Drug Target miRNA Methods and Protocols
This volume provides a concise and technical discussion of recently developed approaches to overcome challenges, such as pharmacodynamics and pharmacokinetics difficulties, in miRNA drug discovery. These strategies cover anti-sense agents targeting miRNA
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Marco F. Schmidt Editor
Drug Target miRNA Methods and Protocols
METHODS
IN
MOLECULAR BIOLOGY
Series Editor John M. Walker School of Life and Medical Sciences University of Hertfordshire Hatfield, Hertfordshire, AL10 9AB, UK
For further volumes: http://www.springer.com/series/7651
Drug Target miRNA Methods and Protocols
Edited by
Marco F. Schmidt BEROCEUTICA GmbH, Potsdam, Germany
Editor Marco F. Schmidt BEROCEUTICA GmbH Potsdam, Germany
ISSN 1064-3745 ISSN 1940-6029 (electronic) Methods in Molecular Biology ISBN 978-1-4939-6561-8 ISBN 978-1-4939-6563-2 (eBook) DOI 10.1007/978-1-4939-6563-2 Library of Congress Control Number: 2016956244 © Springer Science+Business Media New York 2017 This work is subject to copyright. All rights are reserved by the Publisher, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilms or in any other physical way, and transmission or information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed. The use of general descriptive names, registered names, trademarks, service marks, etc. in this publication does not imply, even in the absence of a specific statement, that such names are exempt from the relevant protective laws and regulations and therefore free for general use. The publisher, the authors and the editors are safe to assume that the advice and information in this book are believed to be true and accurate at the date of publication. Neither the publisher nor the authors or the editors give a warranty, express or implied, with respect to the material contained herein or for any errors or omissions that may have been made. Printed on acid-free paper This Humana Press imprint is published by Springer Nature The registered company is Springer Science+Business Media LLC The registered company address is: 233 Spring Street, New York, NY 10013, U.S.A.
Preface 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 miRNAtargeting drug Miravirsen for the treatment of hepatitis C has been successful in clinical phase II trials. Addressing miRNAs as drug targets may enable the cure of diseases, which presently seems impossible. However, due to miRNAs’ chemical structure, generation of potential drug molecules with necessary pharmacokinetic properties is still challenging. Thus, Miravirsen remains an exception. In conclusion, drug target miRNA requires innovation at the level of drug discovery. Until today, several approaches have been developed to modulate miRNAs’ function in the hope of potential therapeutic use. After a short introduction to the field of miRNA drug discovery (Chapter 1) and to miRNA target identification (Chapter 2), the book summarizes the three strategies to overcome pharmacodynamics and pharmacokinetics challenges: Firstly, du
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