This technology is directed to the discovery of specific microRNAs that target and downregulate enzymes within the cholesterol biosynthetic pathway and is currently being tested in vivo.

Briefly, microRNAs regulate the translation of messenger RNAs (mRNAs) into protein. The inventors have discovered a set of specific microRNAs that downregulate the expression of multiple enzymes in the cholesterol biosynthetic pathway. Importantly, this technology may provide the benefits of cholesterol lowering therapies to patients that are not suited for statin-based treatments. Statins block the cholesterol biosynthetic pathway at a single enzymatic step and may result in the deleterious build-up of a metabolic intermediate. In contrast, this technology simultaneously targets the expression of multiple enzymes required for cholesterol biosynthesis and thus may avoid the build-up of metabolic intermediates. The reduction of cholesterol biosynthesis has been indicated for improved cardiovascular health and lowers the risk for heart disease, heart attack, and stroke.