Part I: Basics and Introduction

1. Current Strategies of Muscular Dystrophy Therapeutics: An Overview

Kenji Rowel Q. Lim and Toshifumi Yokota

2. The Story of Viltolarsen: From Preclinical Studies to FDA Approval

Rohini Roy Roshmi and Toshifumi Yokota

Part II: Sample Preparation and Assessment

3. Rapid Freezing of Skeletal and Cardiac Muscles Using Isopentane Cooled with Liquid Nitrogen and Tragacanth Gum for Histological, Genetic, and Protein Expression Studies

Saeed Anwar and Toshifumi Yokota

4. Cardiac and Skeletal Muscle Pathology in the D2/mdx Mouse Model and Caveats Associated with the Quantification of Utrophin

Tahnee L. Kennedy and Hannah F. Dugdale

5. Physiological Assessment of Muscle, Heart, and Whole Body Function in the Canine Model of Duchenne Muscular Dystrophy

Chady H. Hakim, James Teixeira, Stacy Leach, and Dongsheng Duan

Part III: Antisense Oligonucleotides

6. Restoring Dystrophin Expression by Skipping Exon 6 and 8 in Neonatal Dystrophic Dogs

Md Nur Ahad Shah and Toshifumi Yokota

7. Restoring Dystrophin Expression with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes

Yusuke Echigoya and Toshifumi Yokota

8. Restoring Dystrophin Expression with Duchenne Muscular Dystrophy Exon 45 Skipping in Induced-Pluripotent Stem Cell-Derived Cardiomyocytes

Mitsuto Sato, Naoko Shiba, Daigo Miyazaki, Yuji Shiba, and Akinori Nakamura

9. Quantitative Evaluation of Exon Skipping in Urine-Derived Cells for Duchenne Muscular Dystrophy

Katsuhiko Kunitake, Chaitra Sathyaprakash, Norio Motohashi, and Yoshitsugu Aoki

10. Use of Glycine to Augment Exon Skipping and Cell Therapies for Duchenne Muscular Dystrophy

Gang Han, Caorui Lin, and HaiFang Yin

11. Morpholino-Mediated Exons 28-29 Skipping in Dysferlin

Saeed Anwar and Toshifumi Yokota

12. Knocking Down DUX4 in Immortalized Facioscapulohumeral Muscular Dystrophy Patient-Derived Muscle Cells

Kenji Rowel Q. Lim and Toshifumi Yokota

13. Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy

Jessica Stoodley, David Seone Miraz, Yahya Jad, Mathieu Fischer, Matthew J.A. Wood, and Miguel A. Varela

14. Developing Therapeutic Splice-Correcting Antisense Oligomers for Adult-Onset Pompe Disease with c.-32-13T>G Mutation

Kristin A. Ham, Russell D. Johnsen, Michel Tchan, Steve D. Wilton, and May T. Aung-Htut

Part IV: Gene Replacement Therapies