Translational Research in Muscular Dystrophy

• Corporate Author: SpringerLink (Online service)
• Other Authors: Takeda, Shin'ichi (Editor), Miyagoe-Suzuki, Yuko (Editor), Mori-Yoshimura, Madoka (Editor)
• Format: Electronic eBook
• Language: English
• Published: Tokyo : Springer Japan : Imprint: Springer, 2016.
• Edition: 1st ed. 2016.
• Subjects: Medicine.; Gene therapy.; Molecular biology.; Neurology.; Medicine & Public Health.; Molecular Medicine.; Gene Therapy.

This book presents recent advances in translational research on muscular dystrophy (MD) to physicians and researchers, including cutting-edge research on the disease such as regenerative medicine, next-generation DNA sequencing, and nucleic acid therapies. It also describes the current systems for c...

Table of Contents:

• 1. Fukuyama congenital muscular dystrophy - Clinical aspects
• 2. α-Dystroglycanopathy
• 3. Myotonic dystrophy
• 4. Molecular pathogenesis and therapeutic strategy in GNE myopathy
• 5. Targeting the type I TGF-β receptor for treating caveolin-3-deficient autosomal dominant limb-girdle muscular dystrophy type 1C and muscle wasting disorders
• 6. Translational Research in Nucleic Acid Therapies for Muscular Dystrophies
• 7. Toward regenerative medicine for muscular dystrophies-Lessons from regeneration processes-
• 8. Stem cell-based therapy for Duchenne muscular dystrophy
• 9. Therapeutic approach of iPS cell technology for treating muscular dystrophy
• 10. Clinical aspects of GNE myopathy and translational medicine
• 11. Patient registries for international harmonized clinical development
• 12. Muscular Dystrophy Clinical Trial Network in Japan
• 13. Translational research on DMD in Japan From mice to exploratory investigator–initiated clinical trial in humans. .