One-off gene-editing therapy could permanently lower cholesterol

Single-Dose Gene Editing Therapy Shows Promise in Lowering Cholesterol

A groundbreaking study indicates that a single-administration treatment to decrease the lifelong risk of heart disease is moving closer to reality. In a preliminary clinical trial, a solitary dose of a CRISPR-based gene-editing therapy successfully reduced cholesterol levels in patients, with no serious adverse effects reported.

Heart-2 Trial Details

The “Heart-2” trial, conducted by Verve Therapeutics, a biotechnology company based in Boston, enrolled 14 individuals. Participants included those with an inherited disorder causing abnormally high cholesterol and individuals who had developed heart disease at a young age. This research represents an early phase of investigation into this novel therapeutic approach.

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