CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice.

Zhang, Yu; Long, Chengzu; Li, Hui; McAnally, John R; Baskin, Kedryn K; Shelton, John M; Bassel-Duby, Rhonda; Olson, Eric N

Zhang, Yu; Long, Chengzu; Li, Hui; McAnally, John R; Baskin, Kedryn K; Shelton, John M; Bassel-Duby, Rhonda; Olson, Eric N.

Afiliación

Zhang Y; Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Long C; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Li H; Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
McAnally JR; Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Baskin KK; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Shelton JM; Hamon Center for Regenerative Science and 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 ; 3(4): e1602814, 2017 Apr.

Article en En | MEDLINE | ID: mdl-28439558

Asunto(s)

Sistemas CRISPR-Cas; Distrofina; Distrofia Muscular Animal/terapia; Distrofia Muscular de Duchenne/terapia; Miocitos Cardíacos/metabolismo; Animales; Distrofina/genética; Distrofina/metabolismo; Humanos; Ratones; Ratones Endogámicos mdx; Distrofia Muscular Animal/genética; Distrofia Muscular Animal/metabolismo; Distrofia Muscular Animal/patología; Distrofia Muscular de Duchenne/genética; Distrofia Muscular de Duchenne/metabolismo; Distrofia Muscular de Duchenne/patología; Miocitos Cardíacos/patología

Palabras clave

Duchenne muscular dystrophy; Skeletal muscle; dystrophin; exon skipping; guide RNA; iPSC

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Distrofina / Distrofia Muscular de Duchenne / Miocitos Cardíacos / Sistemas CRISPR-Cas / Distrofia Muscular Animal Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Sci Adv Año: 2017 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos

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