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قابل دانلود از يكشنبه, ۲۰ فروردين ۱۴۰۲
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فهرست مطالب: Preface . . . . . . . . . . . . . . . . . . . vAcknowledgments. . . . . . . . . . . . . . . . . vii Contributors. . . . . . . . . . . . . . . . .. . . . . . xiii PART I OVERVIEW 1 Introduction and History of the Chemistry of Nucleic Acids Therapeutics . . . . . 3 Michael J. Gait and Sudhir Agrawal 2 Antisense RNA Therapeutics: A Brief Overview. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Virginia Arechavala-Gomeza and Alejandro Garanto PART II DESIGN AND FORMULATION OF ANTISENSE TECHNOLOGY 3 Design of Bifunctional Antisense Oligonucleotides for Exon Inclusion . . . . . . . . 53 Haiyan Zhou 4 Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translation . . . . . . .. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63 Michele Arnoldi, Giulia Zarantonello, Stefano Espinoza, Stefano Gustincich, Francesca Di Leva, and Marta Biagioli 5 How to Design U1 snRNA Molecules for Splicing Rescue . . . . . . . . . . . . . . . . . . . 89 Liliana Matos, Juliana I. Santos, Mª. Francisca Coutinho, and Sandra Alves 6 Conjugation of Nucleic Acids and Drugs to Gold Nanoparticles. . . . . . . . . . . . . . 103 Paula Mila´n-Rois, Ciro Rodriguez-Diaz, Milagros Castellanos, and A´ lvaro Somoza 7 Determination of Optimum Ratio of Cationic Polymers and Small Interfering RNA with Agarose Gel Retardation Assay . . . . . . . . . . . . . . . . . . . . . . . 117 Omer Aydin, Dilek Kanarya, Ummugulsum Yilmaz, and Cansu U mran Tunc € ¸ 8 Generation of Protein-Phosphorodiamidate Morpholino Oligomer Conjugates for Efficient Cellular Delivery via Anthrax Protective Antigen. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 129 Valentina Palacio-Castan˜eda, Roland Brock, and Wouter P. R. Verdurmen PART III IN VITRO MODEL SYSTEMS 9 Development and Use of Cellular Systems to Assess and Correct Splicing Defects . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 145 Nuria Sua´rez-Herrera, Tomasz Z. Tomkiewicz, Alejandro Garanto, and Rob W. J. Collin 10 Modeling Splicing Variants Amenable to Antisense Therapy by Use of CRISPR-Cas9-Based Gene Editing in HepG2 Cells. . . . . . . . . . . . . . . . 167 Arı´stides Lopez-Ma´rquez, Ainhoa Martı´nez-Pizarro, Bele´n Pe´rez, Eva Richard, and Lourdes R. Desviat 11 In Vitro Models for the Evaluation of Antisense Oligonucleotides in Skin. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185 Jeroen Bremer and Peter C. van den Akker 12 In Vitro Delivery of PMOs in Myoblasts by Electroporation . . . . . . . . . . . . . . . . . 191 Remko Goossens and Annemieke Aartsma-Rus 13 Rapid Determination of MBNL1 Protein Levels by Quantitative Dot Blot for the Evaluation of Antisense Oligonucleotides in Myotonic Dystrophy Myoblasts . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 207 Nerea Moreno, Irene Gonza´lez-Martı´nez, Rube´n Artero, and Estefanı´a Cerro-Herreros 14 Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystrophy . . . . . . . . . . . . . . . . . . . . . . . 217 Andrea Lopez-Martı´nez, Patricia Soblechero-Martı´n, and Virginia Arechavala-Gomeza 15 Generation of Human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 235 Pablo Herrero-Hernandez, Atze J. Bergsma, and W. W. M. Pim Pijnappel 16 Eye on a Dish Models to Evaluate Splicing Modulation . . . . . . . . . . . . . . . . . . . . . 245 Kwan-Leong Hau, Amelia Lane, Rosellina Guarascio, and Michael E. Cheetham 17 Establishment of In Vitro Brain Models for AON Delivery. . . . . . . . . . . . . . . . . . . 257 Elena Daoutsali and Ronald A. M. Buijsen PART IV IN VIVO MODEL SYSTEMS 18 Considerations for Generating Humanized Mouse Models to Test Efficacy of Antisense Oligonucleotides. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 267 Irene Va´zquez-Domı´nguez and Alejandro Garanto 19 Generation of Humanized Zebrafish Models for the In Vivo Assessment of Antisense Oligonucleotide-Based Splice Modulation Therapies. . . . . . . . . . . . . 281 Renske Schellens, Erik de Vrieze, Ralph Slijkerman, Hannie Kremer, and Erwin van Wijk 20 Use of Small Animal Models for Duchenne and Parameters to Assess Efficiency upon Antisense Treatment . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 301 Ngoc Lu-Nguyen, Alberto Malerba, and Linda Popplewell 21 In Vivo Models for the Evaluation of Antisense Oligonucleotides in Skin . . . . . . 315 Jeroen Bremer and Peter C. van den Akker 22 Delivery of Antisense Oligonucleotides to the Mouse Retina . . . . . . . . . . . . . . . . . 321 Alejandro Garanto 23 Delivery of Antisense Oligonucleotides to the Mouse Brain by Intracerebroventricular Injections . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 333 Tom Metz, Elsa C. Kuijper, and Willeke M. C. van Roon-Mom PART V SAFETY AND TOXICOLOGY 24 Intrathecal Delivery of Therapeutic Oligonucleotides for Potent Modulation of Gene Expression in the Central Nervous System . . . . . . . . . . . . . . 345 Zachary Kennedy, James W. Gilbert, and Bruno M. D. C. Godinho 25 Preclinical Safety Assessment of Therapeutic Oligonucleotides. . . . . . . . . . . . . . . . 355 Patrik Andersson 26 Preclinical Evaluation of the Renal Toxicity of Oligonucleotide Therapeutics in Mice . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 371 Lucı´a Echevarrı´a and Aurelie Goyenvalle 27 Protocol for Isolation and Culture of Mouse Hepatocytes (HCs), Kupffer Cells (KCs), and Liver Sinusoidal Endothelial Cells (LSECs) in Analyses of Hepatic Drug Distribution. . . . . . . . . . . . . . . . . . . . . . . . . . 385 Kjetil Elvevold, Ingelin Kyrrestad, and Ba˚rd Smedsrød PART VI INTELLECTUAL PROPERTY 28 Patent Considerations When Embarking on New Antisense Drug Programs . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405 Laurence D. S. Gainey Index . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419 مشخصات فایل |
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| عنوان (Title): | Antisense RNA Design, Delivery, and Analysis |
| نام فایل (File name): | 906-www.GeneProtocols.ir-Antisense RNA Design, Delivery, and Analysis-Humana Press (2022).pdf |
| عنوان فارسی (Title in Persian): | طراحی، تحویل و تجزیه و تحلیل RNA آنتی سنس |
| ایجاد کننده: | Virginia Arechavala-Gomeza, Alejandro Garanto (eds.) |
| زبان (Language): | انگلیسی English |
| سال انتشار: | 2022 |
| شابک ISBN: | 9781071620090, 9781071620106 |
| نوع سند (Doc. type): | کتاب |
| فرمت (File extention): | |
| حجم فایل (File size): | 11 مگابایت |
| تعداد صفحات (Book length in pages): | 416 |
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