Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.
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ABSTRACT:
SUBMITTER: Seto JT
PROVIDER: S-EPMC4036820 | biostudies-literature | 2014 Jun
REPOSITORIES: biostudies-literature
Publications
Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.
Seto Jane T JT Bengtsson Niclas E NE Chamberlain Jeffrey S JS
Current pediatrics reports 20140601 2
Duchenne muscular dystrophy (DMD) is an inherited, progressive muscle wasting disorder caused by mutations in the dystrophin gene. An increasing variety of approaches are moving towards clinical testing that all aim to restore dystrophin production and to enhance or preserve muscle mass. Gene therapy methods are being developed to replace the defective dystrophin gene or induce dystrophin production from mutant genes. Stem cell approaches are being developed to replace lost muscle cells while al ...[more]
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Project description:Duchenne muscular dystrophy is a genetically determined X-linked disease and the most common, progressive pediatric muscle disorder. For decades, research has been conducted to find an effective therapy. This review presents current therapeutic methods for Duchenne muscular dystrophy, based on scientific articles in English published mainly in the period 2000 to 2014. We used the PubMed database to identify and review the most important studies. An analysis of contemporary studies of stem cell therapy and the use of granulocyte colony-stimulating factor (G-CSF) in muscular dystrophy was performed.
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Project description:Genetic Modifiers of Duchenne Muscular Dystrophy
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