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008			220225s2022 xxua o |||| 0|eng d
020			_a9781071620106
024	7		_a10.1007/978-1-0716-2010-6 _2doi
040			_aES-MaUEC _bspa _cES-MaUEC _dES-MaUEC
050		4	_aQP623 _b2022 EB
245	0	0	_aAntisense RNA Design, Delivery, and Analysis _cedited by Virginia Arechavala-Gomeza, Alejandro Garanto
250			_a1st edition 2022
264		1	_aNew York, NY _bSpringer International Publising _c2022
300			_a1 recurso en línea (XVII, 422 páginas) _b69 ilustraciones, 59 ilustraciones a color
336			_atexto _btxt _2rdacontent
337			_aelectrónico _bc _2rdamedia
338			_arecurso electrónico _bcr _2rdacarrier
347			_aarchivo de texto _bPDF
490	0		_aMethods in Molecular Biology _x1940-6029 _v2434
505	0		_aIntroduction 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.
506	0		_aOpen Access
520			_aThis 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.
988			_aSpringer_Protocols_2022
650		7	_2embne _9144940 _aARN
650		7	_2embne _9138037 _aTerapéutica
776	0	8	_iPrinted edition: _z9781071620090
776	0	8	_iPrinted edition: _z9781071620113
776	0	8	_iPrinted edition: _z9781071620120
856	4	0	_uhttps://go.openathens.net/redirector/universidadeuropea.es?url=https://doi.org/10.1007/978-1-0716-2010-6 _zAcceso a este recurso digital (usuarios Universidad Europea de Madrid)
942			_2lcc _cLE
998			_b07/2023 _dz _eb _zSI