1 The Faculty of Medicine, Aalborg University, VBN2 Department of Health Science and Technology, The Faculty of Medicine, Aalborg University, VBN3 unknown4 Department of Clinical Medicine, The Faculty of Medicine, Aalborg University, VBN
MicroRNAs (miRNAs) are important post-transcriptional regulators of nearly every biological process in the cell and play key roles in the pathogenesis of human disease. As a result, there are many drug discovery programs that focus on developing miRNA-based therapeutics. The most advanced of these programs targets the liver-expressed miRNA-122 using the locked nucleic acid (LNA)â€“modified antisense oligonucleotide miravirsen. Here, we describe the discovery of miravirsen, which is currently in phase 2 clinical trials for treatment of hepatitis C virus (HCV) infection.
Journal of Cell Biology, 2012, Vol 199, Issue 3, p. 407-412