Article
Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice.
FASEB journal : official publication of the Federation of American Societies for Experimental Biology - 1 Jun 2013
van Putten Maaike, Hulsker Margriet, Young Courtney, Nadarajah Vishna D, Heemskerk Hans, van der Weerd Louise, 't Hoen Peter A C, van Ommen Gert-Jan B, Aartsma-Rus Annemieke M
Abstract excerpt
Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by the lack of functional dystrophin. There is no cure, but several clinical trials aimed to restore the synthesis of functional dystrophin are underway. The dystrophin levels needed for improvement of muscle pathology, function, and overall vitality are not known. Here, we describe the mdx/utrn(-/-)/Xist(Δhs) mouse model, which...
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