Expression and characterization of a novel human recombinant factor IX molecule with enhanced in vitro and in vivo clotting activity
E Perot, N Enjolras, S Le Quellec, A Indalecio… - Thrombosis …, 2015 - Elsevier
Introduction Hemophilia B is an inherited X-linked recessive bleeding disorder, due to a
defect in human factor IX (FIX). The main treatment for hemophilia B is replacement therapy
using FIX concentrates. Prophylactic treatment in severe hemophilia B is very effective but is
limited by cost issues. Production of a recombinant FIX (rFIX) with enhanced clotting activity,
offering the possibility of fewer infusions and fewer costs with similar efficacy, is one of the
current challenges for hemophilia B treatment. The present study focused on an important …
defect in human factor IX (FIX). The main treatment for hemophilia B is replacement therapy
using FIX concentrates. Prophylactic treatment in severe hemophilia B is very effective but is
limited by cost issues. Production of a recombinant FIX (rFIX) with enhanced clotting activity,
offering the possibility of fewer infusions and fewer costs with similar efficacy, is one of the
current challenges for hemophilia B treatment. The present study focused on an important …