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CRISPR/Cas9-Induced (CTG⋅CAG)n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing.
van Agtmaal, Ellen L; André, Laurène M; Willemse, Marieke; Cumming, Sarah A; van Kessel, Ingeborg D G; van den Broek, Walther J A A; Gourdon, Geneviève; Furling, Denis; Mouly, Vincent; Monckton, Darren G; Wansink, Derick G; Wieringa, Bé.
Afiliación
  • van Agtmaal EL; Radboud Institute for Molecular Life Sciences, Department of Cell Biology, Radboud University Medical Center, Geert Grooteplein 28, 6525 GA, Nijmegen, the Netherlands.
  • André LM; Radboud Institute for Molecular Life Sciences, Department of Cell Biology, Radboud University Medical Center, Geert Grooteplein 28, 6525 GA, Nijmegen, the Netherlands.
  • Willemse M; Radboud Institute for Molecular Life Sciences, Department of Cell Biology, Radboud University Medical Center, Geert Grooteplein 28, 6525 GA, Nijmegen, the Netherlands.
  • Cumming SA; Institute of Molecular, Cell, and Systems Biology, College of Medical, Veterinary, and Life Sciences, University of Glasgow, Glasgow G12 8QQ, UK.
  • van Kessel IDG; Radboud Institute for Molecular Life Sciences, Department of Cell Biology, Radboud University Medical Center, Geert Grooteplein 28, 6525 GA, Nijmegen, the Netherlands.
  • van den Broek WJAA; Radboud Institute for Molecular Life Sciences, Department of Cell Biology, Radboud University Medical Center, Geert Grooteplein 28, 6525 GA, Nijmegen, the Netherlands.
  • Gourdon G; Inserm UMR 1163, 75015 Paris, France; Imagine Institute, Paris Descartes-Sorbonne Paris Cité University, 75270 Paris, France.
  • Furling D; UPMC Université Paris 06, Inserm UMRS974, CNRS FRE3617, Center for Research in Myology, Sorbonne Universités, 75252 Paris, France.
  • Mouly V; UPMC Université Paris 06, Inserm UMRS974, CNRS FRE3617, Center for Research in Myology, Sorbonne Universités, 75252 Paris, France.
  • Monckton DG; Institute of Molecular, Cell, and Systems Biology, College of Medical, Veterinary, and Life Sciences, University of Glasgow, Glasgow G12 8QQ, UK.
  • Wansink DG; Radboud Institute for Molecular Life Sciences, Department of Cell Biology, Radboud University Medical Center, Geert Grooteplein 28, 6525 GA, Nijmegen, the Netherlands. Electronic address: rick.wansink@radboudumc.nl.
  • Wieringa B; Radboud Institute for Molecular Life Sciences, Department of Cell Biology, Radboud University Medical Center, Geert Grooteplein 28, 6525 GA, Nijmegen, the Netherlands. Electronic address: be.wieringa@radboudumc.nl.
Mol Ther ; 25(1): 24-43, 2017 01 04.
Article en En | MEDLINE | ID: mdl-28129118
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Repeticiones de Trinucleótidos / Expansión de Repetición de Trinucleótido / Inestabilidad Genómica / Sistemas CRISPR-Cas / Proteína Quinasa de Distrofia Miotónica / Edición Génica / Distrofia Miotónica Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2017 Tipo del documento: Article País de afiliación: Países Bajos
Texto completo
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Repeticiones de Trinucleótidos / Expansión de Repetición de Trinucleótido / Inestabilidad Genómica / Sistemas CRISPR-Cas / Proteína Quinasa de Distrofia Miotónica / Edición Génica / Distrofia Miotónica Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2017 Tipo del documento: Article País de afiliación: Países Bajos