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A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
Hiller, Monika; Falzarano, Maria Sofia; Garcia-Jimenez, Iker; Sardone, Valentina; Verheul, Ruurd C; Popplewell, Linda; Anthony, Karen; Ruiz-Del-Yerro, Estibaliz; Osman, Hana; Goeman, Jelle J; Mamchaoui, Kamel; Dickson, George; Ferlini, Alessandra; Muntoni, Francesco; Aartsma-Rus, Annemieke; Arechavala-Gomeza, Virginia; Datson, Nicole A; Spitali, Pietro.
Affiliation
  • Hiller M; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
  • Falzarano MS; UOL of Medical Genetics, University of Ferrara, Ferrara, Italy.
  • Garcia-Jimenez I; Neuromuscular Disorders Group, Biocruces Bizkaia Health Research Institute, Barakaldo, Spain.
  • Sardone V; Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health, London, United Kingdom.
  • Verheul RC; BioMarin Nederland B.V., Leiden, The Netherlands.
  • Popplewell L; Centre of Biomedical Sciences, School of Biological Sciences, Royal Holloway University of London, London, United Kingdom.
  • Anthony K; Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health, London, United Kingdom.
  • Ruiz-Del-Yerro E; Faculty of Health and Society, University of Northampton, Northampton, United Kingdom.
  • Osman H; Neuromuscular Disorders Group, Biocruces Bizkaia Health Research Institute, Barakaldo, Spain.
  • Goeman JJ; UOL of Medical Genetics, University of Ferrara, Ferrara, Italy.
  • Mamchaoui K; Department of Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands.
  • Dickson G; INSERM, Institute of Myology, Center of Research in Myology, Sorbonne Universities, UPMC Univ Paris 6, Paris, France.
  • Ferlini A; Centre of Biomedical Sciences, School of Biological Sciences, Royal Holloway University of London, London, United Kingdom.
  • Muntoni F; UOL of Medical Genetics, University of Ferrara, Ferrara, Italy.
  • Aartsma-Rus A; Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health, London, United Kingdom.
  • Arechavala-Gomeza V; MRC Centre for Neuromuscular Diseases, University College London Great Ormond Street Institute of Child Health, London, United Kingdom.
  • Datson NA; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
  • Spitali P; Neuromuscular Disorders Group, Biocruces Bizkaia Health Research Institute, Barakaldo, Spain.
PLoS One ; 13(10): e0204485, 2018.
Article in En | MEDLINE | ID: mdl-30278058
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: RNA Splicing / Polymerase Chain Reaction / Dystrophin / Oligonucleotides, Antisense / Muscular Dystrophy, Duchenne Type of study: Clinical_trials / Guideline Limits: Humans Language: En Journal: PLoS One Journal subject: CIENCIA / MEDICINA Year: 2018 Document type: Article Affiliation country: Países Bajos
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: RNA Splicing / Polymerase Chain Reaction / Dystrophin / Oligonucleotides, Antisense / Muscular Dystrophy, Duchenne Type of study: Clinical_trials / Guideline Limits: Humans Language: En Journal: PLoS One Journal subject: CIENCIA / MEDICINA Year: 2018 Document type: Article Affiliation country: Países Bajos