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METHOD TO TREAT HEMOPHILIA BY HEPATIC GENE TRANSFER OF FACTOR VIII/IX WITH VESICLE VECTOR
METHOD TO TREAT HEMOPHILIA BY HEPATIC GENE TRANSFER OF FACTOR VIII/IX WITH VESICLE VECTOR
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机译:载体因子VIII / IX肝基因转移治疗血友病的方法。
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摘要
Hemophilia is one of the most common genetic disorders. Standard therapies include transfusions with plasma products to provide clotting factors. The invention is a non-viral vesicle vector and method for the treatment of hemophilia. The vesicle vector contains the hepatitis B envelope protein to target the vesicle ot the liver for delivery of an expression construct containing the coding sequence for factor VIII or IX driven by an appropriate promoter of factor VIII or IX protein.
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