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Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.
Tran, Hélène; Moazami, Michael P; Yang, Huiya; McKenna-Yasek, Diane; Douthwright, Catherine L; Pinto, Courtney; Metterville, Jake; Shin, Minwook; Sanil, Nitasha; Dooley, Craig; Puri, Ajit; Weiss, Alexandra; Wightman, Nicholas; Gray-Edwards, Heather; Marosfoi, Miklos; King, Robert M; Kenderdine, Thomas; Fabris, Daniele; Bowser, Robert; Watts, Jonathan K; Brown, Robert H.
Affiliation
  • Tran H; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Moazami MP; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.
  • Yang H; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • McKenna-Yasek D; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Douthwright CL; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Pinto C; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Metterville J; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Shin M; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.
  • Sanil N; Research Pharmacy, University of Massachusetts Medical School, Worcester, MA, USA.
  • Dooley C; Research Pharmacy, University of Massachusetts Medical School, Worcester, MA, USA.
  • Puri A; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Weiss A; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Wightman N; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Gray-Edwards H; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Marosfoi M; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA.
  • King RM; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Kenderdine T; Department of Biomedical Engineering, Worcester Polytechnic Institute, Worcester, MA, USA.
  • Fabris D; Department of Chemistry, University of Connecticut, Storrs, CT, USA.
  • Bowser R; Department of Chemistry, University of Connecticut, Storrs, CT, USA.
  • Watts JK; Departments of Neurology and Neurobiology, Barrow Neurological Institute, Phoenix, AZ, USA.
  • Brown RH; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA. jonathan.watts@umassmed.edu.
Nat Med ; 28(1): 117-124, 2022 01.
Article in En | MEDLINE | ID: mdl-34949835
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Full text: 1 Database: MEDLINE Main subject: Oligonucleotides, Antisense / C9orf72 Protein / Mutation Limits: Animals / Humans Language: En Year: 2022 Type: Article
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Full text: 1 Database: MEDLINE Main subject: Oligonucleotides, Antisense / C9orf72 Protein / Mutation Limits: Animals / Humans Language: En Year: 2022 Type: Article