Fearful of a heart attack? Experimental gene editing targets cholesterol-causing genes to clear arteries

Credit: Jackson Laboratory
Credit: Jackson Laboratory

Even after decades of drug breakthroughs aimed at preventing heart attacks, they remain the world’s leading cause of death…. Verve Therapeutics Inc. is proposing a radical solution: altering a person’s genome—the body’s instruction manual—to stop the buildup of bad cholesterol. “We’re on the cusp of potentially transforming that model to a one-and-done treatment,” says Sekar Kathiresan, chief executive officer of the Cambridge, Mass.-based company.

Verve plans to initially target those who’ve already had a heart attack because of extremely high cholesterol caused by a hereditary condition known as familial hypercholesterolemia, which affects 31 million people globally.  If it works to reduce low-density lipoprotein (LDL), or “bad,” cholesterol in that group, the company would look to widen the treatment pool.

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Verve will face plenty of hurdles in its attempt to treat the masses. It’s one of the first tests of using Crispr to edit DNA inside the human body, and patients and doctors could be wary of making a permanent change without knowing much about long-term safety, says Elizabeth McNally, director of the Center for Genetic Medicine at the Northwestern University Feinberg School of Medicine. Reluctance to take Covid-19 vaccines suggests some people may be loath to alter their DNA, she says.

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