Abstract

MicroRNAs have been predicted to regulate the stability and translation of many target mRNAs that are involved in modulating disease outcome. Thus, valuable strategies to enhance or to diminish the function of microRNAs are needed to manipulate microRNA-mediated target gene expression. Recently, it has become apparent that one class of antisense oligonucleotides, locked nucleic acids, can be used to sequester microRNAs in the liver of a variety of animals including humans, opening the possibility of applying locked nucleic acid-mediated gene therapy. This review summarizes the success of sequestration of liver-specific microRNA miR-122 by antisense locked nucleic acids and their use in combating hepatitis C virus in clinical trials.

Keywords: Clinical trials, gene targeting, hepatitis C virus, locked nucleic acids, microRNA-mRNA interactions, viral gene expression.