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公开(公告)号：US20060034813A1
公开(公告)日：2006-02-16
申请号：US11244087
申请日：2005-10-06
申请人： Christian Herder , Torsten Tonn , Manuel Grez , Erhard Seifried
发明人： Christian Herder , Torsten Tonn , Manuel Grez , Erhard Seifried
IPC分类号： A61K48/00 , C12N5/08 , C12N15/867
CPC分类号： C12N5/069 , C07K14/755 , C12N2501/10 , C12N2501/115 , C12N2501/125 , C12N2501/165 , C12N2501/998 , C12N2510/02 , C12N2799/021 , C12N2799/027
摘要： The invention provides a population of mature endothelial cells derived from human cord blood. The cells can be transduced with DNA encoding a therapeutically effective protein such as a blood coagulation factor. The cells are useful in a method for the production of a blood coagulation factor. They can further be used in hemophilia A or B gene therapy.
摘要翻译： 本发明提供了源自人脐带血的成熟内皮细胞群。 可以用编码治疗有效的蛋白质如血液凝固因子的DNA转导细胞。 细胞在生产凝血因子的方法中是有用的。 它们可以进一步用于血友病A或B基因治疗。
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2.发明申请Factor IX Variants with Clotting Activity in Absence of Their Cofactor and Their Use for Treating Bleeding Disorders 审中-公开因子IX变体具有凝血活性，缺乏其辅因子及其用于治疗出血性疾病的用途公开(公告)号：US20110217284A1
公开(公告)日：2011-09-08
申请号：US13001187
申请日：2009-07-28
申请人： Erhard Seifried , Jörg Schüttrumpf
发明人： Erhard Seifried , Jörg Schüttrumpf
CPC分类号： C12N9/644 , C12Y304/21022
摘要： The present invention relates to variants of a vitamin K-dependent serine protease of the coagulation cascade, preferably variants of factor IX (F.IX), wherein the variant is characterized in that it has clotting activity in absence of its cofactor. The present invention furthermore relates to the use of these variants for the treatment and/or prophylaxis of bleeding disorders, in particular hemophilia A and/or hemophilia B or hemophilia caused or complicated by inhibitory antibodies to F.VIII. The present invention also relates to further variants of factor IX (F.IX) which have desired properties and can, thus be tailored for respective specific therapeutic applications.
摘要翻译： 本发明涉及凝血级联的维生素K依赖性丝氨酸蛋白酶的变体，优选因子IX（F.IX）的变体，其中所述变体的特征在于其不存在其辅因子时具有凝血活性。 本发明还涉及这些变体用于治疗和/或预防出血性疾病，特别是血友病A和/或血友病B或由F.VIII的抑制性抗体引起或复合的血友病的用途。 本发明还涉及因子IX（F.IX）的其它变体，其具有期望的性质，并且因此可以针对各自特定的治疗应用而定制。
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公开(公告)号：US10125357B2
公开(公告)日：2018-11-13
申请号：US13001187
申请日：2009-07-28
申请人： Erhard Seifried , Jörg Schüttrumpf
发明人： Erhard Seifried , Jörg Schüttrumpf
IPC分类号： C12N9/64
摘要： The present invention relates to variants of a vitamin K-dependent serine protease of the coagulation cascade, preferably variants of factor IX (F.IX), wherein the variant is characterized in that it has clotting activity in absence of its cofactor. The present invention furthermore relates to the use of these variants for the treatment and/or prophylaxis of bleeding disorders, in particular hemophilia A and/or hemophilia B or hemophilia caused or complicated by inhibitory antibodies to F.VIII. The present invention also relates to further variants of factor IX (F.IX) which have desired properties and can, thus be tailored for respective specific therapeutic applications.
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