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
