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Antisense RNA design, delivery, and analysis / edited by Virginia Arechavala-Gomeza, Alejandro Garanto.
New York, NY : Humana Press, 
xvii, 422 pages : illustrations (some color) ; 26 cm.
Methods in molecular biology (Clifton, N.J.) ; v. 2434.
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Springer protocols (Series)
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Methods in molecular biology, 1064-3745 ; 2434
This open access volume gathers a variety of models, delivery systems, and approaches that can be used to assess RNA technology for exploiting antisense as a therapeutic intervention. Beginning with a section on the design of antisense technology and their delivery, the book continues by covering model systems developed to evaluate efficacy, both in vivo and in vitro, as well as methods to evaluate preclinically the toxicity associated with these new potential drugs, and intellectual property considerations. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Antisense RNA Design, Delivery, and Analysis provides basic knowledge and a large collection of methods to facilitate the work of newcomers to this vibrant and expanding field. This book was conceived thanks to the network DARTER (Delivery of Antisense RNA Therapeutics). DARTER is funded by the EU Cooperation of Science and Technology (COST), which aims to enhance interaction and collaborations between researchers in Europe and other countries.
Includes bibliographical references and index.
Rights and reproductions note
Current copyright fee: GBP19.00 42\0.
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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Antisense RNA Design, Delivery, and Analysis [electronic resource] / edited by Virginia Arechavala-Gomeza, Alejandro Garanto.