Oligonucleotide-Based Therapies: Methods and Protocols

ISBN-13: 9781493996698 / Angielski / Twarda / 2019 / 344 str.

Olof Gissberg; Rula Zain; Karin E. Lundin

Spis treści

Part I: Reviews

 

1. Chemical Development of Therapeutic Oligonucleotides

            Karin E. Lundin, Olof Gissberg, C.I. Edvard Smith, and Rula Zain

 

2. Tissue-Specific Delivery of Oligonucleotides

            Xin Xia, Nicolette Pollock, Jiehua Zhou, and John Rossi

 

Part II: Protocols and Strategies

 

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

            Linda Vidarsdottir, Oksana Goroshchuk, Iryna Kolosenko, and Caroline Palm-Apergi

 

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

            Cristina S.J. Rocha

 

5. Assessing Oligonucleotide Binding to Double-Strand DNA

            Negin Mozafari and Tea Umek

 

6. Modelling and Simulation of Oligonucleotide Hybrids: Outlining a Strategy

            Lennart Nilsson and Alessandra Villa

 

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

            Kärt Padari, Ly Porosk, Piret Arukuusk, and Margus Pooga

 

8. Click-Shielded and Targeted Lipopolyplexes

            Philipp Michael Klein and Ernst Wagner

 

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

            Malgorzata Honcharenko, Dmytro Honcharenko, and Roger Stromberg

 

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

            Elsa M. Reyes-Reyes and Paula J. Bates

 

11. 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, and Sterghios A. Moschos

 

12. 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, and Ana P. Pêgo

 

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

            Suzan M. Hammond, Frank Abendroth, Michael J. Gait, and Matthew J.A. Wood

 

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

            Margherita Zamboni

 

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

            Sabine Sewing, Tanja Minz, and Franziska Boess

 

16. Identifying Suitable Target Regions and Analyzing Off-Target Effects

            Lykke Pedersen, Peter H. Hagedorn, and Troels Koch

 

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

            Louise Usher, Pantazis I. Theotokis, and Sterghios A. Moschos

 

18. Studying the Biotransformation of Phosphorothioate-Containing Oligonucleotide Drugs by LC-MS

            Christophe Husser, Erich Koller, Andreas Brink, and Simone Schadt

 

19. ADME: Assessing Pharmacokinetic-Pharmacodynamic Parameters of Oligonucleotides

            Rasmus Jansson-Löfmark*, Christine Ahlström, and Peter Gennemark

Blurb

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.