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AssigneeSeattle Children's Hospital d/b/a Seattle Children's Research Institute
InventorsCarol Hsing MIAO, David J. RAWLINGS, Chong LI
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Methods, compositions, and systems for treating subject(s) in need of plasma Factor VIII, particularly a subject having preexisting anti-FVIII inhibitory antibodies, are provided. The methods involve administering to the subject a therapeutically effective amount of an inflammation suppressor, a therapeutically effective amount of a CD8+ T cell depleting agent, and a therapeutically effective amount of a composition comprising a lentiviral vector (LV) comprising an optimized FVIII expression cassette expressibly linked to a megakaryocyte-specific promoter. Such methods, compositions, and systems are useful to treat subjects with blood clotting disorder(s), such as hemophilia A.
A method of treating a subject in need of plasma Factor VIII, comprising:
The method of any one of the previous claims, which is a method for treating a hemostasis related disorder in the subject, and the subject is in need of such treatment.
The method of any one of the previous claims, wherein the subject has hemophilia A (hemA), von Willebrand disease, bleeding associated with trauma or injury, thrombosis, thrombocytopenia, stroke, coagulopathy, disseminated vascular coagulation (DIC), or over-anticoagulation treatment disorder.
The method of any one of the previous claims, wherein the subject is a HemA subject with preexisting anti-FVIII inhibitory antibodies.
A method of treating a subject in need of plasma Factor VIII, essentially as described herein.
Dependencies are inferred from the claim text.