[Development of therapies against neuromuscular diseases causing muscle atrophy].
Skeletal muscles become atrophied by muscular disorders such as muscular dystrophy, wasting and even aging. In addition to muscle atrophy, progressive muscle damage, inflammation and replacement of muscle fibers with fibrous and fatty tissues are observed in muscular dystrophy. Neuronal innervation is required for skeletal muscle, and muscles become atrophic when motor neurons are affected by neurodegenerative disorders such as amyotrophic lateral sclerosis. Restoring muscle mass and function lost by diseases such as muscular dystrophy and neurodegenerative disorders is important. There are three rational therapies for muscular dystrophy and related diseases: gene therapy, cell therapy and drug therapy. Gene therapies to replace the defective genes have been tried with various degrees of effectiveness. Multiple myogenic stem cells including satellite cells, bone marrow cells, muscle side population cells, muscle-derived stem cells and mesoangioblast have been characterized. Cell therapies using these stem cells are one of the promising therapies for neuromuscular diseases causing muscle atrophy. As pharmacological drug therapies, increasing skeletal muscle mass by myostatin inhibition is quite promising and will be applied clinically in the near future.
Tsuchida
Kunihiro
K
Division for Therapies Against Intractable Diseases, Institute for Comprehensive Medical Science (ICMS), Fujita Health University, Toyoake, 470-1192 Japan. tsuchida@fujita-hu.ac.jp
D000690:Amyotrophic Lateral Sclerosis / Q000235:genetics / Q000628:therapy; D000818:Animals; D002454:Cell Differentiation / Q000235:genetics / Q000502:physiology; D002455:Cell Division / Q000235:genetics / Q000502:physiology; D015195:Drug Design; D015316:Genetic Therapy; D020662:Guanine Nucleotide Exchange Factors / Q000235:genetics; D006801:Humans; D018482:Muscle, Skeletal / Q000166:cytology / Q000196:embryology; D009133:Muscular Atrophy / Q000235:genetics / Q000628:therapy; D009154:Mutation; D055435:Myostatin; D009468:Neuromuscular Diseases / Q000235:genetics / Q000628:therapy*; D015398:Signal Transduction / Q000235:genetics / Q000502:physiology; D033581:Stem Cell Transplantation; D013482:Superoxide Dismutase / Q000235:genetics; D000072105:Superoxide Dismutase-1; D016212:Transforming Growth Factor beta / Q000037:antagonists & inhibitors* / Q000276:immunology / Q000502:physiology
C438187:ALS2 protein, human; D020662:Guanine Nucleotide Exchange Factors; C525911:MSTN protein, human; D055435:Myostatin; C000606290:SOD1 protein, human; D016212:Transforming Growth Factor beta; D013482:Superoxide Dismutase; D000072105:Superoxide Dismutase-1