โค๏ธ Gene Therapy Cut "Bad" Cholesterol in Half, and the Effect Has Lasted a Year
Researchers at the Cleveland Clinic have published encouraging results from the first stage of a clinical trial of CTX310, an experimental gene-editing therapy designed to lower cholesterol.
Patients received the treatment once through an intravenous infusion, and it edited the DNA of their liver cells. The CRISPR-Cas9 "genetic scissors" switched off the ANGPTL3 gene, which encodes a protein that regulates fats in the blood. As a result, the body maintains lower cholesterol and triglyceride levels without repeated treatments.
One year later, among four participants who received the highest dose, LDLโthe so-called "bad" cholesterolโremained an average of 52.5% below baseline, while triglycerides were 47.8% lower.
๐ So far, the study includes only 15 patients whose lipid disorders were difficult to control with conventional treatment. During the first year, researchers reported no serious adverse effects. However, pโฆ
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