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AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.
Crudele, Julie M; Finn, Jonathan D; Siner, Joshua I; Martin, Nicholas B; Niemeyer, Glenn P; Zhou, Shangzhen; Mingozzi, Federico; Lothrop, Clinton D; Arruda, Valder R.
Afiliación
  • Crudele JM; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA;
  • Finn JD; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA;
  • Siner JI; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA;
  • Martin NB; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA;
  • Niemeyer GP; Department of Biochemistry, University of Alabama at Birmingham, Birmingham, AL; and.
  • Zhou S; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA;
  • Mingozzi F; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA;
  • Lothrop CD; Department of Biochemistry, University of Alabama at Birmingham, Birmingham, AL; and.
  • Arruda VR; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA; Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
Blood ; 125(10): 1553-61, 2015 Mar 05.
Article en En | MEDLINE | ID: mdl-25568350
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Factor IX / Terapia Genética / Hemofilia B Tipo de estudio: Prognostic_studies Límite: Animals / Humans / Male Idioma: En Revista: Blood Año: 2015 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Factor IX / Terapia Genética / Hemofilia B Tipo de estudio: Prognostic_studies Límite: Animals / Humans / Male Idioma: En Revista: Blood Año: 2015 Tipo del documento: Article