Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment

EMBO Rep. 2004 Sep;5(9):872-6. doi: 10.1038/sj.embor.7400221.

Abstract

Third in Molecular Medicine Review Series

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Dystrophin / physiology*
  • Genetic Therapy
  • Humans
  • Muscular Dystrophy, Duchenne / physiopathology*
  • Muscular Dystrophy, Duchenne / therapy*
  • Myoblasts / transplantation
  • Oligonucleotides, Antisense / therapeutic use
  • Proteasome Inhibitors
  • Stem Cell Transplantation
  • Up-Regulation / physiology

Substances

  • Dystrophin
  • Oligonucleotides, Antisense
  • Proteasome Inhibitors