“Despite an ever-growing armory of pills, injections and lifestyle interventions to lower the risk of heart disease, it remains the No. 1 killer of Americans. About half of people who start taking a daily statin stop, and many who could benefit from the drugs don’t start them. Lack of access to health care, an inability to tolerate the drugs and concerns about taking a medicine for life all can play a role.
In the new study, funded by the biotechnology company CRISPR Therapeutics, 15 patients received a one-time infusion of lipid nanoparticles, essentially fat bubbles, carrying gene-editing machinery to their livers. That machinery was programmed to snip and disable a gene involved in cholesterol metabolism called ANGPTL3.
The experiment was inspired, in part, by the discovery that there are people who have naturally occurring versions of the ANGPTL3 gene that aren’t functional. Those people have very low levels of cholesterol and triglycerides and a lower risk of cardiovascular disease. Gene-editing technology allows scientists to try to emulate this trait.
In the four patients who received the highest dose of the treatment, levels of LDL cholesterol and triglycerides, both risk factors for heart disease, dropped by about 50 percent and remained steady a year later.”
From New York Times.