Gene-editing studies offer hope for high cholesterol fix

But for now, experts say take your cholesterol-lowering medicine

Published on Feb. 11, 2026

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Scientists are testing gene-editing treatments that could dramatically lower artery-clogging cholesterol, potentially offering a one-time fix for high cholesterol. While the early-stage research in dozens of people shows promise, experts caution that much longer-term safety studies are needed before this approach could replace the cholesterol-lowering medications millions of people currently take.

Why it matters

Cardiovascular disease is the leading killer worldwide, and high cholesterol is a major driver. While diet and lifestyle changes can help, many people struggle to lower their cholesterol enough with just those approaches. Gene-editing could offer a new way to address the genetic factors behind high cholesterol, but it remains experimental and unproven.

The details

The gene-editing studies are targeting two key genes, ANGPTL3 and PCSK9, that play a role in regulating cholesterol levels. Infusions of CRISPR gene-editing tools have been shown to switch off these genes in the liver, leading to dramatic drops in LDL 'bad' cholesterol and triglycerides within just a couple weeks. However, longer-term safety and efficacy studies are still needed to determine if this approach can truly replace the need for lifelong cholesterol medication.

  • The initial studies were conducted in Australia, the U.K. and other countries.
  • A next-step study of the CRISPR Therapeutics approach is expected to start later this year, with sites yet to be announced.
  • Participants in an earlier Verve Therapeutics study have been tracked for two years, with their cholesterol still lowered.

The players

Dr. Luke Laffin

A preventive cardiologist at the Cleveland Clinic who co-authored a promising study on gene-editing for cholesterol published in the New England Journal of Medicine.

Dr. Steven Nissen

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

Dr. Kiran Musunuru

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

Dr. Joseph Wu

A researcher at Stanford University who was not involved in the gene-editing studies but cautioned about the need to address long-term safety questions.

CRISPR Therapeutics

A Swiss-based company developing gene-editing treatments for high cholesterol.

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What they’re saying

“People want a fix, not a bandage.”

— Dr. Luke Laffin, Preventive cardiologist, Cleveland Clinic (local10.com)

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

— Dr. Steven Nissen, Cardiologist, Cleveland Clinic (local10.com)

“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.”

— Dr. Joseph Wu, Researcher, Stanford University (local10.com)

What’s next

A next-step study of CRISPR Therapeutics' approach should start later this year, with sites yet to be announced.

The takeaway

While gene-editing offers hope for a potential one-time fix for high cholesterol, experts caution that much more research is needed to ensure the long-term safety and efficacy of this approach before it could replace the cholesterol-lowering medications that millions of people currently rely on.