Genome editing reduces cholesterol in large animal model, laying human trial groundwork

Using genome editing to inactivate a protein called PCSK9 effectively reduced cholesterol levels in rhesus macaques. This is the first demonstration of a clinically relevant reduction of gene expression in a large animal model using genome editing. The study describes a possible new approach for treating heart disease patients who do not tolerate PCSK9 inhibitors -- drugs that are commonly used to combat high cholesterol.



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