Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly promising therapeutic strategy is to replace or repair the defective dystrophin gene by gene therapy. Numerous animal m...

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Egile Nagusiak: Joe W. McGreevy (Egilea), Chady H. Hakim (Egilea), Mark A. McIntosh (Egilea), Dongsheng Duan (Egilea)
Formatua: Liburua
Argitaratua: The Company of Biologists, 2015-03-01T00:00:00Z.
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