Ontology highlight
ABSTRACT:
SUBMITTER: Liu J
PROVIDER: S-EPMC4120526 | biostudies-literature | 2012 Aug
REPOSITORIES: biostudies-literature
Current gene therapy 20120801 4
Limb Girdle Muscular Dystrophy (LGMD) refers to a group of 25 genetic diseases linked by common clinical features, including wasting of muscles supporting the pelvic and shoulder girdles. Cardiac involvement may also occur. Like other muscular dystrophies, LGMDs are currently incurable, but prospective gene replacement therapies targeting recessive forms have shown promise in pre-clinical and clinical studies. In contrast, little attention has been paid to developing gene therapy approaches for ...[more]