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BioMed Research International
Volume 2013 (2013), Article ID 402369, 8 pages
Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy
1Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK
2Department of Medical Genetics, School of Human Development, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada T6G 2H7
3The Friends of Garrett Cumming Research and Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, University of Alberta, Edmonton, AB, Canada T6G 2H7
Received 30 April 2013; Accepted 18 June 2013
Academic Editor: Akinori Nakamura
Copyright © 2013 Yoshitsugu Aoki et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Citations to this Article [11 citations]
The following is the list of published articles that have cited the current article.
- Yusuke Echigoya, and Toshifumi Yokota, “Skipping Multiple Exons of Dystrophin Transcripts Using Cocktail Antisense Oligonucleotides,” Nucleic Acid Therapeutics, pp. 131231075231009, 2013.
- Hongmei Lisa Li, Takao Nakano, and Akitsu Hotta, “Genetic correction using engineered nucleases for gene therapy applications,” Development, Growth & Differentiation, 2013.
- Stephanie A. Fernandes, Andrew G. L. Douglas, Miguel A. Varela, Matthew J. A. Wood, and Yoshitsugu Aoki, “Oligonucleotide-Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansion: A Perspective,” Journal of Nucleic Acids, vol. 2013, pp. 1–11, 2013.
- Giulietta Riboldi, Chiara Zanetta, Michela Ranieri, Monica Nizzardo, Chiara Simone, Francesca Magri, Nereo Bresolin, Giacomo P. Comi, and Stefania Corti, “Antisense Oligonucleotide Therapy for the Treatment of C9ORF72 ALS/FTD Diseases,” Molecular Neurobiology, 2014.
- Annemieke Aartsma-Rus, Alessandra Ferlini, Nathalie Goemans, Anna M.G. Pasmooij, Dominic J. Wells, Katerine Bushby, Elizabeth Vroom, and Pavel Balabanov, “Translational and Regulatory Challenges for Exon Skipping Therapies,” Human Gene Therapy, vol. 25, no. 10, pp. 885–892, 2014.
- Marcel Veltrop, and Annemieke Aartsma-Rus, “Antisense-mediated exon skipping: Taking advantage of a trick from Mother Nature to treat rare genetic diseases,” Experimental Cell Research, 2014.
- Akitsu Hotta, “Genome Editing Gene Therapy for Duchenne Muscular Dystrophy,” Journal of Neuromuscular Diseases, vol. 2, no. 4, pp. 343–355, 2015.
- Yusuke Echigoya, Yoshitsugu Aoki, Bailey Miskew, Dharminder Panesar, Aleksander Touznik, Tetsuya Nagata, Jun Tanihata, Akinori Nakamura, Kanneboyina Nagaraju, and Toshifumi Yokota, “Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice,” Molecular Therapy—Nucleic Acids, vol. 4, no. 2, pp. e225, 2015.
- Graham McClorey, Suzan M. Hammond, Mattias Hällbrink, Ülo Langel, C.I. Edvard Smith, Matthew J.A. Wood, Michael J. Gait, Samir EL Andaloussi, Peter Järver, Eman M. Zaghloul, Andrey A. Arzumanov, and Amer F. Saleh, “Peptide Nanoparticle Delivery of Charge-Neutral Splice-Switching Morpholino Oligonucleotides,” Nucleic Acid Therapeutics, pp. 150116113510008, 2015.
- Luca Bello, Paola Campadello, Andrea Barp, Marina Fanin, Claudio Semplicini, Gianni Sorarù, Luca Caumo, Chiara Calore, Corrado Angelini, and Elena Pegoraro, “Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies,” Scientific Reports, vol. 6, pp. 32439, 2016.
- Kay E. Davies, “2015 William Allan Award1,” The American Journal of Human Genetics, vol. 98, no. 3, pp. 419–426, 2016.