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Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo.
Cabrera, Gabriela Toro; Meijboom, Katharina E; Abdallah, Abbas; Tran, Helene; Foster, Zachariah; Weiss, Alexandra; Wightman, Nicholas; Stock, Rachel; Gendron, Tania; Gruntman, Alisha; Giampetruzzi, Anthony; Petrucelli, Leonard; Brown, Robert H; Mueller, Christian.
  • Cabrera GT; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Meijboom KE; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Abdallah A; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Tran H; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Foster Z; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Weiss A; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Wightman N; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Stock R; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Gendron T; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Gruntman A; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Giampetruzzi A; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Rd., Jacksonville, FL, 32224, USA.
  • Petrucelli L; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Brown RH; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.
  • Mueller C; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Rd., Jacksonville, FL, 32224, USA.
Gene Ther ; 31(3-4): 105-118, 2024 Mar.
Article en En | MEDLINE | ID: mdl-37752346
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Enfermedades Neurodegenerativas / MicroARNs / Esclerosis Amiotrófica Lateral Límite: Animals / Humans Idioma: En Año: 2024 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Enfermedades Neurodegenerativas / MicroARNs / Esclerosis Amiotrófica Lateral Límite: Animals / Humans Idioma: En Año: 2024 Tipo del documento: Article