AN IMPROVED EXPRESSION CASSETTE FOR PACKAGING AND EXPRESSION OF VARIANT FACTOR VIII FOR THE TREATMENT OF HEMOSTASIS DISORDERS

摘要

Factor VIII variants and methods of use thereof are disclosed. In particular embodiments, Factor VIII variants are expressed more efficiently by cells over wild-type Factor VIII proteins, are secreted at increased levels by cells over wild-type Factor VIII proteins, exhibit enhanced activity over wild-type Factor VIII proteins and are packaged more efficiently into viral vectors.