[Advances of treatment for Duchenne muscular dystrophy with exon skipping]

Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2011 Aug;28(4):406-8. doi: 10.3760/cma.j.issn.1003-9406.2011.04.010.
[Article in Chinese]

Abstract

Duchenne muscular dystrophy (DMD) is a lethal muscular disorder caused by mutations in the dystrophin gene for which no mutation targeted therapy has been available so far. However, a new method named exon-skipping mediated by antisense oligonucleotides has considerable potential for DMD therapy. In this review, the principle, basic research and clinical research of exon-skipping are mainly summarized.

Publication types

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

MeSH terms

  • Animals
  • Exons / genetics*
  • Humans
  • Muscular Dystrophy, Duchenne / genetics*
  • Muscular Dystrophy, Duchenne / therapy*
  • Oligonucleotides, Antisense / genetics

Substances

  • Oligonucleotides, Antisense