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Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models.
Conroy, Faith; Miller, Rachael; Alterman, Julia F; Hassler, Matthew R; Echeverria, Dimas; Godinho, Bruno M D C; Knox, Emily G; Sapp, Ellen; Sousa, Jaquelyn; Yamada, Ken; Mahmood, Farah; Boudi, Adel; Kegel-Gleason, Kimberly; DiFiglia, Marian; Aronin, Neil; Khvorova, Anastasia; Pfister, Edith L.
  • Conroy F; Department of Medicine, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Miller R; Department of Medicine, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Alterman JF; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Hassler MR; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Echeverria D; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Godinho BMDC; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Knox EG; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Sapp E; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, 02114, USA.
  • Sousa J; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Yamada K; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Mahmood F; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, 02114, USA.
  • Boudi A; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, 02114, USA.
  • Kegel-Gleason K; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, 02114, USA.
  • DiFiglia M; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, 02114, USA.
  • Aronin N; Department of Medicine, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Khvorova A; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01605, USA.
  • Pfister EL; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01605, USA. anastasia.khvorova@umassmed.edu.
Nat Commun ; 13(1): 5802, 2022 10 03.
Article en En | MEDLINE | ID: mdl-36192390
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Enfermedad de Huntington Límite: Animals Idioma: En Año: 2022 Tipo del documento: Article
Texto completo
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XML
PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Enfermedad de Huntington Límite: Animals Idioma: En Año: 2022 Tipo del documento: Article