Antisense Rna Design Delivery And Analysis by ARECHAVALA-GOMEZA V., SPRINGER

Description

SPRINGER Antisense Rna Design Delivery And Analysis by ARECHAVALA-GOMEZA V.

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. _x000D_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. _x000D_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._x000D_ Table of Contents :- _x000D_ Part I: Overview_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 1. Introduction and History of the Chemistry of Nucleic Acids Therapeutics_x000D_ _x000D_ _x000D_ _x000D_ Michael J. Gait and Sudhir Agrawal_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 2. Antisense RNA Therapeutics: A Brief Overview_x000D_ _x000D_ _x000D_ _x000D_ Virginia Arechavala-Gomeza and Alejandro Garanto_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ Part II: Design and Formulation of Antisense Technology_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 3. Design of Bifunctional Antisense Oligonucleotides for Exon Inclusion_x000D_ _x000D_ _x000D_ _x000D_ Haiyan Zhou_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 4. Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translation_x000D_ _x000D_ _x000D_ _x000D_ Michele Arnoldi, Giulia Zarantonello, Stefano Espinoza, Stefano Gustincich, Francesca Di Leva, and Marta Biagioli_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 5. How to Design U1 snRNA Molecules for Splicing Rescue_x000D_ _x000D_ _x000D_ _x000D_ Liliana Matos, Juliana I. Santos, M Francisca Coutinho, and Sandra Alves_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 6. Conjugation of Nucleic Acids and Drugs to Gold Nanoparticles_x000D_ _x000D_ _x000D_ _x000D_ Paula Milan-Rois, Ciro Rodriguez-Diaz, Milagros Castellanos, and Alvaro Somoza_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 7. Determination of Optimum Ratio of Cationic Polymers and Small Interfering RNA with Agarose Gel Retardation Assay_x000D_ _x000D_ _x000D_ _x000D_ Omer Aydin, Dilek Kanarya, Ummugulsum Yilmaz, and Cansu Umran Tunc_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 8. Generation of Protein-Phosphorodiamidate Morpholino Oligomer Conjugates for Efficient Cellular Delivery via Anthrax Protective Antigen_x000D_ _x000D_ _x000D_ _x000D_ Valentina Palacio-Castaneda, Roland Brock, and Wouter P.R. Verdurmen_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ Part III: In Vitro Model Systems_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 9. Development and Use of Cellular Systems to Assess and Correct Splicing Defects_x000D_ _x000D_ _x000D_ _x000D_ Nuria Suarez-Herrera, Tomasz Z. Tomkiewicz, Alejandro Garanto, and Rob W.J. Collin_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 10. Modeling Splicing Variants Amenable to Antisense Therapy by Use of CRISPR-Cas9-Based Gene Editing in HepG2 Cells_x000D_ _x000D_ _x000D_ _x000D_ Aristides Lopez-Marquez, Ainhoa Martinez-Pizarro, Belen Perez, Eva Richard, and Lourdes R. Desviat_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 11. In Vitro Models for the Evaluation of Antisense Oligonucleotides in Skin_x000D_ _x000D_ _x000D_ _x000D_ Jeroen Bremer and Peter C. van den Akker_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 12. In Vitro Delivery of PMOs in Myoblasts by Electroporation_x000D_ _x000D_ _x000D_ _x000D_ Remko Goossens and Annemieke Aartsma-Rus_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 13. Rapid Determination of MBNL1 Protein Levels by Quantitative Dot Blot for Evaluation of Antisense Oligonucleotides in Myotonic Dystrophy Myoblasts_x000D_ _x000D_ _x000D_ _x000D_ Nerea Moreno-Cervera, Irene Gonzalez-Martinez, Ruben Artero, and Estefania Cerro-Herreros_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 14. Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystrophy_x000D_ _x000D_ _x000D_ _x000D_ Andrea Lopez-Martinez, Patricia Soblechero-Martin, and Virginia Arechavala-Gomeza_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 15. Generation of Human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitro_x000D_ _x000D_ _x000D_ _x000D_ Pablo Herrero-Hernandez, Atze J. Bergsma, and W.W.M. Pim Pijnappel_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 16. Eye on a Dish Models to Evaluate Splicing Modulation_x000D_ _x000D_ _x000D_ _x000D_ Kwan-Leong Hau, Amelia Lane, Rosellina Guarascio, and Michael E. Cheetham_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 17. Establishment of In Vitro Brain Models for AON Delivery_x000D_ _x000D_ _x000D_ _x000D_ Elena Daoutsali and Ronald A.M. Buijsen_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ Part IV: In Vivo Model Systems_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 18. Considerations for Generating Humanized Mouse Models to Test Efficacy of Antisense Oligonucleotides_x000D_ _x000D_ _x000D_ _x000D_ Irene Vazquez-Dominguez and Alejandro Garanto_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 19. Generation of Humanized Zebrafish Models for the In Vivo Assessment of Antisense Oligonucleotide-Based Splice Modulation Therapies_x000D_ _x000D_ _x000D_ _x000D_ Renske Schellens, Erik de Vrieze, Ralph Slijkerman, Hannie Kremer, and Erwin van Wijk_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 20. Use of Small Animal Models for Duchenne and Parameters to Assess Efficiency upon Antisense Treatment_x000D_ _x000D_ _x000D_ _x000D_ Ngoc Lu-Nguyen, Alberto Malerba, and Linda Popplewell_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 21. In Vivo Models for the Evaluation of Antisense Oligonucleotides in Skin_x000D_ _x000D_ _x000D_ _x000D_ Jeroen Bremer and Peter C. van den Akker_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 22. Delivery of Antisense Oligonucleotides to the Mouse Retina_x000D_ _x000D_ _x000D_ _x000D_ Alejandro Garanto_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 23. Delivery of Antisense Oligonucleotides to the Mouse Brain by Intracerebroventricular Injections_x000D_ _x000D_ _x000D_ _x000D_ Tom Metz, Elsa C. Kuijper, and Willeke M.C. van Roon-Mom_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 24. Intrathecal Delivery of Therapeutic Oligonucleotides for Potent Modulation of Gene Expression in the Central Nervous System_x000D_ _x000D_ _x000D_ _x000D_ Zachary Kennedy, James W. Gilbert, and Bruno M.D.C. Godinho_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ Part V: Safety and Toxicology_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 25. Preclinical Safety Assessment of Therapeutic Oligonucleotides_x000D_ _x000D_ _x000D_ _x000D_ Patrik Andersson_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 26. Preclinical Evaluation of the Renal Toxicity of Oligonucleotide Therapeutics in Mice_x000D_ _x000D_ _x000D_ _x000D_ Lucia Echevarria and Aurelie Goyenvalle_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 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_x000D_ _x000D_ _x000D_ _x000D_ Kjetil Elvevold, Ingelin Kyrrestad, Karen Kristine Sorensen, and Bard Smedsrod_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ Part VI: Intellectual Property_x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ _x000D_ 28. Patent Considerations When Embarking on New Antisense Drug Programs_x000D_ _x000D_ _x000D_ _x000D_ Laurence D.S. Gainey_x000D_