Scientists have developed a novel DNA-based therapy that lowered 'bad' LDL cholesterol by nearly 50% in animal models, according to a study published in the journal Nature on April 30, 2026. The research, led by a team at the University of Texas Southwestern Medical Center, used tiny DNA molecules called 'aptamers' to block a key protein involved in cholesterol production.
The therapy targets PCSK9, a protein that prevents the liver from removing LDL cholesterol from the blood. By inhibiting PCSK9, the DNA aptamers allowed the liver to clear more LDL, resulting in a 47% reduction in cholesterol levels in mice over a four-week period. No serious side effects were reported in the animals.
Lead author Dr. Sarah Chen stated, 'This approach could provide an alternative for patients who cannot tolerate statins or need additional cholesterol-lowering options.' The team plans to begin human clinical trials in early 2027, pending regulatory approval.
Statins remain the standard treatment for high cholesterol, but up to 20% of patients experience muscle pain or other side effects. The new DNA-based therapy, if proven safe and effective in humans, could offer a new avenue for managing cardiovascular disease, which remains the leading cause of death globally.
