Ontology highlight
ABSTRACT:
SUBMITTER: Bish LT
PROVIDER: S-EPMC3293605 | biostudies-literature | 2012 Mar
REPOSITORIES: biostudies-literature
Bish Lawrence T LT Sleeper Meg M MM Forbes Sean C SC Wang Bingjing B Reynolds Caryn C Singletary Gretchen E GE Trafny Dennis D Morine Kevin J KJ Sanmiguel Julio J Cecchini Sylvain S Virag Tamas T Vulin Adeline A Beley Cyriaque C Bogan Janet J Wilson James M JM Vandenborne Krista K Kornegay Joe N JN Walter Glenn A GA Kotin Robert M RM Garcia Luis L Sweeney H Lee HL
Molecular therapy : the journal of the American Society of Gene Therapy 20111206 3
Although restoration of dystrophin expression via exon skipping in both cardiac and skeletal muscle has been successfully demonstrated in the mdx mouse, restoration of cardiac dystrophin expression in large animal models of Duchenne muscular dystrophy (DMD) has proven to be a challenge. In large animals, investigators have focused on using intravenous injection of antisense oligonucleotides (AO) to mediate exon skipping. In this study, we sought to optimize restoration of cardiac dystrophin expr ...[more]