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Precise therapeutic gene correction by a simple nuclease-induced double-stranded break.
Iyer, Sukanya; Suresh, Sneha; Guo, Dongsheng; Daman, Katelyn; Chen, Jennifer C J; Liu, Pengpeng; Zieger, Marina; Luk, Kevin; Roscoe, Benjamin P; Mueller, Christian; King, Oliver D; Emerson, Charles P; Wolfe, Scot A.
Afiliação
  • Iyer S; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Suresh S; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Guo D; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Daman K; Wellstone Muscular Dystrophy Program, University of Massachusetts Medical School, Worcester, MA, USA.
  • Chen JCJ; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Liu P; Wellstone Muscular Dystrophy Program, University of Massachusetts Medical School, Worcester, MA, USA.
  • Zieger M; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
  • Luk K; Wellstone Muscular Dystrophy Program, University of Massachusetts Medical School, Worcester, MA, USA.
  • Roscoe BP; Office of the Vice-Principal (Research), Queen's University, Kingston, Ontario, Canada.
  • Mueller C; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, USA.
  • King OD; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
  • Emerson CP; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA.
  • Wolfe SA; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, USA.
Nature ; 568(7753): 561-565, 2019 04.
Article em En | MEDLINE | ID: mdl-30944467
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Hermanski-Pudlak / Distrofia Muscular do Cíngulo dos Membros / Quebras de DNA de Cadeia Dupla / Reparo do DNA por Junção de Extremidades / Conectina / Proteínas Associadas a CRISPR Limite: Humans Idioma: En Ano de publicação: 2019 Tipo de documento: Article
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Hermanski-Pudlak / Distrofia Muscular do Cíngulo dos Membros / Quebras de DNA de Cadeia Dupla / Reparo do DNA por Junção de Extremidades / Conectina / Proteínas Associadas a CRISPR Limite: Humans Idioma: En Ano de publicação: 2019 Tipo de documento: Article