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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.
Afiliação
  • 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 em En | MEDLINE | ID: mdl-25568350

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Fator IX / Terapia Genética / Hemofilia B Tipo de estudo: Prognostic_studies Limite: Animals / Humans / Male Idioma: En Revista: Blood Ano de publicação: 2015 Tipo de documento: Article País de publicação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Fator IX / Terapia Genética / Hemofilia B Tipo de estudo: Prognostic_studies Limite: Animals / Humans / Male Idioma: En Revista: Blood Ano de publicação: 2015 Tipo de documento: Article País de publicação: Estados Unidos