Hepatic gene therapy for hemophilia B

  • Mark A. Kay
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 386)


A number of different cell types including keratinocytes, hepatodytes, fibroblasts, myoblasts, and endothelial cells have been targeted for factor IX (RIX) gene replacement therapy.1–7 Following gene transfer, these cells have produced functional RIX because of their ability to gamma-carboxylate the appropriate “gla” domains and secrete the functional protein. Most efforts to date have employed an ex vivo gene transfer method in rodents and involve transfer of the F.IX gene into the cells via a recombinant retroviral vector and subsequent transplantation of the cells back into the animal. Two major problems encountered in these studies have been the rather low concentration of circulating F.IX and/or the loss of F.IX expression over time. The inability to achieve long-term expression is primarily the result of loss of the transduced cells or inactivation of the expression vectors.


Gene Transfer Partial Hepatectomy Inhibitor Formation Recombinant Adenoviral Vector Gene Transfer Method 
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Copyright information

© Plenum Press, New York 1995

Authors and Affiliations

  • Mark A. Kay
    • 1
  1. 1.Investigator, Markey Molecular Medicine Center Assistant Professor of Medicine Division of Medical Genetics RG-25University of WashingtonSeattleUSA

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