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miRNA Targeting Drugs

  • Only 20 years after the discovery of small non-coding, single-stranded ribonucleic acids, so-called microRNAs (miRNAs), as post-transcriptional gene regulators, the first miRNA-targeting drug Miravirsen for the treatment of hepatitis C has been successfully tested in clinical Phase II trials. Addressing miRNAs as drug targets may enable the cure, or at least the treatment of diseases, which presently seems impossible. However, due to miRNAs’ chemical structure, generation of potential drug molecules with necessary pharmacokinetic properties is still challenging and requires a re-thinking of the drug discovery process. Therefore, this chapter highlights the potential of miRNAs as drug targets, discusses the challenges, and tries to give a complete overview of recent strategies in miRNA drug discovery.

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Metadaten
Author details:Marco F. SchmidtORCiDGND
DOI:https://doi.org/10.1007/978-1-4939-6563-2_1
ISBN:978-1-4939-6563-2
ISBN:978-1-4939-6561-8
ISSN:1064-3745
ISSN:1940-6029
Pubmed ID:https://pubmed.ncbi.nlm.nih.gov/27924471
Title of parent work (English):Drug Target miRNA: Methods and Protocols
Subtitle (English):the next blockbusters?
Publisher:Springer
Place of publishing:New York
Publication type:Article
Language:English
Date of first publication:2016/12/07
Publication year:2017
Release date:2022/10/11
Tag:Antisense agents; Argonaute 2 protein; Drug discovery; Small-molecule miRNA modulators; miRNA; microRNA-induced silencing complex
Volume:1517
Number of pages:20
First page:3
Last Page:22
Organizational units:Mathematisch-Naturwissenschaftliche Fakultät / Institut für Biochemie und Biologie
DDC classification:5 Naturwissenschaften und Mathematik / 57 Biowissenschaften; Biologie / 570 Biowissenschaften; Biologie
6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Peer review:Referiert
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