Articles with public access mandates - Virginia Arechavala-Gomeza (ORCID 0000-0001-7703-3255)Learn more
Not available anywhere: 3
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
Mandates: US National Institutes of Health
Myoblots: dystrophin quantification by in‐cell western assay for a streamlined development of Duchenne muscular dystrophy (DMD) treatments
E Ruiz‐Del‐Yerro, I Garcia‐Jimenez, K Mamchaoui, ...
Neuropathology and applied neurobiology 44 (5), 463-473, 2018
Mandates: Government of Spain
Lessons learned from developing an oligonucleotide drug for a rare disease
P Soblechero-Martín, V Arechavala-Gomeza
RNA Therapeutics, 121-137, 2022
Mandates: Government of Spain
Available somewhere: 23
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
Mandates: British Heart Foundation
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
Mandates: Danish Council for Independent Research, National Institute of Health and …
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
Mandates: US National Institutes of Health
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
Mandates: Danish Council for Independent Research, National Institute of Health and …
Dystrophin quantification: Biological and translational research implications
K Anthony, V Arechavala-Gomeza, LE Taylor, A Vulin, Y Kaminoh, ...
Neurology 83 (22), 2062-2069, 2014
Mandates: US National Institutes of Health, UK Medical Research Council, National …
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
Mandates: National Institute for Health Research, UK
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
Mandates: Government of Spain
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
Mandates: UK Medical Research Council, Wellcome Trust
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
Mandates: European Commission
Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies
P Soblechero‐Martín, A López‐Martínez, L de la Puente‐Ovejero, ...
Neuropathology and Applied Neurobiology 47 (6), 711-723, 2021
Mandates: Government of Spain
Researcher's perceptions on publishing “negative” results and open access
L Echevarría, A Malerba, V Arechavala-Gomeza
Nucleic acid therapeutics 31 (3), 185-189, 2021
Mandates: European Commission, Government of Spain
Considerations in the preclinical assessment of the safety of antisense oligonucleotides
A Goyenvalle, C Jimenez-Mallebrera, W van Roon, S Sewing, AM Krieg, ...
nucleic acid therapeutics 33 (1), 1-16, 2023
Mandates: National Institute of Health and Medical Research, France, European …
The biomarker potential of miRNAs in myotonic dystrophy type I
E Koehorst, A Ballester-Lopez, V Arechavala-Gomeza, A Martínez-Piñeiro, ...
Journal of Clinical Medicine 9 (12), 3939, 2020
Mandates: Banking Foundation "la Caixa", Fondazione Telethon, Italy, European …
A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures
M Hiller, MS Falzarano, I Garcia-Jimenez, V Sardone, RC Verheul, ...
PLoS One 13 (10), e0204485, 2018
Mandates: National Institute for Health Research, UK, European Commission, Government …
Duchenne muscular dystrophy cell culture models created by CRISPR/Cas9 gene editing and their application in drug screening
P Soblechero-Martín, E Albiasu-Arteta, A Anton-Martinez, ...
Scientific Reports 11 (18188), 2021
Mandates: Government of Spain
miR-106b is a novel target to promote muscle regeneration and restore satellite stem cell function in injured Duchenne dystrophic muscle
L Rodriguez-Outeiriño, F Hernandez-Torres, FR de Acuña, A Rastrojo, ...
Molecular Therapy-Nucleic Acids 29, 769-786, 2022
Mandates: Government of Spain
Joining European Scientific Forces to Face Pandemics
MH Vasconcelos, S Alcaro, V Arechavala-Gomeza, J Baumbach, ...
Trends in microbiology 29 (2), 92-97, 2021
Mandates: European Commission, Luxembourg National Research Fund, Government of Spain …
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