Exon Skipping and Inclusion Therapies

Methods and Protocols

  • Toshifumi Yokota
  • Rika Maruyama

Part of the Methods in Molecular Biology book series (MIMB, volume 1828)

Table of contents

  1. Front Matter
    Pages i-xv
  2. Basics and Introductions

  3. Design of AONs and Strategy

    1. Front Matter
      Pages 77-77
    2. Golnoush Golshirazi, Lukasz Ciszewski, Ngoc Lu-Nguyen, Linda Popplewell
      Pages 91-124
  4. In Vitro Evaluation of Exon Skipping for DMD

    1. Front Matter
      Pages 125-125
    2. Kenji Rowel Q. Lim, Toshifumi Yokota
      Pages 127-139
    3. Akinori Nakamura, Yoshitsugu Aoki, Maria Tsoumpra, Toshifumi Yokota, Shin’ichi Takeda
      Pages 151-163
    4. Mingming Zhao, Emi Shoji, Hidetoshi Sakurai
      Pages 173-189
    5. Masataka Ifuku, Kumiko A. Iwabuchi, Masami Tanaka, Mandy Siu Yu Lung, Akitsu Hotta
      Pages 191-217
    6. Kane Greer, Sue Fletcher, Steve D. Wilton
      Pages 219-228
  5. In Vivo Evaluation of Exon Skipping for DMD

    1. Front Matter
      Pages 229-229
    2. Bo Wu, Mingxing Wang, Sapana Shah, Qi Long Lu
      Pages 231-247
    3. Monika Hiller, Pietro Spitali, Nicole Datson, Annemieke Aartsma-Rus
      Pages 249-262
    4. Yoshitaka Mizobe, Shouta Miyatake, Hotake Takizawa, Yuko Hara, Toshifumi Yokota, Akinori Nakamura et al.
      Pages 275-292
    5. Joseph Kim, Karl Clark, Carrie Barton, Robert Tanguay, Hong Moulton
      Pages 293-306
  6. Enhancing the Efficacy

    1. Front Matter
      Pages 307-307
    2. Florian Barthelemy, Dereck Wang, Stanley F. Nelson, M. Carrie Miceli
      Pages 309-326
    3. Gang Han, Xianjun Gao, Haifang Yin
      Pages 327-342
    4. Rika Maruyama, Yoshitsugu Aoki, Shin’ichi Takeda, Toshifumi Yokota
      Pages 365-379
    5. Karima Relizani, Aurelie Goyenvalle
      Pages 381-394
  7. Exon Inclusion for SMA

    1. Front Matter
      Pages 413-413
    2. Natalia N. Singh, Diou Luo, Ravindra N. Singh
      Pages 415-437
    3. Aleksander Touznik, Rika Maruyama, Toshifumi Yokota
      Pages 439-454
    4. Tejal Aslesh, Rika Maruyama, Toshifumi Yokota
      Pages 455-465
    5. Haiyan Zhou, Francesco Muntoni
      Pages 467-477
  8. Exon Skipping and Splice Modulation for Various Diseases

    1. Front Matter
      Pages 479-479
    2. Florian Barthélémy, Sébastien Courrier, Nicolas Lévy, Martin Krahn, Marc Bartoli
      Pages 489-496
    3. Greer K. Arthur, Glenn Cruse
      Pages 503-518
    4. Matthias Titeux, Sandrina Turczynski, Nathalie Pironon, Alain Hovnanian
      Pages 531-552
    5. Yuko Hara, Yoshitaka Mizobe, Shouta Miyatake, Hotake Takizawa, Tetsuya Nagata, Toshifumi Yokota et al.
      Pages 553-564
  9. Matthias Titeux, Sandrina Turczynski, Nathalie Pironon, Alain Hovnanian
    Pages E1-E1
  10. Back Matter
    Pages 565-569

About this book


This book presents a comprehensive collection of detailed state-of-the-art exon skipping and splices modulation protocols. Chapters detail 14 genetic diseases, AON-mediated therapies, and CRISPR/Cas9-mediated gene editing therapies. Written in the highly successful Methods in Molecular Biology series format, 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 cutting-edge, Exon Skipping and Inclusion Therapies: Methods and Protocols aims to help researchers initiate the development of next-generation therapies.


mRNA splicing iPS-derived muscle zebrafish Tricyclo-DNA myotonic dystrophy

Editors and affiliations

  • Toshifumi Yokota
    • 1
  • Rika Maruyama
    • 2
  1. 1.Medical GeneticsUniversity of AlbertaEdmontonCanada
  2. 2.Medical GeneticsUniversity of AlbertaEdmontonCanada

Bibliographic information

  • DOI
  • Copyright Information Springer Science+Business Media, LLC, part of Springer Nature 2018
  • Publisher Name Humana Press, New York, NY
  • eBook Packages Springer Protocols
  • Print ISBN 978-1-4939-8650-7
  • Online ISBN 978-1-4939-8651-4
  • Series Print ISSN 1064-3745
  • Series Online ISSN 1940-6029
  • Buy this book on publisher's site