Scientists test one-time gene editing therapy that may permanently lower cholesterol
MANILA, Philippines – Scientists have taken a major step toward what could be a permanent fix for high cholesterol — a one-time gene-editing treatment that targets the liver to switch off a gene linked to elevated cholesterol and heart disease.
In a clinical trial reported by CNN on Saturday, November 8, researchers used CRISPR-Cas9 technology to deactivate the ANGPTL3 gene, which regulates cholesterol and triglyceride levels in the blood. People who naturally have mutations in this gene tend to have extremely low “bad” cholesterol and a significantly reduced risk of heart disease.
The early-stage human trial, which involved around 15 participants with severe cholesterol problems unresponsive to conventional drugs, showed promising results. At the highest dose, LDL or “bad” cholesterol dropped by about 50%, while triglycerides decreased by 55%, according to the researchers.
A potential one-time treatment
If proven safe and effective in larger trials, the therapy could eliminate the need for daily cholesterol pills or recurring injections, offering a “once-and-done” approach to controlling cholesterol. Unlike traditional gene therapies, this CRISPR-based treatment targets only liver cells, minimizing risks of unintended genetic changes in other parts of the body.
“This could be a transformative development in preventive cardiology,” one researcher said, noting that the treatment mimics nature’s own protective mutation found in some individuals.
Scientists: Still early, with caveats
Experts caution that the study is still in its infancy. While the preliminary results are encouraging, long-term safety and the durability of cholesterol reduction remain unproven.
One participant in the trial reportedly died six months after receiving the lowest dose, but investigators said the death was not linked to the treatment. Others experienced mild side effects, such as transient liver enzyme elevations and infusion-site reactions.
From rare diseases to common killers
The use of gene editing for high cholesterol marks a shift from experimental therapies once reserved for rare genetic conditions to tackling common diseases affecting millions worldwide. Cardiovascular diseases remain the leading cause of death globally, with high cholesterol being a major risk factor.
While scientists see huge potential, they warn against hype. The study’s authors and health experts stress that standard cholesterol-lowering medications remain essential, and people should not abandon proven therapies.
“This is a scientific breakthrough — but it’s not a cure yet,” the report added.
What this means for Filipinos
In the Philippines, where cardiovascular diseases are among the top causes of death, the development of a one-time gene-editing therapy could be life-changing — but access remains a big question.
Many Filipinos struggle to afford or adhere to long-term maintenance medications for high cholesterol and hypertension. A permanent fix could reduce that burden, but such cutting-edge treatments are expected to come with high costs and complex ethical and regulatory hurdles before they reach local hospitals.
Experts said the research offers hope but must be viewed cautiously. The focus, they said, is on prevention — healthy diet, physical activity, and adherence to current medications — while waiting for these future treatments to mature.
For now, the promise of gene editing underscores the need for the Philippines to strengthen its biomedical research capacity and prepare ethical frameworks for the coming age of genetic medicine.
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