title: Overview on DMD exon skipping.
abstract: Antisense-mediated exon skipping to restore the disrupted dystrophin reading frame is currently in clinical trials for Duchenne muscular dystrophy. This chapter describes the rationale of this approach and gives an overview of in vitro and in vivo experiments with antisense oligonucleotides and antisense genes. Finally, an overview of clinical trials is given and outstanding questions and hurdles are discussed.
authors.affiliation: Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands. a.m.rus@lumc.nl
authors.forename: Annemieke
authors.initials: A
authors.lastname: Aartsma-Rus
mesh_terms: D000818:Animals; D016189:Dystrophin / Q000235:genetics*; D005091:Exons; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics* / Q000628:therapy; D009154:Mutation; D016376:Oligonucleotides, Antisense / Q000235:genetics / Q000627:therapeutic use*; D016364:Reading Frames
chemical_list: D016189:Dystrophin; D016376:Oligonucleotides, Antisense