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In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.
Landau, Dustin J; Brooks, Elizabeth Drake; Perez-Pinera, Pablo; Amarasekara, Hiruni; Mefferd, Adam; Li, Songtao; Bird, Andrew; Gersbach, Charles A; Koeberl, Dwight D.
Afiliación
  • Landau DJ; Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA.
  • Brooks ED; Department of Molecular Genetics and Microbiology, Duke University, Durham, North Carolina, USA.
  • Perez-Pinera P; Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA.
  • Amarasekara H; Division of Laboratory Animal Resources, Duke University Medical Center, Durham, North Carolina, USA.
  • Mefferd A; Department of Biomedical Engineering, Duke University, Durham, North Carolina, USA.
  • Li S; Current address: Department of Bioengineering, University of Illinois at Urbana-Champaign, Urbana, Illinois, USA.
  • Bird A; Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA.
  • Gersbach CA; Current address: Michael E. DeBakey Department of Surgery, Division of Cardiothoracic Surgery, CHI St. Luke's Health-Baylor St. Luke's Medical Center, Houston, Texas, USA.
  • Koeberl DD; Department of Molecular Genetics and Microbiology, Duke University, Durham, North Carolina, USA.
Mol Ther ; 24(4): 697-706, 2016 Apr.
Article en En | MEDLINE | ID: mdl-26865405
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Enfermedad del Almacenamiento de Glucógeno Tipo I / Glucosa-6-Fosfatasa / ARN no Traducido / Endonucleasas Límite: Animals Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2016 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: Terapia Genética / Enfermedad del Almacenamiento de Glucógeno Tipo I / Glucosa-6-Fosfatasa / ARN no Traducido / Endonucleasas Límite: Animals Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2016 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos