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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.
Afiliación
  • 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
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that affects motor neurons, causing progressive muscle weakness and respiratory failure. The presence of an expanded hexanucleotide repeat in chromosome 9 open reading frame 72 (C9ORF72) is the most frequent mutation causing familial ALS and frontotemporal dementia (FTD). To determine if suppressing expression of C9ORF72 gene products can reduce toxicity, we designed a set of artificial microRNAs (amiRNA) targeting the human C9ORF72 gene. Here we report that an AAV9-mediated amiRNA significantly suppresses expression of the C9ORF72 mRNA, protein, and toxic dipeptide repeat proteins generated by the expanded repeat in the brain and spinal cord of C9ORF72 transgenic mice.
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedades Neurodegenerativas / MicroARNs / Esclerosis Amiotrófica Lateral Límite: Animals / Humans Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedades Neurodegenerativas / MicroARNs / Esclerosis Amiotrófica Lateral Límite: Animals / Humans Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos