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Health

One-off gene-editing therapy could permanently lower cholesterol

In an early-stage trial, a single dose of a CRISPR treatment lowered cholesterol levels, possibly permanently

By Michael Le Page

15 April 2025

A light micrograph cross-section showing cholesterol-containing plaque in a heart artery

A light micrograph cross-section showing cholesterol-containing plaque in a heart artery

NIGEL DOWNER/SCIENCE PHOTO LIBRARY

The prospects for a one-time treatment that reduces the risk of heart disease for the rest of a person’s life just got brighter. In an initial trial, a single dose of a CRISPR gene-editing therapy lowered cholesterol levels without any serious side effects.

The “Heart-2” trial – organised by the biotech company Verve Therapeutics in Boston – involved 14 people who either had an inherited condition leading to very high cholesterol or had developed heart disease at a young age. While this was an early-stage trial…

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