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  • Book
  • Open Access
  • © 2022

Antisense RNA Design, Delivery, and Analysis

  • This book is open access, which means that you have free and unlimited access

  • Includes cutting-edge techniques

  • Provides step-by-step detail essential for reproducible results

  • Contains key implementation advice from the experts

Part of the book series: Methods in Molecular Biology (MIMB, volume 2434)

Buying options

Softcover Book USD 49.99
Price excludes VAT (USA)
Hardcover Book USD 59.99
Price excludes VAT (USA)

Table of contents (28 protocols)

  1. Front Matter

    Pages i-xvii
  2. Overview

    1. Front Matter

      Pages 1-1
    2. Introduction and History of the Chemistry of Nucleic Acids Therapeutics

      • Michael J. Gait, Sudhir Agrawal
      Pages 3-31Open Access
    3. Antisense RNA Therapeutics: A Brief Overview

      • Virginia Arechavala-Gomeza, Alejandro Garanto
      Pages 33-49Open Access
  3. Design and Formulation of Antisense Technology

    1. Front Matter

      Pages 51-51
    2. Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translation

      • Michele Arnoldi, Giulia Zarantonello, Stefano Espinoza, Stefano Gustincich, Francesca Di Leva, Marta Biagioli
      Pages 63-87Open Access
    3. How to Design U1 snRNA Molecules for Splicing Rescue

      • Liliana Matos, Juliana I. Santos, Mª. Francisca Coutinho, Sandra Alves
      Pages 89-102Open Access
    4. Conjugation of Nucleic Acids and Drugs to Gold Nanoparticles

      • Paula Milán-Rois, Ciro Rodriguez-Diaz, Milagros Castellanos, Álvaro Somoza
      Pages 103-116Open Access
    5. Determination of Optimum Ratio of Cationic Polymers and Small Interfering RNA with Agarose Gel Retardation Assay

      • Omer Aydin, Dilek Kanarya, Ummugulsum Yilmaz, Cansu Ümran Tunç
      Pages 117-128Open Access
    6. Generation of Protein-Phosphorodiamidate Morpholino Oligomer Conjugates for Efficient Cellular Delivery via Anthrax Protective Antigen

      • Valentina Palacio-Castañeda, Roland Brock, Wouter P. R. Verdurmen
      Pages 129-141Open Access
  4. In Vitro Model Systems

    1. Front Matter

      Pages 143-143
    2. Development and Use of Cellular Systems to Assess and Correct Splicing Defects

      • Nuria Suárez-Herrera, Tomasz Z. Tomkiewicz, Alejandro Garanto, Rob W. J. Collin
      Pages 145-165Open Access
    3. 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, Lourdes R. Desviat
      Pages 167-184Open Access
    4. In Vitro Models for the Evaluation of Antisense Oligonucleotides in Skin

      • Jeroen Bremer, Peter C. van den Akker
      Pages 185-190Open Access
    5. In Vitro Delivery of PMOs in Myoblasts by Electroporation

      • Remko Goossens, Annemieke Aartsma-Rus
      Pages 191-205Open Access
    6. Rapid Determination of MBNL1 Protein Levels by Quantitative Dot Blot for the Evaluation of Antisense Oligonucleotides in Myotonic Dystrophy Myoblasts

      • Nerea Moreno, Irene González-Martínez, Rubén Artero, Estefanía Cerro-Herreros
      Pages 207-215Open Access
    7. Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystrophy

      • Andrea López-Martínez, Patricia Soblechero-Martín, Virginia Arechavala-Gomeza
      Pages 217-233Open Access
    8. Generation of Human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitro

      • Pablo Herrero-Hernandez, Atze J. Bergsma, W. W. M. Pim Pijnappel
      Pages 235-243Open Access
    9. Eye on a Dish Models to Evaluate Splicing Modulation

      • Kwan-Leong Hau, Amelia Lane, Rosellina Guarascio, Michael E. Cheetham
      Pages 245-255Open Access

About this book

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.


  • RNA therapeutics
  • Antisense technology
  • Therapeutic design
  • Oligonucleotides
  • Model systems
  • Oligonucleotide-mediated toxicology
  • Open Access

Editors and Affiliations

  • Ikerbasque, Basque Foundation for Science, Bilbao, Spain

    Virginia Arechavala-Gomeza

  • Department of Pediatrics, Department of Human Genetics, Radboud University Medical Center, Nijmegen, The Netherlands

    Alejandro Garanto

Bibliographic Information

  • Book Title: Antisense RNA Design, Delivery, and Analysis

  • Editors: Virginia Arechavala-Gomeza, Alejandro Garanto

  • Series Title: Methods in Molecular Biology

  • DOI:

  • Publisher: Humana New York, NY

  • eBook Packages: Springer Protocols

  • Copyright Information: The Editor(s) (if applicable) and The Author(s) 2022

  • License: CC BY

  • Hardcover ISBN: 978-1-0716-2009-0Published: 26 February 2022

  • Softcover ISBN: 978-1-0716-2012-0Published: 26 February 2022

  • eBook ISBN: 978-1-0716-2010-6Published: 25 February 2022

  • Series ISSN: 1064-3745

  • Series E-ISSN: 1940-6029

  • Edition Number: 1

  • Number of Pages: XVII, 422

  • Number of Illustrations: 10 b/w illustrations, 59 illustrations in colour

  • Topics: Therapeutics, Nucleic Acid

Buying options

Softcover Book USD 49.99
Price excludes VAT (USA)
Hardcover Book USD 59.99
Price excludes VAT (USA)