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Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B

Miesbach, W., Meijer, K., Coppens, M., Kampmann, P., Klamroth, R., Schutgens, R., Tangelder, M., Castaman, G., Schwaeble, J., Bonig, H., Seifried, E., Cattaneo, F., Meyer, C. & Leebeek, F. W. G., 1-Mar-2018, In : Blood. 131, 9, p. 1022-1031 10 p.

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  • Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

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DOI

  • Wolfgang Miesbach
  • Karina Meijer
  • Michiel Coppens
  • Peter Kampmann
  • Robert Klamroth
  • Roger Schutgens
  • Marco Tangelder
  • Giancarlo Castaman
  • Joachim Schwaeble
  • Halvard Bonig
  • Erhard Seifried
  • Federica Cattaneo
  • Christian Meyer
  • Frank W. G. Leebeek

Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for FIX concentrate. AMT-060 combines an adeno-associated virus-5 (AAV5) vector with a liver-specific promoter driving expression of a codon-optimized wild-type human FIX gene. This multinational, open-label study included 10 adults with hemophilia B (FIX

Original languageEnglish
Pages (from-to)1022-1031
Number of pages10
JournalBlood
Volume131
Issue number9
Publication statusPublished - 1-Mar-2018

    Keywords

  • REFERENCE-STANDARD MATERIAL, CLINICAL-TRIAL, LIVER, EFFICACY, SAFETY, TYPE-2, PADUA

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