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The discovery that tiny RNA molecules play a central role in the expression of genes (JW Gen Med Dec 31 2003) continues to demonstrate its importance to medicine.
In a multi-institutional study, two microRNAs — miR-33a and miR-33b — that inhibit production of HDL cholesterol were rendered inactive by anti-sense oligonucleotides that specifically bind them. When these anti-sense oligonucleotides were injected subcutaneously into monkeys, production of HDL cholesterol increased 50% and levels of VLDL triglycerides decreased 50%. These changes persisted at 12 weeks; no toxic effects from the treatment were noted. When expression levels were measured for a large number of genes that play roles in determining levels of HDL cholesterol and triglyc…