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G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects.
Wang, Feng; Calvo-Roitberg, Ezequiel; Rembetsy-Brown, Julia M; Fang, Minggang; Sousa, Jacquelyn; Kartje, Zachary J; Krishnamurthy, Pranathi Meda; Lee, Jonathan; Green, Michael R; Pai, Athma A; Watts, Jonathan K.
Afiliação
  • Wang F; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
  • Calvo-Roitberg E; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
  • Rembetsy-Brown JM; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
  • Fang M; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
  • Sousa J; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
  • Kartje ZJ; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
  • Krishnamurthy PM; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
  • Lee J; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
  • Green MR; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
  • Pai AA; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
  • Watts JK; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.
Nucleic Acids Res ; 50(22): 12657-12673, 2022 12 09.
Article em En | MEDLINE | ID: mdl-36511872

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Ataxia de Friedreich / Regulação da Expressão Gênica / Oligonucleotídeos Antissenso Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Ataxia de Friedreich / Regulação da Expressão Gênica / Oligonucleotídeos Antissenso Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article