potential for this is enormous,” said Dr. Ann Marie Navar of UT Southwestern Medical Center. “For a 20-year-old with high cholesterol, it could mean freedom from daily pills or injections for life.”

Credit: CNN Newsource

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