Antisense RNA Design, Delivery, and Analysis
portes grátis
Antisense RNA Design, Delivery, and Analysis
Garanto, Alejandro; Arechavala-Gomeza, Virginia
Springer-Verlag New York Inc.
02/2022
422
Dura
Inglês
9781071620090
15 a 20 dias
1019
Descrição não disponível.
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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RNA therapeutics;Antisense technology;Therapeutic design;Oligonucleotides;Model systems;Oligonucleotide-mediated toxicology;Open Access
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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