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Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice.
Duncan, F Jason; Naughton, Bartholomew J; Zaraspe, Kimberly; Murrey, Darren A; Meadows, Aaron S; Clark, Kelly Reed; Newsom, David E; White, Peter; Fu, Haiyan; McCarty, Douglas M.
Afiliação
  • Duncan FJ; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Naughton BJ; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Zaraspe K; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Murrey DA; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Meadows AS; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Clark KR; 1] Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, College of Medicine and Public Health, The Ohio State University, Columbus, Ohio, USA.
  • Newsom DE; Biomedical Genetics Core, Center for Microbial Pathogenesis, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • White P; 1] Department of Pediatrics, College of Medicine and Public Health, The Ohio State University, Columbus, Ohio, USA [2] Biomedical Genetics Core, Center for Microbial Pathogenesis, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Fu H; 1] Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, College of Medicine and Public Health, The Ohio State University, Columbus, Ohio, USA.
  • McCarty DM; 1] Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, College of Medicine and Public Health, The Ohio State University, Columbus, Ohio, USA.
Mol Ther ; 23(4): 638-47, 2015 Apr.
Article em En | MEDLINE | ID: mdl-25592334
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridose III / Técnicas de Transferência de Genes / Dependovirus / Vetores Genéticos / Hidrolases Limite: Animals / Humans Idioma: En 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: Mucopolissacaridose III / Técnicas de Transferência de Genes / Dependovirus / Vetores Genéticos / Hidrolases Limite: Animals / Humans Idioma: En Ano de publicação: 2015 Tipo de documento: Article