Discovering the first microRNA-targeted drug
Research output: Contribution to journal › Journal article › Research › peer-review
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.
Original language | English |
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Journal | Journal of Cell Biology |
Volume | 199 |
Issue number | 3 |
Pages (from-to) | 407-412 |
Number of pages | 6 |
ISSN | 0021-9525 |
DOIs | |
Publication status | Published - 2012 |
ID: 48996204