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. |
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| 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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