Researchers at Victoria's Victorian Heart Hospital and Monash University have, for the first time in the world, successfully used a one-off CRISPR-based gene therapy to lower cholesterol and triglyceride levels.
The method works by switching off the ANGPTL3 gene, known as the body's “fat brake,” which prevents the body from efficiently clearing fats. This natural mechanism is found in a small number of people, protecting them from cardiovascular disease. It has now been reproduced in 15 patients with critically high cholesterol who did not respond to standard drugs.
After a single injection of the CTX310 drug, levels began to fall within two weeks. After 60 days, cholesterol had dropped by almost half, and triglycerides by 55% to 80%. All participants will remain under observation for 15 years.
This is the first use of CRISPR to treat such a common problem. About 25 to 30 million people worldwide have genetically inherited high cholesterol. If further studies confirm its effectiveness, the therapy could become an alternative to lifelong medication for them.
