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Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system.
Zhang, Yu; Li, Hui; Min, Yi-Li; Sanchez-Ortiz, Efrain; Huang, Jian; Mireault, Alex A; Shelton, John M; Kim, Jiwoong; Mammen, Pradeep P A; Bassel-Duby, Rhonda; Olson, Eric N.
Afiliação
  • Zhang Y; Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Li H; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Min YL; Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Sanchez-Ortiz E; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Huang J; Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Mireault AA; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Shelton JM; Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Kim J; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Mammen PPA; Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Bassel-Duby R; Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Olson EN; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Sci Adv ; 6(8): eaay6812, 2020 02.
Article em En | MEDLINE | ID: mdl-32128412
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Distrofina / Dependovirus / Distrofia Muscular de Duchenne / Sistemas CRISPR-Cas / Edição de Genes / Vetores Genéticos Idioma: En Ano de publicação: 2020 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Distrofina / Dependovirus / Distrofia Muscular de Duchenne / Sistemas CRISPR-Cas / Edição de Genes / Vetores Genéticos Idioma: En Ano de publicação: 2020 Tipo de documento: Article