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Antisense targeting of 3' end elements involved in DUX4 mRNA processing is an efficient therapeutic strategy for facioscapulohumeral dystrophy: a new gene-silencing approach.
Marsollier, Anne-Charlotte; Ciszewski, Lukasz; Mariot, Virginie; Popplewell, Linda; Voit, Thomas; Dickson, George; Dumonceaux, Julie.
Affiliation
  • Marsollier AC; Sorbonne Universités UPMC Univ Paris 06, Inserm, CNRS, Centre de Recherche en Myologie (CRM), GH Pitié Salpêtrière, 47 bld de l'hôpital, Paris 13, France and.
  • Ciszewski L; Centre of Biomedical Sciences, School of Biological Sciences, Royal Holloway University of London, Surrey TW20 0EX, UK.
  • Mariot V; Sorbonne Universités UPMC Univ Paris 06, Inserm, CNRS, Centre de Recherche en Myologie (CRM), GH Pitié Salpêtrière, 47 bld de l'hôpital, Paris 13, France and.
  • Popplewell L; Centre of Biomedical Sciences, School of Biological Sciences, Royal Holloway University of London, Surrey TW20 0EX, UK.
  • Voit T; Sorbonne Universités UPMC Univ Paris 06, Inserm, CNRS, Centre de Recherche en Myologie (CRM), GH Pitié Salpêtrière, 47 bld de l'hôpital, Paris 13, France and.
  • Dickson G; Centre of Biomedical Sciences, School of Biological Sciences, Royal Holloway University of London, Surrey TW20 0EX, UK.
  • Dumonceaux J; Sorbonne Universités UPMC Univ Paris 06, Inserm, CNRS, Centre de Recherche en Myologie (CRM), GH Pitié Salpêtrière, 47 bld de l'hôpital, Paris 13, France and julie.dumonceaux@upmc.fr.
Hum Mol Genet ; 25(8): 1468-78, 2016 Apr 15.
Article in En | MEDLINE | ID: mdl-26787513
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: RNA Precursors / Oligonucleotides, Antisense / Homeodomain Proteins / Muscular Dystrophy, Facioscapulohumeral / Morpholinos Type of study: Prognostic_studies Limits: Humans Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2016 Document type: Article
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: RNA Precursors / Oligonucleotides, Antisense / Homeodomain Proteins / Muscular Dystrophy, Facioscapulohumeral / Morpholinos Type of study: Prognostic_studies Limits: Humans Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2016 Document type: Article