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Integration-deficient lentiviral vectors expressing codon-optimized R338L human FIX restore normal hemostasis in Hemophilia B mice.
Suwanmanee, Thipparat; Hu, Genlin; Gui, Tong; Bartholomae, Cynthia C; Kutschera, Ina; von Kalle, Christof; Schmidt, Manfred; Monahan, Paul E; Kafri, Tal.
Afiliación
  • Suwanmanee T; Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina, USA.
  • Hu G; Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina, USA.
  • Gui T; Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina, USA.
  • Bartholomae CC; Department of Translational Oncology, National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Heidelberg, Germany.
  • Kutschera I; Department of Translational Oncology, National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Heidelberg, Germany.
  • von Kalle C; Department of Translational Oncology, National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Heidelberg, Germany.
  • Schmidt M; Department of Translational Oncology, National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Heidelberg, Germany.
  • Monahan PE; Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina, USA; Department of Pediatrics, Division of Hematology/Oncology, University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA.
  • Kafri T; Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina, USA; Department of Microbiology and Immunology, University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA. Electronic address: kafri@med.unc.edu.
Mol Ther ; 22(3): 567-574, 2014 Mar.
Article en En | MEDLINE | ID: mdl-23941813
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Factor IX / Hemofilia B / Lentivirus / Vectores Genéticos Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2014 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
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PubMed Links
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Factor IX / Hemofilia B / Lentivirus / Vectores Genéticos Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2014 Tipo del documento: Article País de afiliación: Estados Unidos