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CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells.
Min, Yi-Li; Li, Hui; Rodriguez-Caycedo, Cristina; Mireault, Alex A; Huang, Jian; Shelton, John M; McAnally, John R; Amoasii, Leonela; Mammen, Pradeep P A; Bassel-Duby, Rhonda; Olson, Eric N.
Afiliación
  • Min YL; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Li H; Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Rodriguez-Caycedo C; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Mireault AA; Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Huang J; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Shelton JM; Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • McAnally JR; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Amoasii L; Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Mammen PPA; Department of Internal Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Bassel-Duby R; Department of Internal Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Olson EN; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
Sci Adv ; 5(3): eaav4324, 2019 03.
Article en En | MEDLINE | ID: mdl-30854433
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Exones / Distrofina / Eliminación de Secuencia / Distrofia Muscular de Duchenne / Sistemas CRISPR-Cas Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Sci Adv Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Exones / Distrofina / Eliminación de Secuencia / Distrofia Muscular de Duchenne / Sistemas CRISPR-Cas Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Sci Adv Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos