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Antisense RNA Design, Delivery, and Analysis
Published 2022Table of Contents: “…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 Intracerebroventricular Injections -- Intrathecal Delivery of Therapeutic Oligonucleotides for Potent Modulation of Gene Expression in the Central Nervous System -- Preclinical Safety Assessment of Therapeutic Oligonucleotides -- Preclinical Evaluation of the Renal Toxicity of Oligonucleotide Therapeutics in Mice -- Protocol for Isolation and Culture of Mouse Hepatocytes (HCs), Kupffer Cells (KCs), and Liver Sinusoidal Endothelial Cells (LSECs) in Analyses of Hepatic Drug Distribution -- Patent Considerations When Embarking on New Antisense Drug Programs.…”
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