Genome Editing Reduces Cholesterol in Large Animal Model, Laying the Groundwork for In-Human Trials
Perelman School of Medicine at the University of PennsylvaniaUsing genome editing to inactivate a protein called PCSK9 effectively reduces cholesterol levels in rhesus macaques, the first demonstration of a clinically relevant reduction of gene expression in a large animal model using genome editing. This finding could lead to a possible new approach for treating heart disease patients who do not tolerate PCSK9 inhibitors—drugs that are commonly used to combat high cholesterol.