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Scientific Articles

Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy

key information

source: Current Pediatrics Reports

year: 2014

authors: Seto JT, Bengtsson NE, Chamberlain JS


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 also bringing in new dystrophin genes. This review summarizes recent progress in the field with an emphasis on clinical applications.

organization: University of Washington, USA

DOI: 10.1007/s40124-014-0044-x

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