Oligonucleotide-Based Therapies

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.