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1.发明申请READTHROUGH ACETYLCHOLINESTERASE (ACHE-R) FOR TREATING OR PREVENTING PARKINSON'S DISEASE 审中-公开准备乙酰胆碱酯酶（ACHE-R）治疗或预防帕金森病公开(公告)号：US20130164288A1
公开(公告)日：2013-06-27
申请号：US13820525
申请日：2011-09-07
申请人： Hermona Soreq , Ilya Ruderfer , Yoseph Shaaltiel
发明人： Hermona Soreq , Ilya Ruderfer , Yoseph Shaaltiel
CPC分类号： A61K38/465 , A61K47/60 , C12N9/18 , C12Y301/01007
摘要： A method of treating or preventing Parkinson's disease in a subject in need thereof is disclosed. The method comprises administering to the subject a therapeutically effective amount of AChE-R, wherein the AChE-R is devoid of an N-terminal extension. An additional method of treating or preventing Parkinson's disease in a subject is disclosed. The method comprises administering to the subject a therapeutically effective amount of AChE-R, wherein the AChE-R comprises a modification for increasing bioavailability.
摘要翻译： 公开了在有需要的受试者中治疗或预防帕金森病的方法。 该方法包括向受试者施用治疗有效量的AChE-R，其中AChE-R缺乏N-末端延伸。 公开了一种在受试者中治疗或预防帕金森病的另外的方法。 该方法包括向受试者施用治疗有效量的AChE-R，其中AChE-R包含用于提高生物利用度的修饰。
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公开(公告)号：US09194011B2
公开(公告)日：2015-11-24
申请号：US13582482
申请日：2011-03-02
申请人： Avidor Shulman , Ilya Ruderfer , Tehila Ben-Moshe , Talia Shekhter , Yaniv Azulay , Yoseph Shaaltiel , Tali Kizhner
发明人： Avidor Shulman , Ilya Ruderfer , Tehila Ben-Moshe , Talia Shekhter , Yaniv Azulay , Yoseph Shaaltiel , Tali Kizhner
CPC分类号： C12N9/2465 , A61K38/00 , C12Y302/01022
摘要： Multimeric protein structures comprising at least two alpha-galactosidase monomers being covalently linked to one another via a linking moiety are disclosed herein, as well a process for preparing same, and methods of treating Fabry disease via administration of a multimeric protein structure. The disclosed multimeric protein structures exhibit an improved performance, in terms of enhanced activity and/or a longer lasting activity under both lysosomal conditions and in a serum environment.
摘要翻译： 本文公开了包含通过连接部分彼此共价连接的至少两个α-半乳糖苷酶单体的多聚体蛋白质结构，以及其制备方法，以及通过施用多聚体蛋白质结构治疗法布里病的方法。 根据在溶酶体条件和血清环境中增强的活性和/或更持久的活性方面，所公开的多聚体蛋白质结构表现出改进的性能。
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公开(公告)号：US20120328589A1
公开(公告)日：2012-12-27
申请号：US13582441
申请日：2011-03-02
申请人： Ilya Ruderfer , Tali Kizhner , Avidor Shulman , Yoseph Shaaltiel
发明人： Ilya Ruderfer , Tali Kizhner , Avidor Shulman , Yoseph Shaaltiel
CPC分类号： C07K14/525
摘要： Multimeric protein structures comprising at least two glucocerebrosidase molecules being covalently linked to one another via a linking moiety are disclosed herein, as well a process for preparing same, and uses thereof in the treatment of Gaucher disease. The multimeric protein structures are characterized by longer-lasting activity as compared to native glucocerebrosidase both in serum and in lysosomes.
摘要翻译： 本文公开了包含通过连接部分彼此共价连接的至少两种葡糖脑苷脂酶分子的多聚体蛋白质结构，以及其制备方法及其在治疗戈谢病中的用途。 与血浆和溶酶体中的天然葡糖脑苷脂酶相比，多聚体蛋白质结构的特征在于持久的活性。
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4.发明授权Production of high mannose proteins in plant culture and therapeutic uses thereof 有权植物培养中高甘露糖蛋白的生产及其治疗用途公开(公告)号：US08790641B2
公开(公告)日：2014-07-29
申请号：US12385894
申请日：2009-04-23
CPC分类号： A61K38/47 , A61K9/0053 , A61K36/23 , C07K2319/04 , C12N9/2405 , C12N9/2434 , C12N9/2465 , C12N15/8257 , C12P21/005 , C12Y302/01022 , C12Y302/01045
摘要： A device, system and method for producing glycosylated proteins in plant culture, particularly proteins having a high mannose glycosylation, while targeting such proteins with an ER signal and/or by-passing the Golgi. The invention further relates to vectors and methods for expression and production of enzymatically active high mannose lysosomal enzymes using transgenic plant root, particularly carrot cells. More particularly, the invention relates to host cells, particularly transgenic suspended carrot cells, vectors and methods for high yield expression and production of biologically active high mannose Glucocerebrosidase (GCD). The invention further provides for compositions and methods for the treatment of lysosomal storage diseases.
摘要翻译： 用于在植物培养物中生产糖基化蛋白质的装置，系统和方法，特别是具有高甘露糖糖基化的蛋白质，同时用ER信号靶向这种蛋白质和/或旁路高尔基体。 本发明进一步涉及使用转基因植物根特别是胡萝卜细胞表达和产生酶活性高甘露糖溶酶体酶的载体和方法。 更具体地，本发明涉及宿主细胞，特别是转基因悬浮的胡萝卜细胞，用于高产量表达和生产生物活性高甘露糖葡糖脑苷脂酶（GCD）的载体和方法。 本发明还提供了用于治疗溶酶体贮积病的组合物和方法。
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公开(公告)号：US08227230B2
公开(公告)日：2012-07-24
申请号：US13080692
申请日：2011-04-06
CPC分类号： A61K38/47 , A61K9/0053 , A61K36/23 , C07K2319/04 , C12N9/2405 , C12N9/2434 , C12N9/2465 , C12N15/8257 , C12P21/005 , C12Y302/01022 , C12Y302/01045
摘要： A device, system and method for producing glycosylated proteins in plant culture, particularly proteins having a high mannose glycosylation, while targeting such proteins with an ER signal and/or by-passing the Golgi. The invention further relates to vectors and methods for expression and production of enzymatically active high mannose lysosomal enzymes using transgenic plant root, particularly carrot cells. More particularly, the invention relates to host cells, particularly transgenic suspended carrot cells, vectors and methods for high yield expression and production of biologically active high mannose Glucocerebrosidase (GCD). The invention further provides for compositions and methods for the treatment of lysosomal storage diseases.
摘要翻译： 用于在植物培养物中生产糖基化蛋白质的装置，系统和方法，特别是具有高甘露糖糖基化的蛋白质，同时用ER信号靶向这种蛋白质和/或旁路高尔基体。 本发明进一步涉及使用转基因植物根特别是胡萝卜细胞表达和产生酶活性高甘露糖溶酶体酶的载体和方法。 更具体地，本发明涉及宿主细胞，特别是转基因悬浮的胡萝卜细胞，用于高产量表达和生产生物活性高甘露糖葡糖脑苷脂酶（GCD）的载体和方法。 本发明还提供了用于治疗溶酶体贮积病的组合物和方法。
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公开(公告)号：US20100196345A1
公开(公告)日：2010-08-05
申请号：US12451188
申请日：2008-04-30
CPC分类号： C12P21/005 , C07K2319/04 , C12N9/2402 , C12N15/8257 , C12Y302/01022 , C12Y302/01045
摘要： A device, system and method for producing glycosylated proteins in plant culture, particularly proteins having a high mannose glycosylation, while targeting such proteins with an ER signal and/or by-passing the Golgi. The invention further relates to vectors and methods for expression and production of enzymatically active high mannose lysosomal enzymes using transgenic plant root, particularly carrot cells. More particularly, the invention relates to host cells, particularly transgenic suspended carrot cells, vectors and methods for high yield expression and production of biologically active high mannose Glucocerebrosidase (GCD). The invention further provides for compositions and methods for the treatment of lysosomal storage diseases.
摘要翻译： 用于在植物培养物中生产糖基化蛋白质的装置，系统和方法，特别是具有高甘露糖糖基化的蛋白质，同时用ER信号靶向这种蛋白质和/或旁路高尔基体。 本发明进一步涉及使用转基因植物根特别是胡萝卜细胞表达和产生酶活性高甘露糖溶酶体酶的载体和方法。 更具体地，本发明涉及宿主细胞，特别是转基因悬浮的胡萝卜细胞，用于高产量表达和生产生物活性高甘露糖葡糖脑苷脂酶（GCD）的载体和方法。 本发明还提供了用于治疗溶酶体贮积病的组合物和方法。
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公开(公告)号：US20090208477A1
公开(公告)日：2009-08-20
申请号：US12385894
申请日：2009-04-23
CPC分类号： A61K38/47 , A61K9/0053 , A61K36/23 , C07K2319/04 , C12N9/2405 , C12N9/2434 , C12N9/2465 , C12N15/8257 , C12P21/005 , C12Y302/01022 , C12Y302/01045
摘要： A device, system and method for producing glycosylated proteins in plant culture, particularly proteins having a high mannose glycosylation, while targeting such proteins with an ER signal and/or by-passing the Golgi. The invention further relates to vectors and methods for expression and production of enzymatically active high mannose lysosomal enzymes using transgenic plant root, particularly carrot cells. More particularly, the invention relates to host cells, particularly transgenic suspended carrot cells, vectors and methods for high yield expression and production of biologically active high mannose Glucocerebrosidase (GCD). The invention further provides for compositions and methods for the treatment of lysosomal storage diseases.
摘要翻译： 用于在植物培养物中生产糖基化蛋白质的装置，系统和方法，特别是具有高甘露糖糖基化的蛋白质，同时用ER信号靶向这种蛋白质和/或旁路高尔基体。 本发明进一步涉及使用转基因植物根特别是胡萝卜细胞表达和产生酶活性高甘露糖溶酶体酶的载体和方法。 更具体地，本发明涉及宿主细胞，特别是转基因悬浮的胡萝卜细胞，用于高产量表达和生产生物活性高甘露糖葡糖脑苷脂酶（GCD）的载体和方法。 本发明还提供了用于治疗溶酶体贮积病的组合物和方法。
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公开(公告)号：US08741620B2
公开(公告)日：2014-06-03
申请号：US13555243
申请日：2012-07-23
CPC分类号： A61K38/47 , A61K9/0053 , A61K36/23 , C07K2319/04 , C12N9/2405 , C12N9/2434 , C12N9/2465 , C12N15/8257 , C12P21/005 , C12Y302/01022 , C12Y302/01045
摘要： A device, system and method for producing glycosylated proteins in plant culture, particularly proteins having a high mannose glycosylation, while targeting such proteins with an ER signal and/or by-passing the Golgi. The invention further relates to vectors and methods for expression and production of enzymatically active high mannose lysosomal enzymes using transgenic plant root, particularly carrot cells. More particularly, the invention relates to host cells, particularly transgenic suspended carrot cells, vectors and methods for high yield expression and production of biologically active high mannose Glucocerebrosidase (GCD). The invention further provides for compositions and methods for the treatment of lysosomal storage diseases.
摘要翻译： 用于在植物培养物中生产糖基化蛋白质的装置，系统和方法，特别是具有高甘露糖糖基化的蛋白质，同时用ER信号靶向这种蛋白质和/或旁路高尔基体。 本发明进一步涉及使用转基因植物根特别是胡萝卜细胞表达和产生酶活性高甘露糖溶酶体酶的载体和方法。 更具体地，本发明涉及宿主细胞，特别是转基因悬浮的胡萝卜细胞，用于高产量表达和生产生物活性高甘露糖葡糖脑苷脂酶（GCD）的载体和方法。 本发明还提供了用于治疗溶酶体贮积病的组合物和方法。
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9.发明授权公开(公告)号：US08119406B2
公开(公告)日：2012-02-21
申请号：US11665344
申请日：2005-10-11
申请人： Yoseph Shaaltiel , Sharon Hashmueli , Daniel Bartfeld , Gideon Baum , Tal Ratz , Einat Mizrachi , Yehava Forester
发明人： Yoseph Shaaltiel , Sharon Hashmueli , Daniel Bartfeld , Gideon Baum , Tal Ratz , Einat Mizrachi , Yehava Forester
CPC分类号： C12N15/8258 , C07K16/00 , C07K2317/13
摘要： A system and method for production of antibodies in plant cell culture, which results in highly functional antibodies, produced with a high level of expression efficiency. The present invention also encompasses host cells, vectors and methods for mass production of full size assembled immunoglobulins.
摘要翻译： 用于在植物细胞培养物中产生抗体的系统和方法，其产生具有高水平表达效率产生的高度功能的抗体。 本发明还包括用于批量生产全尺寸组装的免疫球蛋白的宿主细胞，载体和方法。
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公开(公告)号：US20080038232A1
公开(公告)日：2008-02-14
申请号：US11790991
申请日：2007-04-30
IPC分类号： A61K38/00 , A01N65/00 , A61P43/00 , C07H21/04 , C07K14/00 , C12N1/20 , C12N5/08 , C12N9/24 , C12P21/06
CPC分类号： A61K38/47 , A61K9/0053 , A61K36/23 , C07K2319/04 , C12N9/2405 , C12N9/2434 , C12N9/2465 , C12N15/8257 , C12P21/005 , C12Y302/01022 , C12Y302/01045
摘要： A device, system and method for producing glycosylated proteins in plant culture, particularly proteins having a high mannose glycosylation, while targeting such proteins with an ER signal and/or by-passing the Golgi. The invention further relates to vectors and methods for expression and production of enzymatically active high mannose lysosomal enzymes using transgenic plant root, particularly carrot cells. More particularly, the invention relates to host cells, particularly transgenic suspended carrot cells, vectors and methods for high yield expression and production of biologically active high mannose Glucocerebrosidase (GCD). The invention further provides for compositions and methods for the treatment of lysosomal storage diseases.
摘要翻译： 用于在植物培养物中生产糖基化蛋白质的装置，系统和方法，特别是具有高甘露糖糖基化的蛋白质，同时用ER信号靶向这种蛋白质和/或旁路高尔基体。 本发明进一步涉及使用转基因植物根特别是胡萝卜细胞表达和产生酶活性高甘露糖溶酶体酶的载体和方法。 更具体地，本发明涉及宿主细胞，特别是转基因悬浮的胡萝卜细胞，用于高产量表达和生产生物活性高甘露糖葡糖脑苷脂酶（GCD）的载体和方法。 本发明还提供了用于治疗溶酶体贮积病的组合物和方法。
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