medicine
One infusion cut cholesterol in half for a year
One dose. One gene switched off. Cholesterol still down a year later.
A single infusion of a CRISPR gene-editing therapy cut LDL cholesterol by 52.5% and triglycerides by 47.8% after one year, according to a Phase 1 trial from Cleveland Clinic published this week in the New England Journal of Medicine. The study followed 15 people with lipid disorders that had not responded well to standard medications. All received one dose of an experimental treatment called CTX310, which carries the editing system to the liver and switches off a gene, ANGPTL3, that helps regulate fats in the blood. No serious adverse events tied to the therapy were reported over the year. "The durability of the lipid-lowering effect was impressive," said cardiologist Luke Laffin, the study's first author. The treatment is still experimental, and 15 people is a small start. Researchers plan to keep tracking participants for another 15 years, which is the part of gene editing nobody rushes.