A Gene-Editing Shot Could Protect Against Heart Attacks
The treatment successfully lowered cholesterol in monkeys
Reengineering Life is a series from OneZero about the astonishing ways genetic technology is changing humanity and the world around us.
Heart disease is the leading cause of death globally, killing nearly 18 million people each year. It’s often caused by a buildup of cholesterol, a waxy substance in the blood. But curiously, some people with rare genetic mutations are naturally protected against high cholesterol. Consequently, these people have a dramatically lower risk of heart attack, a form of heart disease.
That has led scientists to consider whether tweaking the DNA code in people without this beneficial trait could lower their cholesterol levels and protect them against heart disease for life.
One biotech company is putting that idea to the test. A single injection of a gene-editing treatment successfully lowered “bad” LDL cholesterol in 14 monkeys, according to Sekar Kathiresan, co-founder and CEO of Verve Therapeutics. He announced the findings on June 27 at the virtual annual meeting of the International Society for Stem Cell Research. Kathiresan tells OneZero that the approach could be “an entirely new way to treat heart attack.”
“Today’s treatments are not cutting it,” he says. “What we’re looking to do is transform the way this chronic disease is cared for.” Though promising, the findings are preliminary and have not yet been peer-reviewed or published in a scientific journal.
The experiment represents a key test for base editing, a new form of CRISPR that’s more precise and possibly safer than the original version. Base editing has been tried in lab mice before, but this is the first publicized attempt of the technique in monkeys. Because of the biological similarities between humans and our primate cousins, monkey tests are important for learning about the safety and effectiveness of a new drug or treatment.
Scientists at Verve Therapeutics targeted two genes in the monkeys: PCSK9 and ANGPTL3, which are both found in the liver. These genes help regulate levels of cholesterol as well a type of fat called…