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Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates.
Walia, Jagdeep S; Altaleb, Naderah; Bello, Alexander; Kruck, Christa; LaFave, Matthew C; Varshney, Gaurav K; Burgess, Shawn M; Chowdhury, Biswajit; Hurlbut, David; Hemming, Richard; Kobinger, Gary P; Triggs-Raine, Barbara.
Afiliação
  • 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 Instit
  • 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.
Mol Ther ; 23(3): 414-22, 2015 Mar.
Article em En | MEDLINE | ID: mdl-25515709
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Sandhoff / Terapia Genética / Dependovirus / Cadeia beta da beta-Hexosaminidase / Gangliosídeo G(M2) / Vetores Genéticos Tipo de estudo: Etiology_studies / Prognostic_studies Limite: Animals Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2015 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Sandhoff / Terapia Genética / Dependovirus / Cadeia beta da beta-Hexosaminidase / Gangliosídeo G(M2) / Vetores Genéticos Tipo de estudo: Etiology_studies / Prognostic_studies Limite: Animals Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2015 Tipo de documento: Article