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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2015-03-01T00:00:00Z.
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