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Virginie Mariot

Great Ormond Street Hospital

7

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35

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Myostatin is a quantifiable biomarker for monitoring pharmaco-gene therapy in Duchenne Muscular Dystrophy

2020 Molecular therapy. Methods & clinical development
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Gene Editing Targeting the DUX4 Polyadenylation Signal: A Therapy for FSHD?

2020 Journal of Personalized Medicine
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A deoxyribonucleic acid decoy trapping DUX4 for the treatment of Facioscapulohumeral Muscular Dystrophy

2020 Molecular therapy. Nucleic acids
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Chitosan-Shelled Nanobubbles Irreversibly Encapsulate Morpholino Conjugate Antisense Oligonucleotides and Are Ineffective for Phosphorodiamidate Morpholino-Mediated Gene Silencing of DUX4

2020 Nucleic Acid Therapeutics
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A one‐hour universal protocol for mouse genotyping

2020 Muscle & Nerve
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