Publicaciones (45) Publicaciones de VIRGINIA ARECHAVALA GOMEZA

2023

  1. Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides

    Nucleic acid therapeutics, Vol. 33, Núm. 1, pp. 1-16

  2. Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics

    Nucleic Acid Therapeutics, Vol. 33, Núm. 4, pp. 238-247

  3. Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi

    Molecular Therapy Nucleic Acids, Vol. 34

2022

  1. Antisense RNA Therapeutics: A Brief Overview

    Methods in Molecular Biology (Humana Press Inc.), pp. 33-49

  2. 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

  3. Lessons learned from developing an oligonucleotide drug for a rare disease

    RNA Therapeutics: The Evolving Landscape of RNA Therapeutics (Elsevier), pp. 121-137

  4. Preface

    Methods in Molecular Biology

  5. 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

  1. Delivery of oligonucleotide-based therapeutics: challenges and opportunities

    EMBO Molecular Medicine, Vol. 13, Núm. 4

  2. Duchenne muscular dystrophy cell culture models created by CRISPR/Cas9 gene editing and their application in drug screening

    Scientific Reports, Vol. 11, Núm. 1

  3. Joining European Scientific Forces to Face Pandemics

    Trends in Microbiology

  4. Researcher's Perceptions on Publishing "negative" Results and Open Access

    Nucleic Acid Therapeutics, Vol. 31, Núm. 3, pp. 185-189

  5. Sharing 'Negative' Results in Neuromuscular Research: A Positive Experience

    Journal of Neuromuscular Diseases

  6. Special issue “genetic advances in neuromuscular disorders: From gene identification to gene therapy”

    Genes

  7. Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies

    Neuropathology and Applied Neurobiology, Vol. 47, Núm. 6, pp. 711-723

2020

  1. An overview of alternative splicing defects implicated in myotonic dystrophy type i

    Genes, Vol. 11, Núm. 9, pp. 1-27

  2. The biomarker potential of mirnas in myotonic dystrophy type I

    Journal of Clinical Medicine, Vol. 9, Núm. 12, pp. 1-18

2019

  1. COST Actions: fostering collaborative research for rare diseases

    The Lancet Neurology

  2. Report of a TREAT-NMD/World duchenne organisation meeting on dystrophin quantification methodology

    Journal of Neuromuscular Diseases

2018

  1. 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