Identification of muscle-specific microRNAs in serum of muscular dystrophy animal models: promising novel blood-based markers for muscular dystrophy H Mizuno, A Nakamura, Y Aoki, N Ito, S Kishi, K Yamamoto, M Sekiguchi, ... PloS one 6 (3), e18388, 2011 | 216 | 2011 |
Bodywide skipping of exons 45–55 in dystrophic mdx52 mice by systemic antisense delivery Y Aoki, T Yokota, T Nagata, A Nakamura, J Tanihata, T Saito, ... Proceedings of the National Academy of Sciences 109 (34), 13763-13768, 2012 | 185 | 2012 |
C9orf72 and RAB7L1 regulate vesicle trafficking in amyotrophic lateral sclerosis and frontotemporal dementia Y Aoki, R Manzano, Y Lee, R Dafinca, M Aoki, AGL Douglas, MA Varela, ... Brain 140 (4), 887-897, 2017 | 173 | 2017 |
In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52–deficient mdx mouse Y Aoki, A Nakamura, T Yokota, T Saito, H Okazawa, T Nagata, S Takeda Molecular Therapy 18 (11), 1995-2005, 2010 | 147 | 2010 |
Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy H Komaki, T Nagata, T Saito, S Masuda, E Takeshita, M Sasaki, ... Science Translational Medicine 10 (437), eaan0713, 2018 | 130 | 2018 |
Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials Y Shimizu-Motohashi, S Miyatake, H Komaki, S Takeda, Y Aoki American journal of translational research 8 (6), 2471, 2016 | 109 | 2016 |
Identification of disease specific pathways using in vivo SILAC proteomics in dystrophin deficient mdx mouse S Rayavarapu, W Coley, E Cakir, V Jahnke, S Takeda, Y Aoki, ... Molecular & cellular proteomics 12 (5), 1061-1073, 2013 | 109 | 2013 |
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 | 105 | 2017 |
Self-assembly into nanoparticles is essential for receptor mediated uptake of therapeutic antisense oligonucleotides K Ezzat, Y Aoki, T Koo, G McClorey, L Benner, A Coenen-Stass, ... Nano letters 15 (7), 4364-4373, 2015 | 103 | 2015 |
Restoring dystrophin expression in Duchenne muscular dystrophy: current status of therapeutic approaches Y Shimizu-Motohashi, H Komaki, N Motohashi, S Takeda, T Yokota, ... Journal of personalized medicine 9 (1), 1, 2019 | 102 | 2019 |
Three novel serum biomarkers, miR-1, miR-133a, and miR-206 for Limb-girdle muscular dystrophy, Facioscapulohumeral muscular dystrophy, and Becker muscular dystrophy Y Matsuzaka, S Kishi, Y Aoki, H Komaki, Y Oya, S Takeda, K Hashido Environmental health and preventive medicine 19, 452-458, 2014 | 101 | 2014 |
Extracellular microRNAs are dynamic non-vesicular biomarkers of muscle turnover TC Roberts, C Godfrey, G McClorey, P Vader, D Briggs, C Gardiner, ... Nucleic acids research 41 (20), 9500-9513, 2013 | 101 | 2013 |
Anti-inflammatory drugs for Duchenne muscular dystrophy: focus on skeletal muscle-releasing factors S Miyatake, Y Shimizu-Motohashi, S Takeda, Y Aoki Drug design, development and therapy, 2745-2758, 2016 | 84 | 2016 |
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 | 84 | 2012 |
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 | 83 | 2015 |
Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases MK Tsoumpra, S Fukumoto, T Matsumoto, S Takeda, MJA Wood, Y Aoki EBioMedicine 45, 630-645, 2019 | 78 | 2019 |
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 | 77 | 2017 |
Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice Y Aoki, T Nagata, T Yokota, A Nakamura, MJA Wood, T Partridge, ... Human molecular genetics 22 (24), 4914-4928, 2013 | 73 | 2013 |
Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy Y Aoki, T Yokota, MJA Wood BioMed research international 2013, 2013 | 62 | 2013 |
Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient T Saito, A Nakamura, Y Aoki, T Yokota, T Okada, M Osawa, S Takeda PLoS One 5 (8), e12239, 2010 | 62 | 2010 |