Translational Research in Muscular Dystrophy

Softcover Nachdruck of the original 1. Auflage 2016
ISBN: 978-4-431-56674-8
Verlag: Springer

Buch, Englisch, 199 Seiten, Previously published in hardcover, Format (B × H): 155 mm x 235 mm, Gewicht: 362 g

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 clinical trials and MD patient databases, resources, which will support the early realization of clinical application and improve patients’ quality of life. 

MD is the one of the most widely known inherited neuromuscular diseases and is classified into diverse types by symptoms, age of onset, mode of inheritance, and clinical progression. With the development of molecular biology, the occurrence mechanisms of each type of MD are gradually being elucidated. Although there is no known permanent cure yet, the stage of treatment research has now advanced to clinical trials.

Zielgruppe

Professional/practitioner

Autoren/Hrsg.

Takeda, Shin'ichi

Miyagoe-Suzuki, Yuko

Mori-Yoshimura, Madoka

Fachgebiete

• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Klinische und Innere Medizin Muskuloskelettales System, Bewegungsapparat
• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Klinische und Innere Medizin Neurologie, Klinische Neurowissenschaft

Inhaltsverzeichnis

1. Fukuyama congenital muscular dystrophy - Clinical aspects.- 2. a-Dystroglycanopathy.- 3. Myotonic dystrophy.- 4. Molecular pathogenesis and therapeutic strategy in GNE myopathy.- 5. Targeting the type I TGF-ß 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.