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Premature termination codon readthrough upregulates progranulin expression and improves lysosomal function in preclinical models of GRN deficiency.
Frew, Jonathan; Baradaran-Heravi, Alireza; Balgi, Aruna D; Wu, Xiujuan; Yan, Tyler D; Arns, Steve; Shidmoossavee, Fahimeh S; Tan, Jason; Jaquith, James B; Jansen-West, Karen R; Lynn, Francis C; Gao, Fen-Biao; Petrucelli, Leonard; Feldman, Howard H; Mackenzie, Ian R; Roberge, Michel; Nygaard, Haakon B.
  • Frew J; Division of Neurology, University of British Columbia, Vancouver, British Columbia, Canada.
  • Baradaran-Heravi A; Department of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, British Columbia, Canada.
  • Balgi AD; Department of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, British Columbia, Canada.
  • Wu X; Division of Neurology, University of British Columbia, Vancouver, British Columbia, Canada.
  • Yan TD; Division of Neurology, University of British Columbia, Vancouver, British Columbia, Canada.
  • Arns S; adMare BioInnovations, Vancouver, British Columbia, Canada.
  • Shidmoossavee FS; adMare BioInnovations, Vancouver, British Columbia, Canada.
  • Tan J; adMare BioInnovations, Vancouver, British Columbia, Canada.
  • Jaquith JB; JAQJAM Consulting, Cobourg, Ontario, Canada.
  • Jansen-West KR; Department of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.
  • Lynn FC; Department of Surgery, University of British Columbia, Faculty of Medicine, Vancouver, British Columbia, Canada.
  • Gao FB; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Petrucelli L; Department of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.
  • Feldman HH; Division of Neurology, University of British Columbia, Vancouver, British Columbia, Canada.
  • Mackenzie IR; Department of Neurosciences, University of California, San Diego, San Diego, CA, USA.
  • Roberge M; Department of Pathology and Laboratory Medicine, University of British Columbia, Vancouver, British Columbia, Canada.
  • Nygaard HB; Department of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, British Columbia, Canada.
Mol Neurodegener ; 15(1): 21, 2020 03 16.
Article en En | MEDLINE | ID: mdl-32178712

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Gentamicinas / Expresión Génica / Degeneración Lobar Frontotemporal / Progranulinas / Lisosomas Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Año: 2020 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Gentamicinas / Expresión Génica / Degeneración Lobar Frontotemporal / Progranulinas / Lisosomas Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Año: 2020 Tipo del documento: Article