Lecture6genetargetingHemophiliahumanchromosomes

Hemophilia a occurs in about 1 in 500010000 male

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Hemophilia A occurs in about 1 in 5,000–10,000 male births, while Hemophilia B occurs at about 1 in 20,000-34,000. Hemophilias A and B, X-linked recessive disorders
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Source and more info: hHp://www.ncbi.nlm.nih.gov/ books/NBK22589/ Hemophilia A Hemophilia B Blue: inactive clotting factor Orange: activated clotting factor Green: stimulatory clotting factor (not an enzyme) The clotting cascade
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Thrombin cleaves fibrinogen, the resulting fibrin units form a mesh = clot hHp://www.ncbi.nlm.nih.gov/books/NBK22589/
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Clicker question: Which of the following treatments are likely to help patients with Hemophilia A? A. Intravenous injections with Factor VIII B. Intravenous injections of Factor IX C. Blood transfusions with Thrombin D. Intravenous injection with activated Factor X E. All of the above
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Management of Hemophilia Standard care: replacement of the deficient factor with recombinant Factor VIII (Hemophilia A) or IX (Hemophilia B), produced from cultured hamster cells Factor replacement therapy has increased life expectancy from an average of 11 years before 1960s to about 65 years today. Commercially produced factor concentrates such as "Advate", a recombinant Factor VIII produced by Baxter International , come as a white powder in a vial which must be mixed with sterile water prior to intravenous injection. Wikipedia
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Summary
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Hemophilia A occurs in about 1 in 500010000 male births...

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