In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.
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| Title: | In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA. |
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| Authors: | Landau DJ; Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA.; Department of Molecular Genetics and Microbiology, Duke University, Durham, North Carolina, USA., Brooks ED; Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA.; Division of Laboratory Animal Resources, Duke University Medical Center, Durham, North Carolina, USA., Perez-Pinera P; Department of Biomedical Engineering, Duke University, Durham, North Carolina, USA.; Current address: Department of Bioengineering, University of Illinois at Urbana-Champaign, Urbana, Illinois, USA., Amarasekara H; Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA.; 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., Mefferd A; Department of Molecular Genetics and Microbiology, Duke University, Durham, North Carolina, USA., Li S; Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA., Bird A; Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA., Gersbach CA; Department of Biomedical Engineering, Duke University, Durham, North Carolina, USA., Koeberl DD; Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA.; Department of Molecular Genetics and Microbiology, Duke University, Durham, North Carolina, USA. |
| Source: | Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2016 Apr; Vol. 24 (4), pp. 697-706. Date of Electronic Publication: 2016 Feb 11. |
| Publication Type: | Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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