Skip to main content
Article thumbnail
Location of Repository

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

By J. Alter, F. Lou, A. Rabinowitz, H.F. Yin, J. Rosenfeld, S.D. Wilton, T.A. Partridge and Q.L. Lu

Abstract

For the majority of Duchenne muscular dystrophy (DMD) mutations, antisense oligonucleotide (AON)-mediated exon skipping has the potential to restore a functional protein. Here we show that weekly intravenous injections of morpholino phosphorodiamidate (morpholino) AONs induce expression of functional levels of dystrophin in body-wide skeletal muscles of the dystrophic mdx mouse, with resulting improvement in muscle function. Although the level of dystrophin expression achieved varies considerably between muscles, antisense therapy may provide a realistic hope for the treatment of a majority of individuals with DMD

Publisher: Nature Publishing Group
Year: 2006
OAI identifier: oai:researchrepository.murdoch.edu.au:21578
Provided by: Research Repository
Download PDF:
Sorry, we are unable to provide the full text but you may find it at the following location(s):
  • http://researchrepository.murd... (external link)
  • http://researchrepository.murd... (external link)
  • http://researchrepository.murd... (external link)
  • http://researchrepository.murd... (external link)
  • Suggested articles


    To submit an update or takedown request for this paper, please submit an Update/Correction/Removal Request.