AAV

Gene therapy for Hemophilia B

sa smith | December 14, 2011

For the millionth time-- Viruses are not just pathogens that make us sick. In the modern world, viruses have been domesticated, and are now used to treat/cure diseases. Example #gazillion: Hemophilia B: Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B This study is far from perfect, and of course very preliminary, but still very exciting. Hemophilia B is a disease in males caused by point mutations/deletions/etc in the clotting Factor IX gene. If you dont make Factor IX, you wont clot properly, and will have all of the health issues we associate with hemophilia.…

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Gene therapy for Hemophilia B

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