Advances of treatment for Duchenne muscular dystrophy with exon skipping.
10.3760/cma.j.issn.1003-9406.2011.04.010
- VernacularTitle:外显子跳跃治疗Duchenne型肌营养不良症的研究进展
- Author:
Juan YANG
1
;
Cheng ZHANG
Author Information
1. Department of Neurology, the First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, Guangdong 510080, P. R. China.
- Publication Type:Journal Article
- MeSH:
Animals;
Exons;
genetics;
Humans;
Muscular Dystrophy, Duchenne;
genetics;
therapy;
Oligonucleotides, Antisense;
genetics
- From:
Chinese Journal of Medical Genetics
2011;28(4):406-408
- CountryChina
- Language: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.
