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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...

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Detalles Bibliográficos
Autores principales: Lindow, Morten, Kauppinen, Sakari
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Rockefeller University Press 2012
Materias:
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.
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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
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