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Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo.
Ibraheim, Raed; Tai, Phillip W L; Mir, Aamir; Javeed, Nida; Wang, Jiaming; Rodríguez, Tomás C; Namkung, Suk; Nelson, Samantha; Khokhar, Eraj Shafiq; Mintzer, Esther; Maitland, Stacy; Chen, Zexiang; Cao, Yueying; Tsagkaraki, Emmanouela; Wolfe, Scot A; Wang, Dan; Pai, Athma A; Xue, Wen; Gao, Guangping; Sontheimer, Erik J.
Afiliação
  • Ibraheim R; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Tai PWL; Tessera Therapeutics, Inc., 200 Sidney Street, Cambridge, MA, 02139, USA.
  • Mir A; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Javeed N; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Wang J; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Rodríguez TC; Tessera Therapeutics, Inc., 200 Sidney Street, Cambridge, MA, 02139, USA.
  • Namkung S; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Nelson S; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Khokhar ES; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Mintzer E; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Maitland S; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Chen Z; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Cao Y; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Tsagkaraki E; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Wolfe SA; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Wang D; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Pai AA; Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Xue W; University of Crete School of Medicine, Heraklion, Crete, 71003, Greece.
  • Gao G; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Sontheimer EJ; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
Nat Commun ; 12(1): 6267, 2021 11 01.
Article em En | MEDLINE | ID: mdl-34725353
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridose II / Dependovirus / Tirosinemias / Reparo de DNA por Recombinação / Edição de Genes / Proteína 9 Associada à CRISPR / Vetores Genéticos Tipo de estudo: Evaluation_studies Limite: Animals / Female / Humans / Male Idioma: En Revista: Nat Commun Assunto da revista: BIOLOGIA / CIENCIA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridose II / Dependovirus / Tirosinemias / Reparo de DNA por Recombinação / Edição de Genes / Proteína 9 Associada à CRISPR / Vetores Genéticos Tipo de estudo: Evaluation_studies Limite: Animals / Female / Humans / Male Idioma: En Revista: Nat Commun Assunto da revista: BIOLOGIA / CIENCIA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos