Ontology highlight
ABSTRACT:
SUBMITTER: Qiao C
PROVIDER: S-EPMC1189311 | biostudies-literature | 2005 Aug
REPOSITORIES: biostudies-literature
Proceedings of the National Academy of Sciences of the United States of America 20050815 34
Congenital muscular dystrophy (CMD) is characterized by severe muscle wasting, premature death in early childhood, and lack of effective treatment. Most of the CMD cases are caused by genetic mutations of laminin-alpha2, which is essential for the structural integrity of muscle extracellular matrix. Here, we report that somatic gene delivery of a structurally unrelated protein, a miniature version of agrin, functionally compensates for laminin-alpha2 deficiency in the murine models of CMD. Adeno ...[more]