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New developments in exon skipping and splice modulation therapies for neuromuscular diseases

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S Fletcher, MI Bellgard, L Price, AP Akkari & SD Wilton. (2017) Translational development of splice-modifying antisense oligomers. Expert Opinion on Biological Therapy 17:1, pages 15-30.
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Bo Bao & Toshifumi Yokota. (2015) Potential of antisense therapy for facioscapulohumeral muscular dystrophy. Expert Opinion on Orphan Drugs 3:12, pages 1365-1374.
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Emily M. Mills, Victoria L. Barlow, Louis Y. P. Luk & Yu-Hsuan Tsai. (2019) Applying switchable Cas9 variants to in vivo gene editing for therapeutic applications. Cell Biology and Toxicology 36:1, pages 17-29.
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Shouta Miyatake, Yoshitaka Mizobe, Maria K. Tsoumpra, Kenji Rowel Q. Lim, Yuko Hara, Fazel Shabanpoor, Toshifumi Yokota, Shin’ichi Takeda & Yoshitsugu Aoki. (2019) Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle. Molecular Therapy - Nucleic Acids 14, pages 520-535.
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Takenori Shimo, Keisuke Tachibana & Satoshi Obika. (2018) Construction of a tri-chromatic reporter cell line for the rapid and simple screening of splice-switching oligonucleotides targeting DMD exon 51 using high content screening. PLOS ONE 13:5, pages e0197373.
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Chuan Xu, Jiajun Chen, Yingyu Zhang & Jia Li. (2018) Limb-girdle muscular dystrophy type 2B misdiagnosed as polymyositis at the early stage. Medicine 97:21, pages e10539.
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David J. Coote, Mark R. Davis, Macarena Cabrera, Merrilee Needham, Nigel G. Laing & Kristen J. Nowak. (2018) CUGC for Duchenne muscular dystrophy (DMD). European Journal of Human Genetics 26:5, pages 749-757.
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Sean Porazinski & Michael Ladomery. (2018) Alternative Splicing in the Hippo Pathway—Implications for Disease and Potential Therapeutic Targets. Genes 9:3, pages 161.
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Tejal Aslesh, Rika Maruyama & Toshifumi Yokota. (2018) Skipping Multiple Exons to Treat DMD—Promises and Challenges. Biomedicines 6:1, pages 1.
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Tanya Bardakjian & Pedro Gonzalez-Alegre. 2018. Neurogenetics, Part I. Neurogenetics, Part I 93 102 .
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Rika Maruyama & Toshifumi Yokota. 2018. Exon Skipping and Inclusion Therapies. Exon Skipping and Inclusion Therapies 79 90 .
Kara Goodkey, Tejal Aslesh, Rika Maruyama & Toshifumi Yokota. 2018. Exon Skipping and Inclusion Therapies. Exon Skipping and Inclusion Therapies 69 76 .
Hae-Won Son & Toshifumi Yokota. 2018. Exon Skipping and Inclusion Therapies. Exon Skipping and Inclusion Therapies 57 68 .
Tejal Aslesh, Rika Maruyama & Toshifumi Yokota. 2018. Exon Skipping and Inclusion Therapies. Exon Skipping and Inclusion Therapies 455 465 .
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Yoshitaka Mizobe, Shouta Miyatake, Hotake Takizawa, Yuko Hara, Toshifumi Yokota, Akinori Nakamura, Shin’Ichi Takeda & Yoshitsugu Aoki. 2018. Exon Skipping and Inclusion Therapies. Exon Skipping and Inclusion Therapies 275 292 .
Shouta Miyatake, Yoshitaka Mizobe, Hotake Takizawa, Yuko Hara, Toshifumi Yokota, Shin’ichi Takeda & Yoshitsugu Aoki. 2018. Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy 123 141 .
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Rachel Z. BlumhagenBrenna R. HedinKenneth C. MalcolmEllen L. BurnhamMarc MossEdward AbrahamTristan J. HuieJerry A. NickTasha E. FingerlinScott Alper. (2017) Alternative pre-mRNA splicing of Toll-like receptor signaling components in peripheral blood mononuclear cells from patients with ARDS. American Journal of Physiology-Lung Cellular and Molecular Physiology 313:5, pages L930-L939.
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Quynh Nguyen & Toshifumi Yokota. (2017) Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy. Journal of Personalized Medicine 7:4, pages 13.
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James S. Novak, Marshall W. Hogarth, Jessica F. Boehler, Marie Nearing, Maria C. Vila, Raul Heredia, Alyson A. Fiorillo, Aiping Zhang, Yetrib Hathout, Eric P. Hoffman, Jyoti K. Jaiswal, Kanneboyina Nagaraju, Sebahattin Cirak & Terence A. Partridge. (2017) Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle. Nature Communications 8:1.
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Lucy Norcliffe-Kaufmann, Susan A. Slaugenhaupt & Horacio Kaufmann. (2017) Familial dysautonomia: History, genotype, phenotype and translational research. Progress in Neurobiology 152, pages 131-148.
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Rika Maruyama, Yusuke Echigoya, Oana Caluseriu, Yoshitsugu Aoki, Shin’ichi Takeda & Toshifumi Yokota. 2017. Morpholino Oligomers. Morpholino Oligomers 201 213 .
Glenn CruseYuzhi YinTomoki FukuyamaAvanti DesaiGreer K. ArthurWolfgang BäumerMichael A. Beaven & Dean D. Metcalfe. (2016) Exon skipping of FcεRIβ eliminates expression of the high-affinity IgE receptor in mast cells with therapeutic potential for allergy. Proceedings of the National Academy of Sciences 113:49, pages 14115-14120.
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Yuri Kitamura, Eri Kondo, Mari Urano, Ryoko Aoki & Kayoko Saito. (2016) Target resequencing of neuromuscular disease-related genes using next-generation sequencing for patients with undiagnosed early-onset neuromuscular disorders. Journal of Human Genetics 61:11, pages 931-942.
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Gregory P. Holmes-Hampton, Daniel R. Crooks, Ronald G. Haller, Shuling Guo, Susan M. Freier, Brett P. Monia & Tracey A. Rouault. (2016) Use of antisense oligonucleotides to correct the splicing error in ISCU myopathy patient cell lines. Human Molecular Genetics, pages ddw338.
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Jacqueline N. Robinson-Hamm & Charles A. Gersbach. (2016) Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy. Human Genetics 135:9, pages 1029-1040.
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Pierre G. Carlier, Benjamin Marty, Olivier Scheidegger, Paulo Loureiro de Sousa, Pierre-Yves Baudin, Eduard Snezhko & Dmitry Vlodavets. (2016) Imagerie et spectroscopie par résonance magnétique nucléaire du muscle strié squelettique. Les Cahiers de Myologie:13, pages 34-67.
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Christopher S. Rogers. (2016) Genetically engineered livestock for biomedical models. Transgenic Research 25:3, pages 345-359.
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Pierre G. Carlier, Benjamin Marty, Olivier Scheidegger, Paulo Loureiro de Sousa, Pierre-Yves Baudin, Eduard Snezhko & Dmitry Vlodavets. (2016) Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials. Journal of Neuromuscular Diseases 3:1, pages 1-28.
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Merryl Rodrigues, Yusuke Echigoya, So-ichiro Fukada & Toshifumi Yokota. (2016) Current Translational Research and Murine Models For Duchenne Muscular Dystrophy. Journal of Neuromuscular Diseases 3:1, pages 29-48.
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Mary H. Wertz & Mustafa Sahin. (2016) Developing therapies for spinal muscular atrophy. Annals of the New York Academy of Sciences 1366:1, pages 5-19.
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Susana Igreja, Luka A. Clarke, Hugo M. Botelho, Luís Marques & Margarida D. Amaral. (2016) Correction of a Cystic Fibrosis Splicing Mutation by Antisense Oligonucleotides. Human Mutation 37:2, pages 209-215.
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Joshua J. A. Lee & Toshifumi Yokota. 2016. Translational Research in Muscular Dystrophy. Translational Research in Muscular Dystrophy 87 102 .
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Ashley Guncay & Toshifumi Yokota. (2015) Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold?. Future Medicinal Chemistry 7:13, pages 1631-1635.
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Bailey Nichols, Shin'ichi Takeda & Toshifumi Yokota. (2015) Nonmechanical Roles of Dystrophin and Associated Proteins in Exercise, Neuromuscular Junctions, and Brains. Brain Sciences 5:3, pages 275-298.
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Dorota Sienkiewicz, Wojciech Kulak, Bożena Okurowska-Zawada, Grażyna Paszko-Patej & Katarzyna Kawnik. (2015) Duchenne muscular dystrophy: current cell therapies. Therapeutic Advances in Neurological Disorders 8:4, pages 166-177.
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Mirjam Ketema, Pablo Secades, Maaike Kreft, Leila Nahidiazar, Hans Janssen, Kees Jalink, Jose M. de Pereda & Arnoud Sonnenberg. (2015) The rod domain is not essential for the function of plectin in maintaining tissue integrity. Molecular Biology of the Cell 26:13, pages 2402-2417.
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Nicolas Wein, Lindsay Alfano & Kevin M. Flanigan. (2015) Genetics and Emerging Treatments for Duchenne and Becker Muscular Dystrophy. Pediatric Clinics of North America 62:3, pages 723-742.
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Yusuke Echigoya, Vincent Mouly, Luis Garcia, Toshifumi Yokota & William Duddy. (2015) In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy. PLOS ONE 10:3, pages e0120058.
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Casey A. Maguire, Servio H. Ramirez, Steven F. Merkel, Miguel Sena-Esteves & Xandra O. Breakefield. (2014) Gene Therapy for the Nervous System: Challenges and New Strategies. Neurotherapeutics 11:4, pages 817-839.
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