Duchenne Muscular Dystrophy

Part I: Reviews and Overview Chapters

1. An Overview of Recent Therapeutics Advances for Duchenne Muscular Dystrophy

Jean K. Mah

2. Clinical Manifestations and Overall Management Strategies for Duchenne Muscular Dystrophy

Takeshi Tsuda

Part II: Skeletal and Cardiac Muscle in DMD

3. Cardiac Involvement in Duchenne Muscular Dystrophy and Related Dystrophinopathies

Sophie I. Mavrogeni, George Markousis-Mavrogenis, Antigoni Papavasiliou, George Papadopoulos, and Genovefa Kolovou

4. Characterization of the Inflammatory Response in Dystrophic Muscle Using Flow Cytometry

Jenna M. Kastenschmidt, Ileen Avetyan, and S. Armando Villalta

5. Imaging Analysis of the Neuromuscular Junction in Dystrophic Muscle

Stephen J.P. Pratt, Shama R. Iyer, Sameer B. Shah, and Richard M. Lovering

Part III: Omics Approaches

6. System Biology Approach: Gene Network Analysis for Muscular Dystrophy

Federica Censi, Giovanni Calcagnini, Eugenio Mattei, and Alessandro Giuliani

7. Proteomic Profiling of the Dystrophin-Deficient Brain

Sandra Murphy and Kay Ohlendieck

8. Probing the Pathogenesis of Duchenne Muscular Dystrophy Using Mouse Models

Alexander Morrison-Nozik and Saptarsi M. Haldar

Part IV: Exon Skipping and Antisense Oligonucleotides (ASOs)

9. Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy

Shouta Miyatake, Yoshitaka Mizobe, Hotake Takizawa, Yuko Hara, Toshifumi Yokota, Shin'ichi Takeda, and Yoshitsugu Aoki

10. Designing Effective Antisense Oligonucleotides for Exon Skipping

Takenori Shimo, Rika Maruyama, and Toshifumi Yokota

11. Identification of Splicing Factors Involved in DMD Exon Skipping Events Using an In Vitro RNA Binding Assay

Julie Miro, Cyril F. Bourgeois, Mireille Claustres, Michel Koenig, and Sylvie Tuffery-Giraud

12. The Use of Antisense Oligonucleotides for the Treatment of Duchenne Muscular Dystrophy

Karima Relizani and Aurelie Goyenvalle

13. PMO Delivery System Using Bubble Liposomes and Ultrasound Exposure for Duchenne Muscular Dystrophy Treatment

Ryo Suzuki, and Kazuo Maruyama

Part V: Biomarkers and Drug Discovery

14. Proton Nuclear Magnetic Resonance (¹H NMR) Spectroscopy-Based Analysis of Lipid Components in Serum / Plasma of Patients with Duchenne Muscular Dystrophy (DMD)

Niraj Kumar Srivastava

15. Test of Anti-Fibrotic Drugs in a Cellular Model of Fibrosis Based on Muscle-Derived Fibroblasts from Duchenne Muscular Dystrophy Patients

Simona Zanotti and Marina Mora

16. Flow Cytometry-Defined CD49d Expression in Circulating T-Lymphocytes is a Biomarker for Disease Progression in Duchenne Muscular Dystrophy

Wilson Savino, Fernanda Pinto-Mariz, and Vincent Mouly

Part VI: Last Discoveries and Future Prospectives

17. Advanced Methods to Study the Cross-Talk Between Fibro-Adipogenic Progenitors and Muscle Stem Cells

Luca Tucciarone, Usue Etxaniz, Martina Sandoná, Silvia Consalvi, Pier Lorenzo Puri, and V