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Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice.
Klein, Arnaud F; Varela, Miguel A; Arandel, Ludovic; Holland, Ashling; Naouar, Naira; Arzumanov, Andrey; Seoane, David; Revillod, Lucile; Bassez, Guillaume; Ferry, Arnaud; Jauvin, Dominic; Gourdon, Genevieve; Puymirat, Jack; Gait, Michael J; Furling, Denis; Wood, Matthew Ja.
Afiliación
  • Klein AF; Sorbonne Université, Inserm, Association Institut de Myologie, Centre de Recherche en Myologie, Paris, France.
  • Varela MA; Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford, United Kingdom.
  • Arandel L; Department of Paediatrics, John Radcliffe Hospital, and.
  • Holland A; MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, United Kingdom.
  • Naouar N; Sorbonne Université, Inserm, Association Institut de Myologie, Centre de Recherche en Myologie, Paris, France.
  • Arzumanov A; Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford, United Kingdom.
  • Seoane D; Department of Paediatrics, John Radcliffe Hospital, and.
  • Revillod L; MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, United Kingdom.
  • Bassez G; Sorbonne Université, Inserm, Association Institut de Myologie, Centre de Recherche en Myologie, Paris, France.
  • Ferry A; Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford, United Kingdom.
  • Jauvin D; Medical Research Council, Laboratory of Molecular Biology, Cambridge, United Kingdom.
  • Gourdon G; Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford, United Kingdom.
  • Puymirat J; Department of Paediatrics, John Radcliffe Hospital, and.
  • Gait MJ; MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, United Kingdom.
  • Furling D; Sorbonne Université, Inserm, Association Institut de Myologie, Centre de Recherche en Myologie, Paris, France.
  • Wood MJ; Sorbonne Université, Inserm, Association Institut de Myologie, Centre de Recherche en Myologie, Paris, France.
J Clin Invest ; 129(11): 4739-4744, 2019 11 01.
Article en En | MEDLINE | ID: mdl-31479430
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Músculo Esquelético / Oligodesoxirribonucleótidos Antisentido / Péptidos de Penetración Celular / Proteína Quinasa de Distrofia Miotónica / Distrofia Miotónica Idioma: En Revista: J Clin Invest Año: 2019 Tipo del documento: Article
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Músculo Esquelético / Oligodesoxirribonucleótidos Antisentido / Péptidos de Penetración Celular / Proteína Quinasa de Distrofia Miotónica / Distrofia Miotónica Idioma: En Revista: J Clin Invest Año: 2019 Tipo del documento: Article