| Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy Y Echigoya, A Nakamura, T Nagata, N Urasawa, KRQ Lim, N Trieu, ... Proceedings of the National Academy of Sciences 114 (16), 4213-4218, 2017 | 108 | 2017 |
| Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45–55 with a cocktail of vivo-morpholinos in mdx52 mice Y Echigoya, Y Aoki, B Miskew, D Panesar, A Touznik, T Nagata, ... Molecular Therapy-Nucleic Acids 4, 2015 | 91 | 2015 |
| Multiple exon skipping in the Duchenne muscular dystrophy hot spots: prospects and challenges Y Echigoya, KRQ Lim, A Nakamura, T Yokota Journal of personalized medicine 8 (4), 41, 2018 | 86 | 2018 |
| Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs T Yokota, A Nakamura, T Nagata, T Saito, M Kobayashi, Y Aoki, ... Nucleic acid therapeutics 22 (5), 306-315, 2012 | 85 | 2012 |
| Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of … A Nakamura, N Shiba, D Miyazaki, H Nishizawa, Y Inaba, N Fueki, ... Journal of human genetics 62 (4), 459-463, 2017 | 80 | 2017 |
| Quantitative antisense screening and optimization for exon 51 skipping in Duchenne muscular dystrophy Y Echigoya, KRQ Lim, N Trieu, B Bao, BM Nichols, MC Vila, JS Novak, ... Molecular Therapy 25 (11), 2561-2572, 2017 | 79 | 2017 |
| Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides Y Echigoya, T Yokota Nucleic acid therapeutics 24 (1), 57-68, 2014 | 67 | 2014 |
| Deletion of exons 3− 9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy A Nakamura, N Fueki, N Shiba, H Motoki, D Miyazaki, H Nishizawa, ... Journal of human genetics 61 (7), 663-667, 2016 | 66 | 2016 |
| Current translational research and murine models for Duchenne muscular dystrophy M Rodrigues, Y Echigoya, S Fukada, T Yokota Journal of neuromuscular diseases 3 (1), 29-48, 2016 | 66 | 2016 |
| Dystrophin-deficient large animal models: translational research and exon skipping X Yu, B Bao, Y Echigoya, T Yokota American journal of translational research 7 (8), 1314, 2015 | 66 | 2015 |
| In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy Y Echigoya, V Mouly, L Garcia, T Yokota, W Duddy PLoS One 10 (3), e0120058, 2015 | 62 | 2015 |
| Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy KRQ Lim, R Maruyama, Y Echigoya, Q Nguyen, A Zhang, H Khawaja, ... Proceedings of the National Academy of Sciences 117 (28), 16509-16515, 2020 | 57 | 2020 |
| Impaired regenerative capacity and lower revertant fibre expansion in dystrophin-deficient mdx muscles on DBA/2 background M Rodrigues, Y Echigoya, R Maruyama, KRQ Lim, S Fukada, T Yokota Scientific reports 6 (1), 38371, 2016 | 57 | 2016 |
| LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts A Touznik, R Maruyama, K Hosoki, Y Echigoya, T Yokota Scientific reports 7 (1), 3672, 2017 | 54 | 2017 |
| Exons 45–55 skipping using mutation-tailored cocktails of antisense morpholinos in the DMD gene Y Echigoya, KRQ Lim, D Melo, B Bao, N Trieu, Y Mizobe, R Maruyama, ... Molecular Therapy 27 (11), 2005-2017, 2019 | 45 | 2019 |
| Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients? T Yokota, W Duddy, Y Echigoya, H Kolski Expert opinion on biological therapy 12 (9), 1141-1152, 2012 | 44 | 2012 |
| Mutation Types and Aging Differently Affect Revertant Fiber Expansion in Dystrophic Mdx and Mdx52 Mice Y Echigoya, J Lee, M Rodrigues, T Nagata, J Tanihata, ... PloS one 8 (7), e69194, 2013 | 41 | 2013 |
| Development of DG9 peptide-conjugated single-and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy KRQ Lim, S Woo, D Melo, Y Huang, K Dzierlega, MNA Shah, T Aslesh, ... Proceedings of the National Academy of Sciences 119 (9), e2112546119, 2022 | 31 | 2022 |
| DUX4 transcript knockdown with antisense 2′-O-methoxyethyl gapmers for the treatment of facioscapulohumeral muscular dystrophy KRQ Lim, A Bittel, R Maruyama, Y Echigoya, Q Nguyen, Y Huang, ... Molecular Therapy 29 (2), 848-858, 2021 | 30 | 2021 |
| Efficacy of multi-exon skipping treatment in Duchenne muscular dystrophy dog model neonates KRQ Lim, Y Echigoya, T Nagata, M Kuraoka, M Kobayashi, Y Aoki, ... Molecular Therapy 27 (1), 76-86, 2019 | 30 | 2019 |
Toshifumi Yokota, PhD, FCAHSProfessor, Garrett Cumming Muscular Dystrophy Canada Research Chair, University of AlbertaПодтвержден адрес электронной почты в домене ualberta.ca