Antisense RNA Design, Delivery, and Analysis

Introduction and History of the Chemistry of Nucleic Acids Therapeutics.- Antisense RNA Therapeutics: A Brief Overview.- Design of Bifunctional Antisense Oligonucleotides for Exon Inclusion.- Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translation.- How to Design U1 snRNA Molecules for Splicing Rescue.- Conjugation of Nucleic Acids and Drugs to Gold Nanoparticles.- Determination of Optimum Ratio of Cationic Polymers and Small Interfering RNA with Agarose Gel Retardation Assay.- Generation of Protein-Phosphorodiamidate Morpholino Oligomer Conjugates for Efficient Cellular Delivery via Anthrax Protective Antigen.- Development and Use of Cellular Systems to Assess and Correct Splicing Defects.- Modeling Splicing Variants Amenable to Antisense Therapy by Use of CRISPR-Cas9-Based Gene Editing in HepG2 Cells.- In Vitro Models for the Evaluation of Antisense Oligonucleotides in Skin.- In Vitro Delivery of PMOs in Myoblasts by Electroporation.- Rapid Determination of MBNL1 Protein Levels by Quantitative Dot Blot for Evaluation of Antisense Oligonucleotides in Myotonic Dystrophy Myoblasts.- Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystrophy.- Generation of Human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitro.- Eye on a Dish Models to Evaluate Splicing Modulation.- Establishment of In Vitro Brain Models for AON Delivery.- Considerations for Generating Humanized Mouse Models to Test Efficacy of Antisense Oligonucleotides.- Generation of Humanized Zebrafish Models for the In Vivo Assessment of Antisense Oligonucleotide-Based Splice Modulation Therapies.- Use of Small Animal Models for Duchenne and Parameters to Assess Efficiency upon Antisense Treatment.- In Vivo Models for the Evaluation of Antisense Oligonucleotides in Skin.- Delivery of Antisense Oligonucleotides to the Mouse Retina.- Delivery of Antisense Oligonucleotides to the Mouse Brain by