Scientists are testing experimental Gene-editing cholesterol treatment therapies that may offer a one-time solution for high cholesterol, with early trials showing sharp reductions in harmful fats, though experts stress patients should continue proven medications for now.
Early Trials Suggest One-Time Treatment May Cut Harmful Cholesterol
Researchers are exploring a new strategy against heart disease: gene editing designed to switch off genes that drive high cholesterol. Early studies involving only a few dozen participants suggest the approach could significantly lower artery-clogging fats.
The experimental treatments, developed by CRISPR Therapeutics and Verve Therapeutics, aim to permanently disable genes linked to elevated low-density lipoprotein, or LDL, cholesterol. High LDL is a major contributor to heart attacks and strokes, which remain the leading causes of death worldwide.
“People want a fix, not a bandage,” said Dr. Luke Laffin, a preventive cardiologist at the Cleveland Clinic and co-author of a recent study published in the New England Journal of Medicine. He said interest in this gene-editing cholesterol treatment surged after the findings, with many patients asking how to join future trials
In one study, 15 adults received a single infusion carrying the CRISPR gene-editing tool to the liver. Within two weeks, those given the highest dose saw LDL cholesterol and triglycerides drop by about half, according to researchers, including Dr. Steven Nissen of the Cleveland Clinic.
Boston-based Verve Therapeutics reported similar results earlier, saying an infusion targeting the PCSK9 gene cut LDL levels by roughly 50% in a small trial.
Both studies were conducted in countries including Australia and the United Kingdom. A spokesperson for Eli Lilly, Verve’s parent company, said U.S. study sites are now opening, while additional trials for CRISPR Therapeutics’ approach are expected later this year.
Scientists Examine Genetic Clues While Warning of Safety Questions
Scientists’ interest in Gene-editing cholesterol treatment stems from naturally occurring mutations. Some people inherit gene changes that keep cholesterol unusually low, dramatically reducing their risk of heart disease.
Dr. Kiran Musunuru, a cardiologist at the University of Pennsylvania, previously identified individuals whose mutation disables a gene called ANGPTL3, lowering both LDL cholesterol and triglycerides. Others have low LDL because of loss of function in the PCSK9 gene.
“It’s a natural experiment in what would happen if we actually changed the gene,” Nissen said.
Still, experts caution that long-term safety remains unclear. CRISPR therapies are considered permanent because edited cells pass genetic changes to future cells.
“There are major safety questions to be answered,” said Dr. Joseph Wu of Stanford University, who was not involved in the studies. He warned the delivery particles could inflame the liver and said researchers must confirm the edits affect only intended targets.
Musunuru added that participants in an earlier Verve study have been followed for two years with cholesterol still reduced, but larger and longer trials are needed before the therapy could become widely available.
For now, most trials focus on patients at extremely high risk who cannot control cholesterol through existing treatments.
Doctors Urge Proven Treatments and Heart-Healthy Habits
Despite the excitement, physicians emphasize that this gene-editing cholesterol treatment remains experimental and should not replace established care.
Millions rely on statins, which reduce the liver’s cholesterol production and remain the cornerstone of treatment. Some patients stop taking them due to side effects, while others struggle to lower cholesterol enough even with medication.
The American Heart Association recommends several immediate steps to improve heart health. These include eating diets rich in fruits, vegetables, whole grains, and healthy fats; staying physically active; maintaining a healthy weight; quitting smoking; and getting sufficient sleep.
Medical factors also matter. Blood pressure below 120 over 80 is considered optimal, and controlling blood sugar helps prevent heart damage linked to diabetes.
Guidelines generally consider LDL levels below 100 acceptable for healthy adults, but those with heart disease are advised to aim for at least 70 or lower if risk is high.
When lifestyle changes fall short, doctors say medications, including statins, additional pills, and injectable drugs. They remain effective tools.
Whether Gene-editing cholesterol treatment ultimately fulfills its promise is uncertain, but researchers say the goal is clear: preventing heart attacks with treatments that may one day require just a single dose.
