| Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2 … S Cirak, V Arechavala-Gomeza, M Guglieri, L Feng, S Torelli, K Anthony, ... The Lancet 378 (9791), 595-605, 2011 | 1161 | 2011 |
| Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of … M Kinali, V Arechavala-Gomeza, L Feng, S Cirak, D Hunt, C Adkin, ... The Lancet Neurology 8 (10), 918-928, 2009 | 889 | 2009 |
| Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle V Arechavala-Gomeza, IR Graham, LJ Popplewell, AM Adams, ... Human gene therapy 18 (9), 798-810, 2007 | 252 | 2007 |
| Delivery of oligonucleotide‐based therapeutics: challenges and opportunities SM Hammond, A Aartsma‐Rus, S Alves, SE Borgos, RAM Buijsen, ... EMBO molecular medicine 13 (4), e13243, 2021 | 217 | 2021 |
| Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials K Anthony, S Cirak, S Torelli, G Tasca, L Feng, V Arechavala-Gomeza, ... Brain 134 (12), 3547-3559, 2011 | 163 | 2011 |
| Muscle histology vs MRI in Duchenne muscular dystrophy M Kinali, V Arechavala-Gomeza, S Cirak, A Glover, M Guglieri, L Feng, ... Neurology 76 (4), 346-353, 2011 | 161 | 2011 |
| Delivery is key: lessons learnt from developing splice‐switching antisense therapies C Godfrey, LR Desviat, B Smedsrød, F Piétri‐Rouxel, MA Denti, P Disterer, ... EMBO molecular medicine 9 (5), 545-557, 2017 | 159 | 2017 |
| Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy S Cirak, L Feng, K Anthony, V Arechavala-Gomeza, S Torelli, C Sewry, ... Molecular Therapy 20 (2), 462-467, 2012 | 148 | 2012 |
| A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping GL Walmsley, V Arechavala-Gomeza, M Fernandez-Fuente, MM Burke, ... PloS one 5 (1), e8647, 2010 | 147 | 2010 |
| Immunohistological intensity measurements as a tool to assess sarcolemma‐associated protein expression V Arechavala‐Gomeza, M Kinali, L Feng, SC Brown, C Sewry, JE Morgan, ... Neuropathology and applied neurobiology 36 (4), 265-274, 2010 | 129 | 2010 |
| Dystrophin quantification: Biological and translational research implications K Anthony, V Arechavala-Gomeza, LE Taylor, A Vulin, Y Kaminoh, ... Neurology 83 (22), 2062-2069, 2014 | 126 | 2014 |
| Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials LJ Popplewell, C Adkin, V Arechavala-Gomeza, A Aartsma-Rus, ... Neuromuscular Disorders 20 (2), 102-110, 2010 | 113 | 2010 |
| Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: implication for clinical trials V Arechavala-Gomeza, M Kinali, L Feng, M Guglieri, G Edge, M Main, ... Neuromuscular Disorders 20 (5), 295-301, 2010 | 107 | 2010 |
| Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice A Malerba, PS Sharp, IR Graham, V Arechavala-Gomeza, K Foster, ... Molecular Therapy 19 (2), 345-354, 2011 | 104 | 2011 |
| Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy V Straub, P Balabanov, K Bushby, M Ensini, N Goemans, A De Luca, ... The Lancet Neurology 15 (8), 882-890, 2016 | 95 | 2016 |
| Antisense oligonucleotide-mediated exon skipping for Duchenne muscular dystrophy: progress and challenges V Arechavala-Gomeza, K Anthony, J Morgan, F Muntoni Current gene therapy 12 (3), 152-160, 2012 | 92 | 2012 |
| Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping K Anthony, V Arechavala-Gomeza, V Ricotti, S Torelli, L Feng, N Janghra, ... JAMA neurology 71 (1), 32-40, 2014 | 89 | 2014 |
| An overview of alternative splicing defects implicated in myotonic dystrophy type I A López-Martínez, P Soblechero-Martín, L de-la-Puente-Ovejero, ... Genes 11 (9), 1109, 2020 | 86 | 2020 |
| Splicing modulation therapy in the treatment of genetic diseases V Arechavala-Gomeza, B Khoo, A Aartsma-Rus The application of clinical genetics, 245-252, 2014 | 56 | 2014 |
| Exon skipping quantification by quantitative reverse-transcription polymerase chain reaction in Duchenne muscular dystrophy patients treated with the antisense oligomer eteplirsen K Anthony, L Feng, V Arechavala-Gomeza, M Guglieri, V Straub, ... Human Gene Therapy, Part B: Methods 23 (5), 336-345, 2012 | 55* | 2012 |
Volker StraubNewcastle University在 ncl.ac.uk 的电子邮件经过验证
