Translational Research in Muscular Dystrophy

Translational Research in Muscular Dystrophy

Takeda, Shin'ichi; Miyagoe-Suzuki, Yuko; Mori-Yoshimura, Madoka

Springer Verlag, Japan

03/2018

199

Mole

Inglês

9784431566748

15 a 20 dias

454

Descrição não disponível.
1. Fukuyama congenital muscular dystrophy - Clinical aspects.- 2. ?-Dystroglycanopathy.- 3. Myotonic dystrophy.- 4. Molecular pathogenesis and therapeutic strategy in GNE myopathy.- 5. Targeting the type I TGF-? 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.
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Duchenne muscular dystrophy;Molecular Pathogenesis;molecular therapy;neuromuscular disorders database;regenerative medicine;gene therapy