Antithrombin deficiency is a hereditary disease causing low levels or defects of antithrombin, a blood protein required for controlling clot formation. Patients are at risk of blood clots, organ damage, and death. They usually have to take oral anticoagulant drugs like warfarin for life.
During high-risk procedures like surgery or childbirth, oral anticoagulants must be discontinued to minimize the chance of bleeding complications. While patients are off oral anticoagulants, they are given preventive treatment with antithrombin derived from pooled human blood. With any human blood product there is a small risk of infection with diseases like hepatitis C. And human antithrombin supplies are not plentiful.
Clever researchers found an ingenious solution. Put a human antithrombin gene in goats, milk them, isolate the human antithrombin protein from the milk, and voila! An udderly safe and plentiful source. A Brit might call it bleatin’ brilliant. (more…)