Studies test whether gene-editing can fix high cholesterol. For now, take your medicine

Scientists are testing an entirely new way to fight heart disease: a gene-editing treatment that might offer a one-time fix for high cholesterol. It's very early-stage research, tried in only a few dozen people so far. But gene-editing approaches being developed by two companies show hints that switching off certain genes could dramatically lower artery-clogging cholesterol, raising hopes of one day being able to prevent heart attacks without having to take pills. "People want a fix, not a bandage," said Dr. Luke Laffin, a preventive cardiologist at the Cleveland Clinic. After co-authoring a promising study published in the New England Journal of Medicine, he said he was flooded with queries about how to participate in the next clinical trial. Everyone needs a certain amount of cholesterol. But too much, especially a "bad" kind called LDL cholesterol, builds plaque in the artery walls and is a main driver of heart attacks and strokes. Cardiovascular disease is the nation's—and world's—leading killer. Millions take cholesterol-lowering medicines such as statins, the cornerstone of treatment. But many still struggle to lower their cholesterol enough, and sticking with the drugs for life is difficult, with some quitting because of side effects. Years ago, Dr. Kiran Musunuru, a cardiologist now at the University of Pennsylvania, reported that some lucky people harbor a mutation that turns off a gene named ANGPTL3, lowering their levels of both LDL cholesterol and another bad fat, triglycerides. Separately, geneticists at UT Southwestern Medical Center found that still other people's extremely low LDL was due to loss of function of another gene named PCSK9. "It's a natural experiment in what would happen if we actually changed the gene," said the Cleveland Clinic's Dr. Steven Nissen, who, with Laffin, oversaw an ANGPTL3 study funded by Swiss-based CRISPR Therapeutics. Today, there are injected medicines that block proteins produced by the PCSK9 and ANGPTL3 genes in the liver, thus helping the body clear away cholesterol. The new research uses CRISPR, the Nobel Prize-winning gene-editing tool, to try switching off one of those genes in people at high risk of uncontrolled cholesterol. This article was provided by The Associated Press.