Eteplirsen in the treatment of Duchenne muscular dystrophy KRQ Lim, R Maruyama, T Yokota Drug design, development and therapy, 533-545, 2017 | 496 | 2017 |
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 | 106 | 2017 |
EGG-3 regulates cell-surface and cortex rearrangements during egg activation in Caenorhabditis elegans R Maruyama, NV Velarde, R Klancer, S Gordon, P Kadandale, JM Parry, ... Current Biology 17 (18), 1555-1560, 2007 | 92 | 2007 |
EGG-4 and EGG-5 link events of the oocyte-to-embryo transition with meiotic progression in C. elegans JM Parry, NV Velarde, AJ Lefkovith, MH Zegarek, JS Hang, J Ohm, ... Current Biology 19 (20), 1752-1757, 2009 | 85 | 2009 |
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 | 78 | 2017 |
Drosophila as a model for epithelial tube formation R Maruyama, DJ Andrew Developmental Dynamics, 2011 | 76 | 2011 |
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 | 70 | 2017 |
Nusinersen in the treatment of spinal muscular atrophy K Goodkey, T Aslesh, R Maruyama, T Yokota Exon skipping and inclusion therapies: Methods and protocols, 69-76, 2018 | 53 | 2018 |
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 | 53 | 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 | 52 | 2017 |
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 | 51 | 2020 |
Skipping multiple exons to treat DMD—promises and challenges T Aslesh, R Maruyama, T Yokota Biomedicines 6 (1), 1, 2018 | 45 | 2018 |
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 | 44 | 2019 |
Organ-specific gene expression: the bHLH protein Sage provides tissue specificity to Drosophila FoxA RM Fox, A Vaishnavi, R Maruyama, DJ Andrew Development 140 (10), 2160-2171, 2013 | 41 | 2013 |
Designing effective antisense oligonucleotides for exon skipping T Shimo, R Maruyama, T Yokota Duchenne Muscular Dystrophy: Methods and Protocols, 143-155, 2018 | 40 | 2018 |
Identification of novel antisense-mediated exon skipping targets in DYSF for therapeutic treatment of dysferlinopathy JJA Lee, R Maruyama, W Duddy, H Sakurai, T Yokota Molecular Therapy-Nucleic Acids 13, 596-604, 2018 | 36 | 2018 |
Knocking down long noncoding RNAs using antisense oligonucleotide gapmers R Maruyama, T Yokota Gapmers: methods and protocols, 49-56, 2020 | 35 | 2020 |
Caenorhabditis elegans DAZ-1 is expressed in proliferating germ cells and directs proper nuclear organization and cytoplasmic core formation during oogenesis R Maruyama, S Endo, A Sugimoto, M Yamamoto Developmental Biology 277 (1), 142-154, 2005 | 33 | 2005 |
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 | 28 | 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 | 27 | 2021 |