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Open Access
Peer-reviewed
Research Article
A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures
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Monika Hiller,
Roles Formal analysis, Investigation, Methodology, Resources, Visualization, Writing – original draft, Writing – review & editing
Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands
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Maria Sofia Falzarano,
Roles Formal analysis, Investigation, Methodology, Writing – review & editing
Affiliation UOL of Medical Genetics, University of Ferrara, Ferrara, Italy
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Iker Garcia-Jimenez,
Roles Formal analysis, Investigation, Writing – review & editing
Affiliation Neuromuscular Disorders Group, Biocruces Bizkaia Health Research Institute, Barakaldo, Spain
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Valentina Sardone,
Roles Formal analysis, Investigation, Methodology, Writing – review & editing
Affiliation Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health, London, United Kingdom
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Ruurd C. Verheul,
Roles Formal analysis, Methodology, Writing – review & editing
Affiliation BioMarin Nederland B.V., Leiden, The Netherlands
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Linda Popplewell,
Roles Formal analysis, Investigation, Methodology, Writing – review & editing
Affiliation Centre of Biomedical Sciences, School of Biological Sciences, Royal Holloway University of London, London, United Kingdom
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Karen Anthony,
Roles Formal analysis, Investigation, Methodology, Writing – review & editing
Affiliations Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health, London, United Kingdom, Faculty of Health and Society, University of Northampton, Northampton, United Kingdom
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Estibaliz Ruiz-Del-Yerro,
Roles Formal analysis, Writing – review & editing
Affiliation Neuromuscular Disorders Group, Biocruces Bizkaia Health Research Institute, Barakaldo, Spain
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Hana Osman,
Roles Formal analysis, Investigation, Methodology, Writing – review & editing
Affiliation UOL of Medical Genetics, University of Ferrara, Ferrara, Italy
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Jelle J. Goeman,
Roles Formal analysis, Validation, Visualization, Writing – review & editing
Affiliation Department of Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands
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Kamel Mamchaoui,
Roles Resources, Writing – review & editing
Affiliation INSERM, Institute of Myology, Center of Research in Myology, Sorbonne Universities, UPMC Univ Paris 6, Paris, France
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George Dickson,
Roles Project administration, Writing – review & editing
Affiliation Centre of Biomedical Sciences, School of Biological Sciences, Royal Holloway University of London, London, United Kingdom
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Alessandra Ferlini,
Roles Conceptualization, Project administration, Writing – review & editing
Affiliation UOL of Medical Genetics, University of Ferrara, Ferrara, Italy
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Francesco Muntoni,
Roles Conceptualization, Project administration, Writing – review & editing
Affiliations Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health, London, United Kingdom, MRC Centre for Neuromuscular Diseases, University College London Great Ormond Street Institute of Child Health, London, United Kingdom
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Annemieke Aartsma-Rus,
Roles Conceptualization, Project administration, Resources, Supervision, Writing – review & editing
Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands
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Virginia Arechavala-Gomeza,
Roles Conceptualization, Formal analysis, Investigation, Methodology, Project administration, Resources, Writing – review & editing
Affiliation Neuromuscular Disorders Group, Biocruces Bizkaia Health Research Institute, Barakaldo, Spain
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Nicole A. Datson,
Roles Investigation, Project administration, Writing – review & editing
Affiliation BioMarin Nederland B.V., Leiden, The Netherlands
⨯ - [ ... ],
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Pietro Spitali
Roles Conceptualization, Investigation, Project administration, Supervision, Writing – original draft, Writing – review & editing
* E-mail: p.spitali@lumc.nl
Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands
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A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures
- Monika Hiller,
- Maria Sofia Falzarano,
- Iker Garcia-Jimenez,
- Valentina Sardone,
- Ruurd C. Verheul,
- Linda Popplewell,
- Karen Anthony,
- Estibaliz Ruiz-Del-Yerro,
- Hana Osman,
- Jelle J. Goeman
- Published: October 2, 2018
- https://doi.org/10.1371/journal.pone.0204485