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AN IMPROVED EXPRESSION CASSETTE FOR PACKAGING AND EXPRESSION OF VARIANT FACTOR VIII FOR THE TREATMENT OF HEMOSTASIS DISORDERS
专利权人:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
发明人:
Denise SABATINO,Katherine A. HIGH,Liron ELKOUBY
申请号:
US15503120
公开号:
US20170233456A1
申请日:
2015.08.13
申请国别(地区):
US
年份:
2017
代理人:
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.
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