CRISPR gene editing therapy cuts LDL cholesterol in small study

A CRISPR-based gene editing therapy dramatically reduced levels of low-density lipoprotein, often called “bad cholesterol,” and triglycerides in patients with heart disease, a new study found.

Some researchers said that the treatment, developed by the biotech CRISPR Therapeutics and so far tested in just 15 people, pointed to a possible future where some people prevent heart attacks with one-time gene editing. But this research is also at a very early stage, and scientists expressed concerns about the potential liver toxicities of the treatment and those like it. There is also the question of how many patients would want permanent editing for common illnesses that might be treated with a shot or a pill.

The results were published in the New England Journal of Medicine and are being presented Saturday at the annual scientific meeting of the American Heart Association in New Orleans.