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Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection.
Chen, Shukkwan K; Hawley, Zachary C E; Zavodszky, Maria I; Hana, Sam; Ferretti, Daniel; Grubor, Branka; Hawes, Michael; Xu, Shanqin; Hamann, Stefan; Marsh, Galina; Cullen, Patrick; Challa, Ravi; Carlile, Thomas M; Zhang, Hang; Lee, Wan-Hung; Peralta, Andrea; Clarner, Pete; Wei, Cong; Koszka, Kathryn; Gao, Feng; Lo, Shih-Ching.
Afiliação
  • Chen SK; Biogen, Cambridge, MA, USA.
  • Hawley ZCE; Biogen, Cambridge, MA, USA.
  • Zavodszky MI; Biogen, Cambridge, MA, USA.
  • Hana S; Biogen, Cambridge, MA, USA.
  • Ferretti D; Biogen, Cambridge, MA, USA.
  • Grubor B; Biogen, Cambridge, MA, USA.
  • Hawes M; Charter Preclinical Services, Hudson, MA, USA.
  • Xu S; Biogen, Cambridge, MA, USA.
  • Hamann S; Biogen, Cambridge, MA, USA.
  • Marsh G; Biogen, Cambridge, MA, USA.
  • Cullen P; Biogen, Cambridge, MA, USA.
  • Challa R; Biogen, Cambridge, MA, USA.
  • Carlile TM; Biogen, Cambridge, MA, USA.
  • Zhang H; Biogen, Cambridge, MA, USA.
  • Lee WH; Biogen, Cambridge, MA, USA.
  • Peralta A; Biogen, Cambridge, MA, USA.
  • Clarner P; Biogen, Cambridge, MA, USA.
  • Wei C; Biogen, Cambridge, MA, USA.
  • Koszka K; Biogen, Cambridge, MA, USA.
  • Gao F; Biogen, Cambridge, MA, USA.
  • Lo SC; Biogen, Cambridge, MA, USA.
Mol Ther Nucleic Acids ; 34: 102057, 2023 Dec 12.
Article em En | MEDLINE | ID: mdl-37928442
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Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2023 Tipo de documento: Article