Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates.

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Title: Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates.
Authors: Walia JS; 1] Department of Pediatrics, Queen's University, Kingston, Ontario, Canada [2] Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada [3] Department of Pediatrics & Child Health, University of Manitoba, Winnipeg, Manitoba, Canada [4] Manitoba Institute of Child Health, Winnipeg, Manitoba, Canada., Altaleb N; Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada., Bello A; 1] Department of Medical Microbiology, University of Manitoba, Winnipeg, Manitoba, Canada [2] Special Pathogens Program, Public Health Agency of Canada, Winnipeg, Manitoba, Canada., Kruck C; Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada., LaFave MC; Developmental Genomics Section, Translational and Functional Genomics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA., Varshney GK; Developmental Genomics Section, Translational and Functional Genomics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA., Burgess SM; Developmental Genomics Section, Translational and Functional Genomics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA., Chowdhury B; Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada., Hurlbut D; Department of Pathology & Molecular Medicine, Queen's University, Kingston, Ontario, Canada., Hemming R; Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada., Kobinger GP; 1] Department of Medical Microbiology, University of Manitoba, Winnipeg, Manitoba, Canada [2] Special Pathogens Program, Public Health Agency of Canada, Winnipeg, Manitoba, Canada., Triggs-Raine B; 1] Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada [2] Department of Pediatrics & Child Health, University of Manitoba, Winnipeg, Manitoba, Canada [3] Manitoba Institute of Child Health, Winnipeg, Manitoba, Canada.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2015 Mar; Vol. 23 (3), pp. 414-22. Date of Electronic Publication: 2014 Dec 17.
Publication Type: Journal Article; Research Support, N.I.H., Intramural; 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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  Data: Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates.
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  Data: <searchLink fieldCode="AU" term="%22Walia+JS%22">Walia JS</searchLink>; 1] Department of Pediatrics, Queen's University, Kingston, Ontario, Canada [2] Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada [3] Department of Pediatrics & Child Health, University of Manitoba, Winnipeg, Manitoba, Canada [4] Manitoba Institute of Child Health, Winnipeg, Manitoba, Canada.<br /><searchLink fieldCode="AU" term="%22Altaleb+N%22">Altaleb N</searchLink>; Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada.<br /><searchLink fieldCode="AU" term="%22Bello+A%22">Bello A</searchLink>; 1] Department of Medical Microbiology, University of Manitoba, Winnipeg, Manitoba, Canada [2] Special Pathogens Program, Public Health Agency of Canada, Winnipeg, Manitoba, Canada.<br /><searchLink fieldCode="AU" term="%22Kruck+C%22">Kruck C</searchLink>; Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada.<br /><searchLink fieldCode="AU" term="%22LaFave+MC%22">LaFave MC</searchLink>; Developmental Genomics Section, Translational and Functional Genomics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Varshney+GK%22">Varshney GK</searchLink>; Developmental Genomics Section, Translational and Functional Genomics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Burgess+SM%22">Burgess SM</searchLink>; Developmental Genomics Section, Translational and Functional Genomics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Chowdhury+B%22">Chowdhury B</searchLink>; Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada.<br /><searchLink fieldCode="AU" term="%22Hurlbut+D%22">Hurlbut D</searchLink>; Department of Pathology & Molecular Medicine, Queen's University, Kingston, Ontario, Canada.<br /><searchLink fieldCode="AU" term="%22Hemming+R%22">Hemming R</searchLink>; Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada.<br /><searchLink fieldCode="AU" term="%22Kobinger+GP%22">Kobinger GP</searchLink>; 1] Department of Medical Microbiology, University of Manitoba, Winnipeg, Manitoba, Canada [2] Special Pathogens Program, Public Health Agency of Canada, Winnipeg, Manitoba, Canada.<br /><searchLink fieldCode="AU" term="%22Triggs-Raine+B%22">Triggs-Raine B</searchLink>; 1] Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada [2] Department of Pediatrics & Child Health, University of Manitoba, Winnipeg, Manitoba, Canada [3] Manitoba Institute of Child Health, Winnipeg, Manitoba, Canada.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2015 Mar; Vol. 23 (3), pp. 414-22. <i>Date of Electronic Publication: </i>2014 Dec 17.
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