Advances toward Gene Therapy for Hemophilia at the Millennium

Abstract excerpt

Hemophilia A and hemophilia B are both X chromosome-linked recessive bleeding disorders that affect about 1 in 5000 males and result from a deficiency in the coagulation factors VIII and IX, respectively. Severely affected individuals require frequent administration of factor VIII or factor IX preparations derived from human plasma, and more recently, from recombinant DNA technology. Although these preparations...

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