Rna-Based Therapeutics and Delivery Mechanisms
Preface.................................................................................................................................... VII Chapter 1 Chemistry of Nucleic Acids Therapeutics: Introduction and History...........................1 Michael J. Gait and Sudhir Agrawal Chapter 2 Conjugation of Nucleic Acids and Drugs to Gold Nanoparticles................................ 30 Paula Milán-Rois, Ciro Rodriguez-Diaz, Milagros Castellanos and Álvaro Somoza Chapter 3 Rapid Determination of MBNL1 Protein Levels by Quantitative Dot Blot for the Evaluation of Antisense Oligonucleotides in Myotonic Dystrophy Myoblasts.................................................... 44 Nerea Moreno, Irene González-Martínez, Rubén Artero and Estefanía Cerro-Herreros Chapter 4 Modeling Splicing Variants Amenable to Antisense Therapy by Use of CRISPR-Cas9-Based Gene Editing in HepG2 Cells........................................... 53 Arístides López-Márquez, Ainhoa Martínez-Pizarro, Belén Pérez, Eva Richard and Lourdes R. Desviat Chapter 5 Establishment of In Vitro Brain Models for AON Delivery......................................................................................................................... 71 Elena Daoutsali and Ronald A. M. Buijsen Chapter 6 Antisense RNA Therapeutics: A Brief Overview............................................................ 79 Virginia Arechavala-Gomeza and Alejandro Garanto Chapter 7 In Vitro Models for the Evaluation of Antisense Oligonucleotides in Skin...................................................................................................... 96 Jeroen Bremer and Peter C. van den Akker Chapter 8 How to Design U1 snRNA Molecules for Splicing Rescue......................................... 102 Liliana Matos, Juliana I. Santos, Ma. Francisca Coutinho and Sandra Alves Chapter 9 Eye on a Dish Models to Evaluate Splicing Modulation............................................. 116 Kwan-Leong Hau, Amelia Lane, Rosellina Guarascio and Michael E. Cheetham Chapter 10 In Vitro Delivery of PMOs in Myoblasts by Electroporation..................................... 127 Remko Goossens and Annemieke Aartsma-Rus Chapter 11 Generation of Protein-Phosphorodiamidate Morpholino Oligomer Conjugates for Efficient Cellular Delivery via Anthrax Protective Antigen................................................................................................ 142 Valentina Palacio-Castañeda, Roland Brock and Wouter P. R. Verdurmen Chapter 12 Design of Bifunctional Antisense Oligonucleotides for Exon Inclusion...................................................................................................................... 155 Haiyan Zhou Chapter 13 Generation of Human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitro......................................................................................... 165 Pablo Herrero-Hernandez, Atze J. Bergsma and W. W. M. Pim Pijnappel Chapter 14 Determination of Optimum Ratio of Cationic Polymers and Small Interfering RNA with Agarose Gel Retardation Assay.................................... 174 Omer Aydin, Dilek Kanarya, Ummugulsum Yilmaz and Cansu Ümran Tunç Chapter 15 Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translation.............................................................................................. 186 Michele Arnoldi, Giulia Zarantonello, Stefano Espinoza, Stefano Gustincich, Francesca Di Leva and Marta Biagioli Chapter 16 Development and Use of Cellular Systems to Assess and Correct Splicing Defects..................................................................................................... 211 Nuria Suárez-Herrera, Tomasz Z. Tomkiewicz, Alejandro Garanto and Rob W. J. Collin Permissions List of Contributors Index Contents