Genome editing reduces cholesterol in animals, humans could be next

People who have high cholesterol often turn to statins and newer drugs known as PCSK9 inhibitors to manage the condition. But those who don’t tolerate these drugs may be left without effective therapies to rely on.


A new study, conducted in a primate animal model, offers hope for a new therapeutic strategy for controlling high cholesterol. Researchers Lili Wang and James Wilson of the Perelman School of Medicine led a team in investigating the effectiveness of using genome editing, rather than a pharmacologic, to block the activity of PCSK9, a protein that prevents the removal of cholesterol from the liver. 

Wang, Wilson and colleagues used a non-disease-causing viral vector to ferry in a copy of an enzyme that disrupts PCSK9 to the animals’ genome. They found that PCSK9 levels went down by 45 to 84 percent, and LDL cholesterol (the “bad” cholesterol) levels lowered by 30 to 60 percent. The viral vector has been shown to be safe in clinical trials for patients with hemophilia, suggesting that a human therapy relying on this method could be in the future for patients with hypercholesterolemia.

“Most often these patients are treated with repeated injections of an antibody to PCSK9,” says Wang. “But, our study shows that with successful genome editing, patients who cannot tolerate inhibitor drugs might no longer need this type of treatment.”

Read more at Penn Medicine News.