Παρακολούθηση
Virginia Arechavala-Gomeza (ORCID 0000-0001-7703-3255)
Virginia Arechavala-Gomeza (ORCID 0000-0001-7703-3255)
Άλλα ονόματαVirginia Arechavala Gomeza
Ikerbasque Research Professor, Biobizkaia Health Research Institute, Spain
Η διεύθυνση ηλεκτρονικού ταχυδρομείου έχει επαληθευτεί στον τομέα bio-bizkaia.eus - Αρχική σελίδα
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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
11852011
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
8962009
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
2772021
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
2542007
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
1712017
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
1692011
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
1662011
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
1592010
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
1512012
Dystrophin quantification: Biological and translational research implications
K Anthony, V Arechavala-Gomeza, LE Taylor, A Vulin, Y Kaminoh, ...
Neurology 83 (22), 2062-2069, 2014
1342014
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
1332010
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
1152010
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
1122020
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
1112010
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
1072011
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
982016
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
972012
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
962014
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
582014
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
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