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公开(公告)号:US20130024960A1
公开(公告)日:2013-01-24
申请号:US13382953
申请日:2010-07-08
申请人: Amit Nathwani , Natalie Ward , Adrian Thrasher , Edward Tuddenham , John McVey , John Gray , Andrew Davidoff
发明人: Amit Nathwani , Natalie Ward , Adrian Thrasher , Edward Tuddenham , John McVey , John Gray , Andrew Davidoff
IPC分类号: C12N5/10 , A01K67/027 , C12N15/864 , C12N15/113 , A61K31/7088 , A61P7/04 , C12N15/85 , C12N15/12
CPC分类号: A61K48/0058 , A61K38/00 , A61K38/37 , C07K14/755 , C12N7/00 , C12N15/86 , C12N2710/10041 , C12N2710/14143 , C12N2750/14143 , C12N2800/22 , C12N2830/008
摘要: An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes are alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.
摘要翻译: 人类血液凝固因子8(VIII)和启动子的优化编码序列可用于载体,例如rAAV,用于引入因子VIII和/或其它凝血因子和转基因。 这些因子和转基因的实例是α-1-抗胰蛋白酶,以及参与凝血级联,肝癌生物学,溶酶体储存,尿素循环障碍和脂质储存疾病的那些因子和转基因。 由载体和序列转化的细胞产生的细胞,载体,蛋白质和糖蛋白可用于治疗。
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公开(公告)号:US09393323B2
公开(公告)日:2016-07-19
申请号:US13382953
申请日:2010-07-08
申请人: Amit Nathwani , Natalie Ward , Adrian Thrasher , Edward Tuddenham , John McVey , John Gray , Andrew Davidoff
发明人: Amit Nathwani , Natalie Ward , Adrian Thrasher , Edward Tuddenham , John McVey , John Gray , Andrew Davidoff
IPC分类号: C07H21/04 , A61K31/70 , A61K48/00 , C07K14/755 , C12N15/86 , A61K38/37 , C12N7/00 , A01K67/00 , A61K38/00
CPC分类号: A61K48/0058 , A61K38/00 , A61K38/37 , C07K14/755 , C12N7/00 , C12N15/86 , C12N2710/10041 , C12N2710/14143 , C12N2750/14143 , C12N2800/22 , C12N2830/008
摘要: An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes are alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.
摘要翻译: 人类血液凝固因子8(VIII)和启动子的优化编码序列可用于载体,例如rAAV,用于引入因子VIII和/或其它凝血因子和转基因。 这些因子和转基因的实例是α-1-抗胰蛋白酶,以及参与凝血级联,肝癌生物学,溶酶体储存,尿素循环障碍和脂质储存疾病的那些因子和转基因。 由载体和序列转化的细胞产生的细胞,载体,蛋白质和糖蛋白可用于治疗。
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