ENGLISH

Oligonucleotide-Based Therapies: Methods and Protocols

By Olof Gissberg , Rula Zain , Karin E. Lundin

Book information

Publisher
Springer New York;Humana
Year
2019
ISBN
978-1-4939-9669-8, 978-1-4939-9670-4
Language
english
Format
PDF
Filesize
9 MB (9212249 bytes)
Series
Methods in Molecular Biology 2036
Edition
1st ed. 2019
Pages
XI, 344\345
Time added
2020-02-08 04:40:58
Biomedicine; Laboratory Medicine; Human Genetics;

Description

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. Front Matter ....Pages i-xi Front Matter ....Pages 1-1 Chemical Development of Therapeutic Oligonucleotides (Karin E. Lundin, Olof Gissberg, C. I. Edvard Smith, Rula Zain)....Pages 3-16 Tissue-Specific Delivery of Oligonucleotides (Xin Xia, Nicolette Pollock, Jiehua Zhou, John Rossi)....Pages 17-50 Front Matter ....Pages 51-51 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 Antisense Oligonucleotides for Splice Modulation: Assessing Splice Switching Efficacy (Cristina S. J. Rocha)....Pages 73-90 Assessing Oligonucleotide Binding to Double-Stranded DNA (Negin Mozafari, Tea Umek)....Pages 91-112 Modeling and Simulation of Oligonucleotide Hybrids: Outlining a Strategy (Lennart Nilsson, Alessandra Villa)....Pages 113-126 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 Click-Shielded and Targeted Lipopolyplexes (Philipp Michael Klein, Ernst Wagner)....Pages 141-164 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 Characterizing Oligonucleotide Uptake in Cultured Cells: A Case Study Using AS1411 Aptamer (Elsa M. Reyes-Reyes, Paula J. Bates)....Pages 173-186 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 187-203 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 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 Injection and Infusion of Compounds to the Central Nervous System (Margherita Zamboni)....Pages 237-248 In Vitro Assessment of the Hepatotoxicity Potential of Therapeutic Oligonucleotides (Sabine Sewing, Tanja Minz, Franziska Boess)....Pages 249-259 Identifying Suitable Target Regions and Analyzing Off-Target Effects of Therapeutic Oligonucleotides (Lykke Pedersen, Peter H. Hagedorn, Troels Koch)....Pages 261-282 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 Studying the Biotransformation of Phosphorothioate-Containing Oligonucleotide Drugs by LC-MS (Christophe Husser, Erich Koller, Andreas Brink, Simone Schadt)....Pages 307-315 ADME: Assessing Pharmacokinetic–Pharmacodynamic Parameters of Oligonucleotides (Rasmus Jansson-Löfmark, Christine Ahlström, Peter Gennemark)....Pages 317-339 Back Matter ....Pages 341-344

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