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miRNA Targeting Drugs: The Next Blockbusters?

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Drug Target miRNA

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1517))

Abstract

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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Acknowledgement

The author thanks Dr. Rajavel Srinivasan for critical proofreading of the manuscript.

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Authors and Affiliations

  1. BEROCEUTICA GmbH, c/o Universität Potsdam, August-Bebel-Strasse 89, 14482, Potsdam, Germany

    Marco F. Schmidt

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  1. Marco F. Schmidt
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Correspondence to Marco F. Schmidt .

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  1. BEROCEUTICA GmbH, Potsdam, Germany

    Marco F. Schmidt

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Schmidt, M.F. (2017). miRNA Targeting Drugs: The Next Blockbusters?. In: Schmidt, M. (eds) Drug Target miRNA. Methods in Molecular Biology, vol 1517. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-6563-2_1

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  • DOI: https://doi.org/10.1007/978-1-4939-6563-2_1

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-6561-8

  • Online ISBN: 978-1-4939-6563-2

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