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A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice.
Stavrou, Marina; Kagiava, Alexia; Choudury, Sarah G; Jennings, Matthew J; Wallace, Lindsay M; Fowler, Allison M; Heslegrave, Amanda; Richter, Jan; Tryfonos, Christina; Christodoulou, Christina; Zetterberg, Henrik; Horvath, Rita; Harper, Scott Q; Kleopa, Kleopas A.
Afiliación
  • Stavrou M; Neuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Kagiava A; Neuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Choudury SG; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Jennings MJ; Department of Clinical Neurosciences, University of Cambridge, Cambridge, United Kingdom.
  • Wallace LM; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Fowler AM; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Heslegrave A; Department of Neurodegenerative Disease, University College London (UCL) Institute of Neurology, London, United Kingdom.
  • Richter J; UK Dementia Research Institute at UCL, London, United Kingdom.
  • Tryfonos C; Molecular Virology Laboratory, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Christodoulou C; Molecular Virology Laboratory, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Zetterberg H; Molecular Virology Laboratory, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Horvath R; Department of Neurodegenerative Disease, University College London (UCL) Institute of Neurology, London, United Kingdom.
  • Harper SQ; UK Dementia Research Institute at UCL, London, United Kingdom.
  • Kleopa KA; Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology, The Sahlgrenska Academy at the University of Gothenburg, Mölndal, Sweden.
J Clin Invest ; 132(13)2022 07 01.
Article en En | MEDLINE | ID: mdl-35579942
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedad de Charcot-Marie-Tooth / Proteínas de la Mielina Límite: Animals Idioma: En Revista: J Clin Invest Año: 2022 Tipo del documento: Article País de afiliación: Chipre
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedad de Charcot-Marie-Tooth / Proteínas de la Mielina Límite: Animals Idioma: En Revista: J Clin Invest Año: 2022 Tipo del documento: Article País de afiliación: Chipre