Genetic Editing Could Reduce the Risk of LDL Cholesterol

A clinical trial showed plenty of promise

Gene image
A new form of treatment shows promise.
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When medical professionals talk about the ways cholesterol can threaten a person’s health, they’re probably talking about LDL cholesterol – otherwise known as “bad cholesterol.” On its website, the Centers for Disease Control and Prevention notes that “[h]igh levels of LDL cholesterol raise your risk for heart disease and stroke.” Complicating matters further is a genetic disorder called familial hypercholesterolemia, which can make LDL cholesterol even more hazardous.

According to the American Heart Association, this condition dramatically increases the risk of heart disease for those who have inherited the genetic mutation — a total of approximately 1.3 million Americans.

It’s an unsettling condition to begin with — but people living with this and working to keep it under control might have a reprieve in sight. As Gina Kolata reports at The New York Times, a Boston-based company called Verve Therapeutics recently announced promising results for a new genetic therapy to address familial hypercholesterolemia.

Verve Therapeutics’ trial involved 10 patients, each of whom had been diagnosed with familial hypercholesterolemia. The patients who received the highest doses of the treatment saw their LDL cholesterol levels drop significantly, with the primary side effect being what Kolata described as “flulike symptoms for a few hours.”

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The Times points out that more trials are forthcoming, but it seems likely we’ll hear more about this treatment soon — Eli Lilly paid an eight-figure sum to work with Verve Therapeutics in the future. If all goes well, this could be a breakthrough moment in treating people with a genetic condition that puts their hearts at a high level of risk.

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