Gene-Editing Trials Offer Potential One-Time Fix for High Cholesterol

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Scientists are testing gene-editing treatments that could offer a one-time fix for high cholesterol, a major risk factor for heart disease. While the research is still in early stages, involving only a few dozen people so far, the results show hints that switching off certain genes could dramatically lower artery-clogging cholesterol levels.

Why it matters

Heart disease remains a leading cause of death worldwide, and high cholesterol is a major contributor. If gene-editing treatments prove effective, they could provide a groundbreaking new way to prevent heart attacks without the need for lifelong medication. However, the technology is still in very early development, so traditional cholesterol-lowering drugs remain the recommended treatment for now.

The details

The gene-editing approaches being developed by two companies, Verve Therapeutics and Intellia Therapeutics, aim to permanently turn off a gene called PCSK9 that regulates cholesterol levels. In early trials, participants saw their LDL "bad" cholesterol drop by 50% or more after a single treatment. While promising, the long-term safety and durability of these effects are still unknown.

• The gene-editing research is still in early, small-scale clinical trials.

What’s next

The gene-editing treatments are still in early clinical trials, so traditional cholesterol-lowering medications remain the recommended treatment for now. Researchers will continue to study the long-term safety and effectiveness of the gene-editing approaches.