Studies Test Gene-Editing for High Cholesterol

For now, doctors say take your medicine as gene-editing approaches show early promise

Published on Feb. 16, 2026

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Scientists are testing gene-editing treatments that could offer a one-time fix for high cholesterol, a major driver of heart attacks and strokes. While the early research in just a few dozen people is promising, longer studies in more people are needed to determine the long-term safety and effectiveness of these gene-editing approaches compared to existing cholesterol-lowering medications.

Why it matters

Cardiovascular disease is the leading killer worldwide, and millions struggle to control their cholesterol levels enough with lifelong medication. Gene-editing could potentially provide a one-time solution, but significant safety and efficacy questions remain to be answered before this becomes a viable alternative to current treatments.

The details

The gene-editing approaches being developed by CRISPR Therapeutics and Verve Therapeutics target genes like ANGPTL3 and PCSK9 that play a role in cholesterol production. Early studies have shown these treatments can dramatically lower LDL "bad" cholesterol and triglyceride levels within weeks of a single infusion. However, longer-term studies in larger populations are still needed to assess safety, as gene-editing is considered a permanent change and the long-term impacts are unknown.

  • In November 2025, a study led by Cleveland Clinic researchers reported that a CRISPR-based treatment targeting the ANGPTL3 gene lowered LDL and triglyceride levels by 50% within two weeks in 15 adults.
  • Verve Therapeutics, a subsidiary of Eli Lilly, earlier reported similar LDL cholesterol reductions from its PCSK9-targeted gene-editing treatment in a small study.

The players

Dr. Luke Laffin

A preventive cardiologist at the Cleveland Clinic who co-authored a promising study on a CRISPR-based cholesterol treatment.

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 co-founded Verve Therapeutics and previously reported on people with naturally low cholesterol due to ANGPTL3 and PCSK9 gene mutations.

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 concerns with CRISPR-based therapies.

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What they’re saying

“People want a fix, not a bandage.”

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

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

— Dr. Steven Nissen, Cleveland Clinic cardiologist (ksl.com)

What’s next

A next-step study of CRISPR Therapeutics' ANGPTL3-targeting approach should start later this year, with study sites yet to be announced. Longer-term studies in larger populations are still needed to fully assess the safety and efficacy of these gene-editing treatments compared to existing cholesterol medications.

The takeaway

While gene-editing offers the potential for a one-time fix for high cholesterol, a leading cause of heart disease, significant research is still needed to determine if these treatments can be a viable alternative to lifelong cholesterol-lowering medications. For now, doctors recommend patients focus on lifestyle changes and take their prescribed cholesterol medications as directed.