Oligonucleotide-Based Therapies : Methods and Protocols - Gissberg, Olof.; Zain, Rula.; Lundin, Karin E.; SpringerLink (Online service) (New York, NY : Springer New York : Imprint: Humana, 2019)
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 Mediated by 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.
Keywords: Laboratory medicine; Human genetics; Pharmacology; Laboratory Medicine; Human Genetics; Pharmacology/Toxicology.