Ontology highlight
ABSTRACT:
SUBMITTER: Park KS
PROVIDER: S-EPMC2950915 | biostudies-literature | 2010 Sep
REPOSITORIES: biostudies-literature
Park Kyung Seok KS Oh Donghoon D
Journal of clinical neurology (Seoul, Korea) 20100901 3
Muscular dystrophies are groups of inherited progressive diseases of the muscle caused by mutations of diverse genes related to normal muscle function. Although there is no current effective treatment for these devastating diseases, various molecular strategies have been developed to restore the expressions of the associated defective proteins. In preclinical animal models, both viral and nonviral vectors have been shown to deliver recombinant versions of defective genes. Antisense oligonucleoti ...[more]