Antisense Oligonucleotide Drugs for Duchenne Muscular Dystrophy: How Far Have We Come and What Does the Future Hold?

Future Medicinal ChemistryVol. 7, No. 13 CommentaryAntisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold?Ashley Guncay & Toshifumi YokotaAshley Guncay Department of Medical Genetics, Faculty of Medicine & Dentistry, University of Alberta, 116 St. & 85 Ave., Edmonton, AB, T6G 2R3, Canada & Toshifumi Yokota*Author for correspondence: E-mail Address: toshifum@ualberta.ca 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, CanadaPublished Online:1 Oct 2015https://doi.org/10.4155/fmc.15.116AboutSectionsView ArticleView Full TextPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareShare onFacebookTwitterLinkedInRedditEmail View articleKeywords: 2′-O methyl phosphorothioate antisense oligonucleotidesantisense therapydrisapersenDuchenne muscular dystrophyeteplirsenexon skippingphosphorodiamidate morpholino oligomerstricyclo-DNA oligomersReferences1 Duchenne. 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Med. 21(3), 270–275 (2015).Crossref, Medline, CAS, Google ScholarFiguresReferencesRelatedDetailsCited ByRestoring Dystrophin Expression by Skipping Exons 6 and 8 in Neonatal Dystrophic Dogs19 November 2022A novel mouse model of PMS2 founder mutation that causes mismatch repair defect due to aberrant splicing6 September 2021 | Cell Death & Disease, Vol. 12, No. 9Polypurine Reverse-Hoogsteen Hairpins as a Tool for Exon Skipping at the Genomic Level in Mammalian Cells6 April 2021 | International Journal of Molecular Sciences, Vol. 22, No. 7Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy1 June 2020 | Gene Therapy, Vol. 27, No. 9Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal MuscleMolecular Therapy - Nucleic Acids, Vol. 14Skipping Multiple Exons to Treat DMD—Promises and Challenges2 January 2018 | Biomedicines, Vol. 6, No. 1Designing Effective Antisense Oligonucleotides for Exon Skipping25 October 2017Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy25 October 2017Invention and Early History of Exon Skipping and Splice Modulation1 September 2018Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping1 September 2018In Vivo Evaluation of Single-Exon and Multiexon Skipping in mdx52 Mice1 September 2018An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases1 September 2018In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs1 September 2018Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion1 September 2018Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy16 October 2017 | Journal of Personalized Medicine, Vol. 7, No. 4Translational development of splice-modifying antisense oligomers2 November 2016 | Expert Opinion on Biological Therapy, Vol. 17, No. 1Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy1 April 2017Current Translational Research and Murine Models For Duchenne Muscular DystrophyJournal of Neuromuscular Diseases, Vol. 3, No. 1Frontiers in nucleic acid-based drug research and developmentLun Quan Sun & Jonathan P Wong14 October 2015 | Future Medicinal Chemistry, Vol. 7, No. 13 Vol. 7, No. 13 STAY CONNECTED Metrics Downloaded 144 times History Published online 1 October 2015 Published in print September 2015 Information© Future Science LtdKeywords2′-O methyl phosphorothioate antisense oligonucleotidesantisense therapydrisapersenDuchenne muscular dystrophyeteplirsenexon skippingphosphorodiamidate morpholino oligomerstricyclo-DNA oligomersFinancial & competing interests disclosureThis work was supported by grants from the Canadian Institutes of Health Research, University of Alberta Faculty of Medicine and Dentistry, The Friends of Garrett Cumming Research Fund, HM Toupin Neurological Science Research Fund, Muscular Dystrophy Canada, Canada Foundation for Innovation (CFI), Alberta Advanced Education and Technology, and the Women and Children's Health Research Institute (WCHRI). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript.No writing assistance was used in the production of this manuscript.PDF download