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IN VIVO GENE THERAPY USING INTRAOSSEOUS DELIVERY OF A LENTIVIRALGENE CONSTRUCT
IN VIVO GENE THERAPY USING INTRAOSSEOUS DELIVERY OF A LENTIVIRALGENE CONSTRUCT
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机译:在使用肝癌构建体的体内基因疗法中
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摘要
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.
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