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Oligonucleotide-Based Therapies

Methods and Protocols

  • Book
  • © 2019

Overview

Editors:
  1. Olof Gissberg

    1. Center for Advanced Therapies, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden

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  2. Rula Zain

    1. Center for Advanced Therapies, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden

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    You can also search for this editor in PubMed   Google Scholar

  3. Karin E. Lundin

    1. Center for Advanced Therapies, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden

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    You can also search for this editor in PubMed   Google Scholar

  • Includes cutting-edge techniques
  • Provides step-by-step detail essential for reproducible results
  • Contains key implementation advice from the experts

Part of the book series: Methods in Molecular Biology (MIMB, volume 2036)

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Table of contents (19 protocols)

  1. Front Matter

    Pages i-xi
    Download chapter PDF

  2. Reviews

    1. Front Matter

      Pages 1-1
      Download chapter PDF

    2. Chemical Development of Therapeutic Oligonucleotides

      • Karin E. Lundin, Olof Gissberg, C. I. Edvard Smith, Rula Zain
      Pages 3-16

    3. Tissue-Specific Delivery of Oligonucleotides

      • Xin Xia, Nicolette Pollock, Jiehua Zhou, John Rossi
      Pages 17-50

  3. Protocols and Strategies

    1. Front Matter

      Pages 51-51
      Download chapter PDF

    2. Designing siRNA and Evaluating Its Effect on RNA Targets Using qPCR and Western Blot

      • Linda Vidarsdottir, Oksana Goroshchuk, Iryna Kolosenko, Caroline Palm-Apergi
      Pages 53-72

    3. Antisense Oligonucleotides for Splice Modulation: Assessing Splice Switching Efficacy

      • Cristina S. J. Rocha
      Pages 73-90

    4. Assessing Oligonucleotide Binding to Double-Stranded DNA

      • Negin Mozafari, Tea Umek
      Pages 91-112

    5. Modeling and Simulation of Oligonucleotide Hybrids: Outlining a Strategy

      • Lennart Nilsson, Alessandra Villa
      Pages 113-126

    6. Characterization of Peptide–Oligonucleotide Complexes Using Electron Microscopy, Dynamic Light Scattering, and Protease Resistance Assay

      • Kärt Padari, Ly Porosk, Piret Arukuusk, Margus Pooga
      Pages 127-139

    7. Click-Shielded and Targeted Lipopolyplexes

      • Philipp Michael Klein, Ernst Wagner
      Pages 141-164

    8. Attachment of Peptides to Oligonucleotides on Solid Support Using Copper(I)-Catalyzed Huisgen 1,3-Dipolar Cycloaddition

      • Malgorzata Honcharenko, Dmytro Honcharenko, Roger Strömberg
      Pages 165-171

    9. Characterizing Oligonucleotide Uptake in Cultured Cells: A Case Study Using AS1411 Aptamer

      • Elsa M. Reyes-Reyes, Paula J. Bates
      Pages 173-186

    10. Measuring the Action of Oligonucleotide Therapeutics in the Lung at the Cell Type-Specific Level by Tissue Disruption and Cell Sorting (TDCS)

      • Helen Graves, Steven Evans, Michael Fauler, Manfred Frick, Sterghios A. Moschos
      Pages 187-203Open Access

    11. Delivery of Antisense Oligonucleotides Mediated by a Hydrogel System: In Vitro and In Vivo Application in the Context of Spinal Cord Injury

      • Pedro M. D. Moreno, Teresa Rodrigues, Marília Torrado, Isabel F. Amaral, Ana P. Pêgo
      Pages 205-219

    12. Evaluation of Cell-Penetrating Peptide Delivery of Antisense Oligonucleotides for Therapeutic Efficacy in Spinal Muscular Atrophy

      • Suzan M. Hammond, Frank Abendroth, Michael J. Gait, Matthew J. A. Wood
      Pages 221-236

    13. Injection and Infusion of Compounds to the Central Nervous System

      • Margherita Zamboni
      Pages 237-248

    14. In Vitro Assessment of the Hepatotoxicity Potential of Therapeutic Oligonucleotides

      • Sabine Sewing, Tanja Minz, Franziska Boess
      Pages 249-259

    15. Identifying Suitable Target Regions and Analyzing Off-Target Effects of Therapeutic Oligonucleotides

      • Lykke Pedersen, Peter H. Hagedorn, Troels Koch
      Pages 261-282

    16. RACE-SEQ and Population-Wide Polymorphism Susceptibility Testing for Endonucleolytically Active, RNA-Targeting Therapeutics

      • Louise Usher, Pantazis I. Theotokis, Sterghios A. Moschos
      Pages 283-305
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Keywords

About this book

This book provides a collection of comprehensive, up-to-date, and broadly applicable guides to the research and development fields of oligonucleotide (ON) therapeutics. Covering topics from the study of antisense and anti-gene effects to oligonucleotides in the context of drug discovery and development, the volume explores a wide-ranging and useful spectrum of methods and protocols needed to take full advantage of therapeutic applications involving ONs. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. 

Authoritative and practical, Oligonucleotide-Based Therapies: Methods and Protocols aims to be a great aid in the laboratory as well as an ideal reference guide when designing antisense and anti-gene oligonucleotides for therapeutic applications.

Editors and Affiliations

  • Center for Advanced Therapies, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden

    Olof Gissberg, Rula Zain, Karin E. Lundin

Bibliographic Information

  • Book Title: Oligonucleotide-Based Therapies

  • Book Subtitle: Methods and Protocols

  • Editors: Olof Gissberg, Rula Zain, Karin E. Lundin

  • Series Title: Methods in Molecular Biology

  • DOI: https://doi.org/10.1007/978-1-4939-9670-4

  • Publisher: Humana New York, NY

  • eBook Packages: Springer Protocols

  • Copyright Information: Springer Science+Business Media, LLC, part of Springer Nature 2019

  • Hardcover ISBN: 978-1-4939-9669-8Published: 14 August 2019

  • Softcover ISBN: 978-1-4939-9672-8Published: 15 August 2020

  • eBook ISBN: 978-1-4939-9670-4Published: 13 August 2019

  • Series ISSN: 1064-3745

  • Series E-ISSN: 1940-6029

  • Edition Number: 1

  • Number of Pages: XI, 344

  • Number of Illustrations: 23 b/w illustrations, 46 illustrations in colour

  • Topics: Laboratory Medicine, Human Genetics, Pharmacology/Toxicology

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