Oligonucleotide-Based Therapies

Methods and Protocols

AvOlof Gissberg,Rula Zain

Häftad, Engelska, 2020

Del i serien Methods in Molecular Biology

1 333 kr

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Beskrivning

This book provides a collection of comprehensive, up-to-date, and broadly applicable guides to the research and development fields of oligonucleotide (ON) therapeutics.

Produktinformation

Utgivningsdatum:2020-08-15
Mått:178 x 254 x 20 mm
Vikt:670 g
Format:Häftad
Språk:Engelska
Serie:Methods in Molecular Biology
Antal sidor:344
Förlag:Humana Press Inc.
ISBN:9781493996728

Utforska kategorier

Farmakologi inom Medicin
Genterapi inom Medicin

Innehållsförteckning

Chemical Development of Therapeutic Oligonucleotides.- Tissue-Specific Delivery of Oligonucleotides.- Designing siRNA and Evaluating Its Effect on RNA Targets Using qPCR and Western Blot.- Antisense Oligonucleotides for Splice Modulation: Assessing Splice Switching Efficacy.- Assessing Oligonucleotide Binding to Double-Strand DNA.- Modelling and Simulation of Oligonucleotide Hybrids: Outlining a Strategy.- Characterization of Peptide/Oligonucleotide Complexes Using Electron Microscopy, Dynamic Light Scattering, and Protease Resistance Assay.- Click-Shielded and Targeted Lipopolyplexes.- Attachment of Peptides to Oligonucleotides on Solid Support Using Copper(I)-Catalyzed Huisgen 1,3-Dipolar Cycloaddition.- Characterizing Oligonucleotide Uptake in Cultured Cells: A Case Study Using AS1411 Aptamer.- Measuring the Action of Oligonucleotide Therapeutics in the Lung at the Cell Type-Specific Level by Tissue Disruption and Cell Sorting (TDCS).- Delivery of Antisense Oligonucleotides Mediatedby a Hydrogel System: In Vitro and In Vivo Application in the Context of Spinal Cord Injury.- Evaluation of Cell-Penetrating Peptide Delivery of Antisense Oligonucleotides for Therapeutic Efficacy in Spinal Muscular Atrophy.- Injection and Infusion of Compounds to the Central Nervous System.- In Vitro Assessment of the Hepatotoxicity Potential of Therapeutic Oligonucleotides.- Identifying Suitable Target Regions and Analyzing Off-Target Effects.- RACE-SEQ and Population-Wide Polymorphism Susceptibility Testing for Endonucleolytically Active, RNA-Targeting Therapeutics.- Studying the Biotransformation of Phosphorothioate-Containing Oligonucleotide Drugs by LC-MS.- ADME: Assessing Pharmacokinetic-Pharmacodynamic Parameters of Oligonucleotides.