Muscular dystrophy: from pathogenesis to strategy.
Muscular dystrophies are a genetically heterogeneous group of degenerative muscle disorders. It characterized by progressive muscle wasting and weakness of variable distribution and severity. There are several subgroups including Duchenne/Becker, fascioscapulohumeral, limb-girdle, oculopharngeal, and congenital muscular dystrophy. Diagnosis is dependent to the characteristic clinical features in distribution of predominant muscle weakness, disease course and age onset as well as variable serum concentration creatine kinase, muscle histology, and genetic inheritance. Nearly 30 genes and encoded proteins are known to give rise to various forms of muscular dystrophy. Development of new prospects therapy for the muscular dystrophies is a big challenge. The target of strategies is aimed at inducing of a functional protein and improving the function of muscle weakness. These strategies include gene, cell and pharmacological therapies. However, efficiency of systemic delivery vectors to targets, immune reaction to vector and gene products, and toxicity to vector that must be solved before an effective treatment is available.
Department of Neurology, Tri-Service General Hospital, National Defense Medical Center, National Defense University, Taipei, Taiwan. hsu1487@mail.tsghndmc.edu.tw
Yaw-Don
YD
Hsu
D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D009136:Muscular Dystrophies / Q000175:diagnosis* / Q000235:genetics / Q000628:therapy*