Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

Yin, Hao; Xue, Wen; Chen, Sidi; Bogorad, Roman L; Benedetti, Eric; Grompe, Markus; Koteliansky, Victor; Sharp, Phillip A; Jacks, Tyler; Anderson, Daniel G

Yin, Hao; Xue, Wen; Chen, Sidi; Bogorad, Roman L; Benedetti, Eric; Grompe, Markus; Koteliansky, Victor; Sharp, Phillip A; Jacks, Tyler; Anderson, Daniel G.

Afiliación

Yin H; 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [2].
Xue W; 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [2].
Chen S; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA.
Bogorad RL; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA.
Benedetti E; Oregon Stem Cell Center, Department of Pediatrics, Oregon Health and Science University, Portland, Oregon, USA.
Grompe M; Oregon Stem Cell Center, Department of Pediatrics, Oregon Health and Science University, Portland, Oregon, USA.
Koteliansky V; Skolkovo Institute of Science and Technology, Skolkovo, Russian Federation.
Sharp PA; 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [2] Department of Biology, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA.
Jacks T; 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [2] Department of Biology, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [3] Howard Hughes Medical Institute, Massachusetts Institute of Technolog
Anderson DG; 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [2] Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [3] Harvard-MIT Division of Health Sciences & Technology

Nat Biotechnol ; 32(6): 551-3, 2014 Jun.

Article en En | MEDLINE | ID: mdl-24681508

Asunto(s)

Proteínas Asociadas a CRISPR/genética; Sistemas CRISPR-Cas; Edición Génica/métodos; Terapia Genética/métodos; Hidrolasas/genética; Tirosinemias/terapia; Animales; Genoma/genética; Ratones; Ratones Transgénicos; Mutación/genética; Fenotipo; Resultado del Tratamiento

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Tirosinemias / Proteínas Asociadas a CRISPR / Sistemas CRISPR-Cas / Edición Génica / Hidrolasas Límite: Animals Idioma: En Revista: Nat Biotechnol Asunto de la revista: BIOTECNOLOGIA Año: 2014 Tipo del documento: Article Pais de publicación: Estados Unidos

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