首页>
外国专利>
IN VIVO GENE THERAPY USING DELIVERY OF A LENTIVIRAL GENE CONSTRUCT
IN VIVO GENE THERAPY USING DELIVERY OF A LENTIVIRAL GENE CONSTRUCT
展开▼
机译:使用慢病毒基因构建体进行体内基因治疗
展开▼
页面导航
摘要
著录项
相似文献
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.
展开▼