"A Factor IX-Deficient Mouse Model for Hemophilia B Gene Therapy."

According to an abstract submitted by the authors to the International Symposium on Gene Therapy for Hemophilia, held September 4-6, 1997, in Chapel Hill, North Carolina, "Hemophilia B is an X-linked bleeding disorder caused by mutations in the coagulation factor IX gene. The disease affects 1 in 30,000 males, and patients usually require blood transfusion or regular injections of plasma-derived or recombinant human factor IX which may cause life-threatening complications. Gene therapy is one of the alternative forms of therapy that has been proposed and tried on hemophilic canine models. However, a small animal model of the disease is desirable for studies of factor IX function and...

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