title: [Advances of treatment for Duchenne muscular dystrophy with exon skipping].
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.
authors.affiliation: Department of Neurology, the First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, Guangdong 510080, P. R. China.
authors.forename: Juan
authors.initials: J
authors.lastname: Yang
authors.forename: Cheng
authors.initials: C
authors.lastname: Zhang
mesh_terms: D000818:Animals; D005091:Exons / Q000235:genetics*; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics* / Q000628:therapy*; D016376:Oligonucleotides, Antisense / Q000235:genetics
chemical_list: D016376:Oligonucleotides, Antisense