Method to treat hemophilia by hepatic gene transfer of factor VIII/IX with vesicle vector

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 to the liver for delivery of an expression construct containing the coding sequence for factor VIII or IX driven by an appropriate promoter or factor VIII or IX protein.