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Discovering the first microRNA-targeted drug
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 p...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
The Rockefeller University Press
2012
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3483128/ https://www.ncbi.nlm.nih.gov/pubmed/23109665 http://dx.doi.org/10.1083/jcb.201208082 |
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author | Lindow, Morten Kauppinen, Sakari |
author_facet | Lindow, Morten Kauppinen, Sakari |
author_sort | Lindow, Morten |
collection | PubMed |
description | 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. |
format | Online Article Text |
id | pubmed-3483128 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2012 |
publisher | The Rockefeller University Press |
record_format | MEDLINE/PubMed |
spelling | pubmed-34831282013-04-29 Discovering the first microRNA-targeted drug Lindow, Morten Kauppinen, Sakari J Cell Biol News 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. The Rockefeller University Press 2012-10-29 /pmc/articles/PMC3483128/ /pubmed/23109665 http://dx.doi.org/10.1083/jcb.201208082 Text en © 2012 Lindow and Kauppinen This article is distributed under the terms of an Attribution–Noncommercial–Share Alike–No Mirror Sites license for the first six months after the publication date (see http://www.rupress.org/terms). After six months it is available under a Creative Commons License (Attribution–Noncommercial–Share Alike 3.0 Unported license, as described at http://creativecommons.org/licenses/by-nc-sa/3.0/). |
spellingShingle | News Lindow, Morten Kauppinen, Sakari Discovering the first microRNA-targeted drug |
title | Discovering the first microRNA-targeted drug |
title_full | Discovering the first microRNA-targeted drug |
title_fullStr | Discovering the first microRNA-targeted drug |
title_full_unstemmed | Discovering the first microRNA-targeted drug |
title_short | Discovering the first microRNA-targeted drug |
title_sort | discovering the first microrna-targeted drug |
topic | News |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3483128/ https://www.ncbi.nlm.nih.gov/pubmed/23109665 http://dx.doi.org/10.1083/jcb.201208082 |
work_keys_str_mv | AT lindowmorten discoveringthefirstmicrornatargeteddrug AT kauppinensakari discoveringthefirstmicrornatargeteddrug |