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Curr Pediatr Rep. 2014 Jun 1;2(2):102-112.

Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.

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Department of Neurology, 1959 NE Pacific St, University of Washington, Seattle, Washington 98195-7720, USA, Tel: 206-616-6645.


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.


Duchenne muscular dystrophy; adeno-associated viral vectors; antisense oligonucleotide; cxmd dog; dystrophin; gene therapy; inducible pluripotent stem cell; mdx mouse; mesoangioblast; morpholino; myoblast; pericyte; premature termination codon; satellite cell; stem cells; utrophin

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