Studies Test Whether Gene-Editing Can Fix High Cholesterol

Two companies show hints that switching off certain genes could dramatically lower cholesterol

Published on Feb. 11, 2026

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Scientists are testing a gene-editing treatment that could offer a one-time fix for high cholesterol. The approach, being developed by two companies, involves switching off certain genes that play a role in cholesterol production. Early studies have shown the treatment can significantly lower artery-clogging LDL cholesterol and triglycerides in a matter of weeks. However, longer-term studies in more people are needed to assess the safety and long-term efficacy of this gene-editing approach.

Why it matters

Cardiovascular disease is the leading cause of death worldwide, and high cholesterol is a major risk factor. Current cholesterol-lowering medications like statins require lifelong treatment, and many patients struggle to lower their cholesterol enough or quit due to side effects. A one-time gene-editing treatment that could dramatically and safely lower cholesterol would be a major breakthrough in preventing heart attacks and strokes.

The details

The gene-editing approach targets genes like ANGPTL3 and PCSK9, which play a role in cholesterol production. In early studies, a single infusion of the gene-editing tool was able to cut LDL and triglyceride levels by up to 50% within 2 weeks. The treatment works by permanently disabling the targeted genes in liver cells, which then pass on the altered genes as those cells reproduce. However, longer-term safety and efficacy studies are still needed to assess potential side effects and ensure the cholesterol-lowering effects persist over a lifetime.

  • In November, researchers reported results from a study where 15 adults received a single infusion of the gene-editing tool, which switched off the ANGPTL3 gene.
  • Verve Therapeutics, a subsidiary of Eli Lilly, previously reported that its PCSK9-targeted gene-editing infusion cut LDL cholesterol by a similar amount in a small study.

The players

Luke Laffin

A preventive cardiologist at the Cleveland Clinic who co-authored a promising study on the gene-editing approach.

Kiran Musunuru

A cardiologist at the University of Pennsylvania who previously reported on people with naturally low cholesterol due to mutations in genes like ANGPTL3.

Steven Nissen

A cardiologist at the Cleveland Clinic who oversaw the ANGPTL3 gene-editing study funded by CRISPR Therapeutics.

Joseph Wu

A professor at Stanford University who cautioned about the need to assess the long-term safety of CRISPR-based therapies.

CRISPR Therapeutics

A Swiss-based company developing a gene-editing approach to lower cholesterol.

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What they’re saying

“People want a fix, not a bandage.”

— Luke Laffin, Preventive Cardiologist, Cleveland Clinic (New England Journal of Medicine)

“It's a natural experiment in what would happen if we actually changed the gene.”

— Steven Nissen, Cardiologist, Cleveland Clinic (New England Journal of Medicine)

“CRISPR-based therapies for any disease haven't been used enough to know long-term safety -- and the particles carrying the gene-editing tool can irritate or inflame the liver.”

— Joseph Wu (Stanford University)

What’s next

A next-step study of CRISPR Therapeutics' approach should start later this year, with sites yet to be announced. Longer studies of the gene-editing approach in far more people are needed to assess long-term safety and efficacy.

The takeaway

This gene-editing research holds promise as a potential one-time fix for high cholesterol, a major risk factor for heart disease. However, significant further testing is required to ensure the treatment is safe and effective over a lifetime before it could become a viable alternative to lifelong cholesterol-lowering medications.