A Future of Gene Therapies to Greatly Reduce the Incidence of Cardiovascular Disease

It seems plausible that one of the first major mainstream areas of development for human gene therapy will involve disabling genes that sustain levels of lipids in the bloodstream. There are a number of credible targets, including those with sizable numbers of human loss of function mutants who seem to do quite well as a result of their mutation: PCSK9, ANGPTL3, ANGPTL4, and ASGR1 for example. Reducing lipid levels in the bloodstream has the effect of slowing the development of cardiovascular disease, reducing the risk of heart attack, stroke, and other related issues. The success of statin drugs is based on exactly this effect, and gene therapies would be much more effective than statins – a one time treatment producing a larger and permenant benefit.

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How does this work under the hood? Why does lowering blood lipids – cholesterol, triglycerides, and so forth – have this beneficial result? The primary mechanism of interest relates to the development of atherosclerosis through damaged lipid molecules. The normal operation of metabolism produces reactive molecules that can oxidize lipids, so some small fraction of the lipids in the bloodstream are damaged in this way. With the progression of aging, various forms


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