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1.发明授权Cloning and expression of biologically active .alpha.-N-acetylgalactosaminidase 失效生物活性α-N-乙酰半乳糖胺酶的克隆和表达
公开(公告)号：US5491075A
公开(公告)日：1996-02-13
申请号：US261578
申请日：1994-06-17
CPC分类号： C12Y302/01049 , C12N9/2465 , A61K38/00
摘要： The present invention involves the production of human .alpha.-GalNAc by cloning and expressing the .alpha.-GalNAc coding sequence in eukaryotic host cell expressions systems. The eukaryotic expression systems, and in particular the mammalian host cell expression systems described herein provide for the appropriate co-translational and post-translation modifications required or proper processing, e.g., glycosylation, phosphorylation, etc. and sorting of the expression product so that an active enzyme is produced.The .alpha.-GalNAc produced in accordance with the invention may be used in the treatment of Schindler disease or for the hydrolysis of .alpha.-N-acetylgalactosaminyl moieties in various glycoconjugates.
摘要翻译： 本发明涉及通过在真核宿主细胞表达系统中克隆和表达α-GalNAc编码序列来生产人α-GalNAc。 本文所述的真核表达系统，特别是哺乳动物宿主细胞表达系统提供了所需的适当的协同翻译和翻译后修饰或适当的加工，例如糖基化，磷酸化等，并且对表达产物进行排序，使得 生产活性酶。 根据本发明产生的α-GalNAc可用于治疗辛德勒病或用于各种糖缀合物中α-N-乙酰半乳糖胺基部分的水解。
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公开(公告)号：US5401650A
公开(公告)日：1995-03-28
申请号：US983451
申请日：1992-11-30
IPC分类号： C12N15/09 , A61K35/18 , A61K38/00 , C07K14/31 , C12N9/10 , C12N9/24 , C12N9/40 , C12N15/54 , C12N15/56 , C12R1/91 , C12N15/00
CPC分类号： C12N9/2465 , A61K35/18 , C07K14/31 , C12N9/1081 , C12Y302/01022 , C12Y302/01049 , A61K38/00 , C07K2319/00 , C07K2319/61 , C07K2319/705
摘要： The present invention involves the production of large quantities of human .alpha.-Gal A by cloning and expressing the .alpha.-Gal A coding sequence in eukaryotic host cell expression systems. The eukaryotic expression systems, and in particular the mammalian host cell expression system described herein provide for the appropriate cotranslational and posttranslational modifications required for proper processing, e.g., glycosylation, phosphorylation, etc. and sorting of the expression product so that an active enzyme is produced. In addition, the expression of fusion proteins which simplify purification is described.Using the methods described herein, the recombinant .alpha.-Gal A is secreted by the engineered host cells so that it is recovered from the culture medium in good yield. The .alpha.-Gal A produced in accordance with the invention may be used, but is not limited to, in the treatment in Fabry Disease; for the hydrolysis of .alpha.-galactosyl residues in glycoconjugates; and/or for the conversion of the blood group B antigen on erythrocytes to the blood group O antigen.
摘要翻译： 本发明涉及通过在真核宿主细胞表达系统中克隆并表达α-GalA编码序列来生产大量的人α-Gal。 真核表达系统，特别是本文所述的哺乳动物宿主细胞表达系统提供适当的共转译和翻译后修饰，以进行适当的加工（例如糖基化，磷酸化等）和排序表达产物，从而产生活性酶 。 此外，描述了简化纯化的融合蛋白的表达。 使用本文所述的方法，重组α-Gal被工程化的宿主细胞分泌，从而以良好的产率从培养基中回收。 可以使用根据本发明产生的α-Gal A，但不限于，在法布里病的治疗中; 用于糖缀合物中α-半乳糖苷残基的水解; 和/或用于将红细胞上的血型B抗原转化为血型O抗原。
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3.发明授权
公开(公告)号：US5356804A
公开(公告)日：1994-10-18
申请号：US602824
申请日：1990-10-24
CPC分类号： C12N9/2465 , A61K35/18 , A61K38/00
摘要： The present invention involves the production of large quantities of human .alpha.-Gal A by cloning and expressing the .alpha.-Gal A coding sequence in eukaryotic host cell expression systems. The eukaryotic expression systems, and in particular the mammalian host cell expression system described herein provide for the appropriate cotranslational and posttranslational modifications required for proper processing, e.g., glycosylation, phosphorylation, etc. and sorting of the expression product so that an glycosylation, phosphorylation, etc. and sorting of the expression product so that an active enzyme is produced. In addition, the expression of fusion proteins which simplify purification is described.Using the methods described herein, the recombinant .alpha.-Gal A is secreted by the engineered host cells so that it is recovered from the culture medium in good yield. The .alpha.-Gal A produced in accordance with the invention may be used in the treatment in Fabry Disease; for the hydrolysis of .alpha.-galactosyl residues in glycoconjugates; and/or for the conversion of the blood group B antigen on erythrocytes to the blood group O antigen.
摘要翻译： 本发明涉及通过在真核宿主细胞表达系统中克隆并表达α-GalA编码序列来生产大量的人α-Gal。 本文所述的真核表达系统，特别是哺乳动物宿主细胞表达系统提供适当的翻译和翻译后修饰，以进行适当的加工，例如糖基化，磷酸化等，并对表达产物进行分选，使得糖基化，磷酸化， 等等，并对表达产物进行分选以产生活性酶。 此外，描述了简化纯化的融合蛋白的表达。 使用本文所述的方法，重组α-Gal被工程化的宿主细胞分泌，从而以良好的产率从培养基中回收。 根据本发明产生的α-Gal可用于法布里病的治疗; 用于糖缀合物中α-半乳糖苷残基的水解; 和/或用于将红细胞上的血型B抗原转化为血型O抗原。
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4.发明授权Cloning and expression of biologically active .alpha.-n-acetylgalactosaminidase 失效生物活性α-N-乙酰半乳糖胺酶的克隆和表达
公开(公告)号：US5382524A
公开(公告)日：1995-01-17
申请号：US602608
申请日：1990-10-24
CPC分类号： C12Y302/01049 , C12N9/2465 , A61K38/00
摘要： The present invention involves the production of human .alpha.-GalNAc by cloning and expressing the .alpha.-GalNAc coding sequence in eukaryotic host cell expressions systems. The eukaryotic expression systems, and in particular the mammalian host cell expression systems described herein provide for the appropriate co-translational and post-translation modifications required or proper processing, e.g., glycosylation, phosphorylation, etc. and sorting of the expression product so that an active enzyme is produced.The .alpha.-GalNAc produced in accordance with the invention may be used in the treatment of Schindler disease or for the hydrolysis of .alpha.-N-acetylgalactosaminyl moieties in various glycoconjugates.
摘要翻译： 本发明涉及通过在真核宿主细胞表达系统中克隆和表达α-GalNAc编码序列来生产人α-GalNAc。 本文所述的真核表达系统，特别是哺乳动物宿主细胞表达系统提供了所需的适当的协同翻译和翻译后修饰或适当的加工，例如糖基化，磷酸化等，并且对表达产物进行排序，使得 生产活性酶。 根据本发明产生的α-GalNAc可用于治疗辛德勒病或用于各种糖缀合物中α-N-乙酰半乳糖胺基部分的水解。
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5.发明授权Cloning and expression of biologically active .alpha.-galactosidase A as a fusion protein 失效生物活性α-半乳糖苷酶A作为融合蛋白的克隆和表达
公开(公告)号：US5580757A
公开(公告)日：1996-12-03
申请号：US261577
申请日：1994-06-17
IPC分类号： C12N15/09 , A61K35/18 , A61K38/00 , C07K14/31 , C12N9/10 , C12N9/24 , C12N9/40 , C12N15/54 , C12N15/56 , C12R1/91 , C12P21/06 , C12N15/62
CPC分类号： C12N9/2465 , A61K35/18 , C07K14/31 , C12N9/1081 , C12Y302/01022 , C12Y302/01049 , A61K38/00 , C07K2319/00 , C07K2319/61 , C07K2319/705
摘要： The present invention involves the production of large quantities of human .alpha.-Gal A by cloning and expressing the .alpha.-Gal A coding sequence in eukaryotic host cell expression systems. The eukaryotic expression systems, and in particular the mammalian host cell expression system described herein provide for the appropriate cotranslational and posttranslational modifications required for proper processing, e.g., glycosylation, phosphorylation, etc. and sorting of the expression product so that an active enzyme is produced. In addition, the expression of fusion proteins which simplify purification is described.Using the methods described herein, the recombinant .alpha.-Gal A is secreted by the engineered host cells so that it is recovered from the culture medium in good yield. The .alpha.-Gal A produced in accordance with the invention may be used, but is not limited to, in the treatment in Fabry Disease; for the hydrolysis of .alpha.-galactosyl residues in glycoconjugates; and/or for the conversion of the blood group B antigen on erythrocytes to the blood group O antigen.
摘要翻译： 本发明涉及通过在真核宿主细胞表达系统中克隆并表达α-GalA编码序列来生产大量的人α-Gal。 真核表达系统，特别是本文所述的哺乳动物宿主细胞表达系统提供适当的共转译和翻译后修饰，以进行适当的加工（例如糖基化，磷酸化等）和排序表达产物，从而产生活性酶 。 此外，描述了简化纯化的融合蛋白的表达。 使用本文所述的方法，重组α-Gal被工程化的宿主细胞分泌，从而以良好的产率从培养基中回收。 可以使用根据本发明产生的α-Gal A，但不限于，在法布里病的治疗中; 用于糖缀合物中α-半乳糖苷残基的水解; 和/或用于将红细胞上的血型B抗原转化为血型O抗原。
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