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    • 2. 发明申请
    • METHODS FOR TREATING BLOOD COAGULATION DISORDERS
    • 治疗血液凝血障碍的方法
    • US20110034539A1
    • 2011-02-10
    • US12566831
    • 2009-09-25
    • Samuel WADSWORTHAbraham SCARIA
    • Samuel WADSWORTHAbraham SCARIA
    • A61K31/713A61P7/02
    • C12N9/6437A61K48/00C07K2319/02C12N2799/021C12Y304/21021
    • The present invention relates to a method of treating an individual having a blood coagulation defect (e.g., hemophilia A, hemophilia B), comprising administering to the individual an effective amount of a DNA vector encoding modified Factor VII (FVII), wherein the modified Factor VII leads to generation of Factor VIIa in vivo. In a particular embodiment, the invention pertains to a method of treating an individual having a blood coagulation defect comprising administering to the individual an effective amount of a nucleic acid encoding a modified FVII wherein the modified FVII comprises a signal which codes for precursor cleavage by furin at the activation cleavage site of the modified FVII. The invention also relates to a method of treating an individual having a blood coagulation disorder comprising administering to the individual an effective amount of a nucleic acid encoding the light chain of human FVII and a nucleic acid encoding the heavy chain of human FVII operably linked to a leader sequence. Compositions, expression vectors and host cells comprising nucleic acid which encodes a modified Factor VII, wherein the modified Factor VII leads to generation of Factor VIIa in vivo is also encompassed by the present invention.
    • 本发明涉及一种治疗患有血液凝固缺陷的个体(例如,血友病A,血友病B)的方法,包括向个体施用有效量的编码修饰因子VII(FVII)的DNA载体,其中修饰因子 VII导致体内因子VIIa的产生。 在一个具体实施方案中,本发明涉及治疗具有凝血缺陷的个体的方法,其包括向个体施用有效量的编码修饰的FVII的核酸,其中修饰的FVII包含编码弗林蛋白酶前体切割的信号 在修饰的FVII的激活切割位点。 本发明还涉及一种治疗患有血液凝固病症的个体的方法,包括向个体施用有效量的编码人FVII轻链的核酸和编码人FVII重链的核酸,其可操作地连接到 领导序列。 包含编码修饰因子VII的核酸的组合物,表达载体和宿主细胞,其中修饰的因子VII导致体内产生因子VIIa也包括在本发明中。
    • 9. 发明授权
    • Methods for treating blood coagulation disorders
    • 治疗凝血障碍的方法
    • US07615537B2
    • 2009-11-10
    • US10057620
    • 2001-10-25
    • Abraham ScariaSamuel C. Wadsworth
    • Abraham ScariaSamuel C. Wadsworth
    • A61K48/00C12N15/63
    • C12N9/6437A61K48/00C07K2319/02C12N2799/021C12Y304/21021
    • The present invention relates to a method of treating an individual having a blood coagulation defect (e.g., hemophilia A, hemophilia B), comprising administering to the individual an effective amount of a DNA vector encoding modified Factor VII (FVII), wherein the modified Factor VII leads to generation of Factor VIIa in vivo. In a particular embodiment, the invention pertains to a method of treating an individual having a blood coagulation defect comprising administering to the individual an effective amount of a nucleic acid encoding a modified FVII wherein the modified FVII comprises a signal which codes for precursor cleavage by furin at the activation cleavage site of the modified FVII. The invention also relates to a method of treating an individual having a blood coagulation disorder comprising administering to the individual an effective amount of a nucleic acid encoding the light chain of human FVII and a nucleic acid encoding the heavy chain of human FVII operably linked to a leader sequence. Compositions, expression vectors and host cells comprising nucleic acid which encodes a modified Factor VII, wherein the modified Factor VII leads to generation of Factor VIIa in vivo is also encompassed by the present invention.
    • 本发明涉及一种治疗患有血液凝固缺陷的个体(例如,血友病A,血友病B)的方法,包括向个体施用有效量的编码修饰因子VII(FVII)的DNA载体,其中修饰因子 VII导致体内因子VIIa的产生。 在一个具体实施方案中,本发明涉及治疗具有凝血缺陷的个体的方法,其包括向个体施用有效量的编码修饰的FVII的核酸,其中修饰的FVII包含编码弗林蛋白酶前体切割的信号 在修饰的FVII的激活切割位点。 本发明还涉及一种治疗患有血液凝固病症的个体的方法,包括向个体施用有效量的编码人FVII轻链的核酸和编码人FVII重链的核酸,其可操作地连接到 领导序列。 包含编码修饰因子VII的核酸的组合物,表达载体和宿主细胞,其中修饰的因子VII导致体内产生因子VIIa也包括在本发明中。