Ontology highlight
ABSTRACT:
SUBMITTER: Lu-Nguyen NB
PROVIDER: S-EPMC4579549 | biostudies-literature | 2015 Aug
REPOSITORIES: biostudies-literature
Lu-Nguyen Ngoc B NB Jarmin Susan A SA Saleh Amer F AF Popplewell Linda L Gait Michael J MJ Dickson George G
Molecular therapy : the journal of the American Society of Gene Therapy 20150511 8
The fatal X-linked Duchenne muscular dystrophy (DMD), characterized by progressive muscle wasting and muscle weakness, is caused by mutations within the DMD gene. The use of antisense oligonucleotides (AOs) modulating pre-mRNA splicing to restore the disrupted dystrophin reading frame, subsequently generating a shortened but functional protein has emerged as a potential strategy in DMD treatment. AO therapy has recently been applied to induce out-of-frame exon skipping of myostatin pre-mRNA, kno ...[more]