title: Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold?
authors.affiliation: Department of Medical Genetics, Faculty of Medicine & Dentistry, University of Alberta, 116 St. & 85 Ave., Edmonton, AB, T6G 2R3, Canada.
authors.forename: Ashley
authors.initials: A
authors.lastname: Guncay
authors.affiliation: Department of Medical Genetics, Faculty of Medicine & Dentistry, University of Alberta, 116 St. & 85 Ave., Edmonton, AB, T6G 2R3, Canada.|Muscular Dystrophy Canada Research Chair, Edmonton, Alberta, Canada.
authors.forename: Toshifumi
authors.initials: T
authors.lastname: Yokota
mesh_terms: D002986:Clinical Trials as Topic; D055808:Drug Discovery; D005091:Exons / Q000187:drug effects; D006801:Humans; D060172:Morpholinos / Q000494:pharmacology / Q000627:therapeutic use; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000235:genetics; D016376:Oligonucleotides, Antisense / Q000494:pharmacology / Q000627:therapeutic use*; D012322:RNA Precursors / Q000235:genetics
chemical_list: D060172:Morpholinos; D016376:Oligonucleotides, Antisense; D012322:RNA Precursors
keywords: 2′-O methyl phosphorothioate antisense oligonucleotides; Duchenne muscular dystrophy; antisense therapy; drisapersen; eteplirsen; exon skipping; phosphorodiamidate morpholino oligomers; tricyclo-DNA oligomers