Antisense RNA Design, Delivery, and Analysis

ISBN-13: 9781071620120 / Angielski / Miękka / 2022

Spis treści

Part I: Overview

 

1. Introduction and History of the Chemistry of Nucleic Acids Therapeutics

            Michael J. Gait and Sudhir Agrawal

 

2. Antisense RNA Therapeutics: A Brief Overview

            Virginia Arechavala-Gomeza and Alejandro Garanto

 

Part II: Design and Formulation of Antisense Technology

 

3. Design of Bifunctional Antisense Oligonucleotides for Exon Inclusion

            Haiyan Zhou

 

4. Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translation

            Michele Arnoldi, Giulia Zarantonello, Stefano Espinoza, Stefano Gustincich, Francesca Di Leva, and Marta Biagioli

 

5. How to Design U1 snRNA Molecules for Splicing Rescue

            Liliana Matos, Juliana I. Santos, Mª Francisca Coutinho, and Sandra Alves

 

6. Conjugation of Nucleic Acids and Drugs to Gold Nanoparticles

            Paula Milán-Rois, Ciro Rodriguez-Diaz, Milagros Castellanos, and Álvaro Somoza

 

7. Determination of Optimum Ratio of Cationic Polymers and Small Interfering RNA with Agarose Gel Retardation Assay

            Omer Aydin, Dilek Kanarya, Ummugulsum Yilmaz, and Cansu Umran Tunc

 

8. Generation of Protein-Phosphorodiamidate Morpholino Oligomer Conjugates for Efficient Cellular Delivery via Anthrax Protective Antigen

            Valentina Palacio-Castañeda, 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

            Nuria Suárez-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

            Arístides López-Márquez, Ainhoa Martínez-Pizarro, Belén Pérez, Eva Richard, and Lourdes R. Desviat

 

11. In Vitro Models for the Evaluation of Antisense Oligonucleotides in Skin

            Jeroen Bremer and Peter C. van den Akker

 

12. In Vitro Delivery of PMOs in Myoblasts by Electroporation

            Remko Goossens and Annemieke Aartsma-Rus

 

13. Rapid Determination of MBNL1 Protein Levels by Quantitative Dot Blot for Evaluation of Antisense Oligonucleotides in Myotonic Dystrophy Myoblasts

            Nerea Moreno-Cervera, Irene Gonzalez-Martinez, Rubén Artero, and Estefanía Cerro-Herreros

 

14. Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystrophy

            Andrea López-Martínez, Patricia Soblechero-Martín, and Virginia Arechavala-Gomeza

 

15. Generation of Human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitro

            Pablo Herrero-Hernandez, Atze J. Bergsma, and W.W.M. Pim Pijnappel

 

16. Eye on a Dish Models to Evaluate Splicing Modulation

            Kwan-Leong Hau, Amelia Lane, Rosellina Guarascio, and Michael E. Cheetham

 

17. Establishment of In Vitro Brain Models for AON Delivery

            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

            Irene Vázquez-Domínguez and Alejandro Garanto

 

19. Generation of Humanized Zebrafish Models for the In Vivo Assessment of Antisense Oligonucleotide-Based Splice Modulation Therapies

            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

            Ngoc Lu-Nguyen, Alberto Malerba, and Linda Popplewell

 

21. In Vivo Models for the Evaluation of Antisense Oligonucleotides in Skin

            Jeroen Bremer and Peter C. van den Akker

 

22. Delivery of Antisense Oligonucleotides to the Mouse Retina

            Alejandro Garanto

 

23. Delivery of Antisense Oligonucleotides to the Mouse Brain by Intracerebroventricular Injections

            Tom Metz, Elsa C. Kuijper, and Willeke M.C. van Roon-Mom

 

24. Intrathecal Delivery of Therapeutic Oligonucleotides for Potent Modulation of Gene Expression in the Central Nervous System

            Zachary Kennedy, James W. Gilbert, and Bruno M.D.C. Godinho

 

Part V: Safety and Toxicology

 

25. Preclinical Safety Assessment of Therapeutic Oligonucleotides

            Patrik Andersson

 

26. Preclinical Evaluation of the Renal Toxicity of Oligonucleotide Therapeutics in Mice

            Lucía Echevarría 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

            Kjetil Elvevold, Ingelin Kyrrestad, Karen Kristine Sørensen, and Bård Smedsrød

 

Part VI: Intellectual Property

 

28. Patent Considerations When Embarking on New Antisense Drug Programs

            Laurence D.S. Gainey

Blurb

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.