Recombinant Factor IX for Clinical and Research Use

Paul E Monahan; Jorge Di Paola

doi:10.1055/s-0030-1255444

Seminars in Thrombosis and Hemostasis, Table of Contents

Semin Thromb Hemost 2010; 36(5): 498-509
DOI: 10.1055/s-0030-1255444

Recombinant Factor IX for Clinical and Research Use

Paul E. Monahan¹ , Jorge Di Paola²

¹Department of Pediatrics, Gene Therapy Center, and the Harold R. Roberts Comprehensive Hemophilia Treatment Center at the University or North Carolina at Chapel Hill, Chapel Hill, North Carolina
²Department of Pediatrics and the Human Medical Genetics Program at the University of Colorado, Colorado, Denver

Abstract

ABSTRACT

The last significant advance in the therapy of hemophilia B was the introduction of recombinant factor IX (FIX), ensuring an advanced level of safety from potential infectious contaminants of plasma-derived clotting factors. Since that time, recombinant DNA techniques have been applied in research to elucidate the role of FIX and its functional domains within coagulation. At the same time, recombinant DNA technology has been applied to engineer an expanding spectrum of novel FIX therapies that are now being translating into clinical trials. The experience with the existing recombinant FIX product is reviewed with a focus on the novel products and the potential to improve the quality of life for individuals with hemophilia B.

KEYWORDS

Factor IX - hemophilia B - recombinant - hemostasis

Full Text

References

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Paul E MonahanM.D.

Associate Professor, Pediatrics, Hematology/Oncology, University of North Carolina at Chapel Hill

CB #7236, 1185 1st Floor Physicians Office Building, 170 Manning Drive, Chapel Hill, North Carolina 27599-7236

Email: Paul_Monahan@med.unc.edu