Discovering the first microRNA-targeted drug-Reference-Cited by-同舟云学术

Discovering the first microRNA-targeted drug

Published:2012-10-29 Issue:3 Volume:199 Page:407-412
ISSN:1540-8140
Container-title:Journal of Cell Biology
language:en
Short-container-title:

Author:

Lindow Morten¹²,Kauppinen Sakari²³

Affiliation:

1. Department of Biology, The Bioinformatics Centre, University of Copenhagen, DK-2200 Copenhagen, Denmark

2. Santaris Pharma, DK-2970 Hørsholm, Denmark

3. Department of Health Science and Technology, Aalborg University Copenhagen, DK-2450 Copenhagen SV, Denmark

Abstract

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)–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.

Publisher

Rockefeller University Press

Subject

Cell Biology

Link

http://rupress.org/jcb/article-pdf/199/3/407/1358183/jcb_201208082.pdf

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