VIRGINIA
ARECHAVALA GOMEZA
Instituto de Investigación Sanitaria Biocruces Bizkaia
Barakaldo, EspañaInstituto de Investigación Sanitaria Biocruces Bizkaia-ko ikertzaileekin lankidetzan egindako argitalpenak (25)
2023
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Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides
Nucleic acid therapeutics, Vol. 33, Núm. 1, pp. 1-16
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Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics
Nucleic Acid Therapeutics, Vol. 33, Núm. 4, pp. 238-247
2022
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Antisense RNA Therapeutics: A Brief Overview
Methods in Molecular Biology (Humana Press Inc.), pp. 33-49
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Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystrophy
Methods in Molecular Biology (Humana Press Inc.), pp. 217-233
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Lessons learned from developing an oligonucleotide drug for a rare disease
RNA Therapeutics: The Evolving Landscape of RNA Therapeutics (Elsevier), pp. 121-137
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Preface
Methods in Molecular Biology
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miR-106b is a novel target to promote muscle regeneration and restore satellite stem cell function in injured Duchenne dystrophic muscle
Molecular Therapy - Nucleic Acids, Vol. 29, pp. 769-786
2021
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Delivery of oligonucleotide-based therapeutics: challenges and opportunities
EMBO Molecular Medicine, Vol. 13, Núm. 4
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Duchenne muscular dystrophy cell culture models created by CRISPR/Cas9 gene editing and their application in drug screening
Scientific Reports, Vol. 11, Núm. 1
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Joining European Scientific Forces to Face Pandemics
Trends in Microbiology
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Researcher's Perceptions on Publishing "negative" Results and Open Access
Nucleic Acid Therapeutics, Vol. 31, Núm. 3, pp. 185-189
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Sharing 'Negative' Results in Neuromuscular Research: A Positive Experience
Journal of Neuromuscular Diseases
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Special issue “genetic advances in neuromuscular disorders: From gene identification to gene therapy”
Genes
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Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies
Neuropathology and Applied Neurobiology, Vol. 47, Núm. 6, pp. 711-723
2020
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An overview of alternative splicing defects implicated in myotonic dystrophy type i
Genes, Vol. 11, Núm. 9, pp. 1-27
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The biomarker potential of mirnas in myotonic dystrophy type I
Journal of Clinical Medicine, Vol. 9, Núm. 12, pp. 1-18
2019
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COST Actions: fostering collaborative research for rare diseases
The Lancet Neurology
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Report of a TREAT-NMD/World duchenne organisation meeting on dystrophin quantification methodology
Journal of Neuromuscular Diseases
2018
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A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures
PLoS ONE, Vol. 13, Núm. 10
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Myoblots: dystrophin quantification by in-cell western assay for a streamlined development of Duchenne muscular dystrophy (DMD) treatments
Neuropathology and Applied Neurobiology, Vol. 44, Núm. 5, pp. 463-473