Stabilization of hepatitis C virus RNA by an Ago2–miR-122 complex
MicroRNAs regulate eukaryotic gene expression by binding to regions of imperfect complementarity in mRNAs, typically in the 3′ UTR, recruiting an Argonaute (Ago) protein complex that usually results in translational repression or destabilization of the target RNA. The translation and decay of mRNAs are closely linked, competing processes, and whether the miRNA-induced silencing complex (RISC) acts primarily to reduce translation or stability of the mRNA remains controversial.
miR-122 is an abundant, liver-specific miRNA that is an unusual host factor for hepatitis C virus (HCV), an important cause of liver disease in humans. Prior studies show that it binds the 5′ UTR of the messenger-sense HCV RNA genome, stimulating translation and promoting genome replication by an unknown mechanism. In this study the authors show that [click to continue…]
Santaris Pharma A/S announced today that it has advanced miravirsen (SPC3649), a microRNA targeted drug for treatment of Hepatitis C virus (HCV) infection into Phase 2 studies to assess the safety and tolerability of the drug.
Santaris Pharma A/S also received acceptance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA). In addition to the United States, the Phase 2a clinical trials will be conducted in the Netherlands, Germany, Poland, Romania, and Slovakia.
Miravirsen is a specific inhibitor of miR-122, a liver specific microRNA that the Hepatitis C virus requires for replication. Miravirsen is designed to recognize and sequester miR-122, making it unavailable to the Hepatitis C virus. As a result, the replication of the virus is effectively inhibited and the level of Hepatitis C virus is reduced.
In addition to miravirsen, Santaris Pharma A/S is developing other drugs targeting mRNAs and microRNAs both internally as well as in partnerships and collaborations with miRagen Therapeutics (cardiovascular diseases), Shire plc (rare genetic disorders), Pfizer (undisclosed therapeutic areas), GlaxoSmithKline (viral disease) and Enzon Pharmaceuticals (oncology). (read more… )
It’s only a matter of time before the first microRNA therapeutic reaches the market.
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MicroRNA — A Switch that Determines Cell Behavior and Holds Answers on Disease, Prevention, Treatment
ROCHESTER, Minn. — Thousands of research studies are under way to better understand microRNA — short for micro ribonucleic acid. These tiny genetic strands may play a role in identifying, treating and possibly preventing many diseases, according to the July issue of Mayo Clinic Health Letter.
MicroRNA acts like a switch that changes cell behavior. Different microRNAs are in each tissue of the body. For instance, what makes liver cells unique is, in part, their expression of a particular microRNA that influences which protein is produced. [click to continue…]