Ontology highlight
ABSTRACT:
SUBMITTER: van Putten M
PROVIDER: S-EPMC3659351 | biostudies-literature | 2013 Jun
REPOSITORIES: biostudies-literature
FASEB journal : official publication of the Federation of American Societies for Experimental Biology 20130304 6
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 expresses a range of low dystrophin levels, depending on the degree of skewing of X inactiva ...[more]