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Allele-specific silencing therapy for Dynamin 2-related dominant centronuclear myopathy.
Trochet, Delphine; Prudhon, Bernard; Beuvin, Maud; Peccate, Cécile; Lorain, Stéphanie; Julien, Laura; Benkhelifa-Ziyyat, Sofia; Rabai, Aymen; Mamchaoui, Kamel; Ferry, Arnaud; Laporte, Jocelyn; Guicheney, Pascale; Vassilopoulos, Stéphane; Bitoun, Marc.
Afiliación
  • Trochet D; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France.
  • Prudhon B; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France.
  • Beuvin M; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France.
  • Peccate C; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France.
  • Lorain S; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France.
  • Julien L; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France.
  • Benkhelifa-Ziyyat S; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France.
  • Rabai A; Department of Translational Medicine and Neurogenetics, IGBMC, INSERM U964, CNRS UMR7104, Collège de France, University of Strasbourg, Illkirch, France.
  • Mamchaoui K; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France.
  • Ferry A; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France.
  • Laporte J; Department of Translational Medicine and Neurogenetics, IGBMC, INSERM U964, CNRS UMR7104, Collège de France, University of Strasbourg, Illkirch, France.
  • Guicheney P; Institute of Cardiometabolism and Nutrition (ICAN), INSERM UMR_S1166, UPMC Univ Paris 06, Sorbonne Universités, Paris, France.
  • Vassilopoulos S; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France.
  • Bitoun M; Research Center for Myology, UPMC Univ Paris 06 and INSERM UMRS 974, Institute of Myology, Sorbonne Universités, Paris, France m.bitoun@institut-myologie.org.
EMBO Mol Med ; 10(2): 239-253, 2018 02.
Article en En | MEDLINE | ID: mdl-29246969
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Terapia Genética / Miopatías Estructurales Congénitas / Dinamina II / ARN Interferente Pequeño Límite: Animals / Humans Idioma: En Año: 2018 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Terapia Genética / Miopatías Estructurales Congénitas / Dinamina II / ARN Interferente Pequeño Límite: Animals / Humans Idioma: En Año: 2018 Tipo del documento: Article