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公开(公告)号：US20060104954A1
公开(公告)日：2006-05-18
申请号：US11329941
申请日：2006-01-11
申请人： Gregory Podsakoff , Gordon Watson , Linda Couto , Bin Yang
发明人： Gregory Podsakoff , Gordon Watson , Linda Couto , Bin Yang
IPC分类号： A61K48/00
CPC分类号： C12N15/86 , A61K48/00 , C12N9/2434 , C12N2750/14143 , C12N2830/008 , C12N2830/15 , C12N2830/42 , C12N2830/85 , C12N2840/44 , C12Y302/01031
摘要： AAV expression vectors and recombinant virions produced using these vectors, which include genes coding for enzymes defective or missing in lysosomal storage disorders, are described. These recombinant AAV virions are useful in the treatment of a variety of lysosomal storage disorders and the methods described herein provide for long-term, sustained expression of the defective or missing enzyme.
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2.发明申请Methods for delivering recombinant adeno-associated virus virions to the liver of a mammal 审中-公开将重组腺相关病毒颗粒递送至哺乳动物肝脏的方法公开(公告)号：US20060188482A1
公开(公告)日：2006-08-24
申请号：US11406867
申请日：2006-04-19
申请人： Mark Kay , Katherine High , Linda Couto
发明人： Mark Kay , Katherine High , Linda Couto
IPC分类号： A61K48/00 , C12N15/861
CPC分类号： C12N15/86 , A61K48/00 , C12N9/644 , C12N2750/14143 , C12Y304/21022
摘要： Methods for introducing recombinant adeno-associated virus (rAAV) virions into the liver of a mammal are provided. In these methods, the liver is partially or completely isolated from its blood supply, a catheter is introduced into the liver via a peripheral blood vessel, and rAAV virions are then infused through the catheter to the liver. The methods described herein may be used, for example, to deliver heterologous genes encoding therapeutic proteins to the hepatocytes of humans. This can be accomplished, for example, by introducing the catheter into a femoral artery, threading the catheter into the hepatic artery, and infusing rAAV virions through the catheter and into the liver. Exemplary examples of heterologous genes include those coding for blood coagulation factors.
摘要翻译： 提供了将重组腺相关病毒（rAAV）病毒粒子引入哺乳动物肝脏的方法。 在这些方法中，肝部分或完全与血液供应隔离，通过外周血管将导管引入肝脏，然后将rAAV病毒粒子通过导管输注到肝脏。 本文描述的方法可以用于例如将编码治疗性蛋白质的异源基因递送至人的肝细胞。 这可以通过例如将导管引入股动脉，将导管穿入肝动脉，并将rAAV病毒粒子通过导管并进入肝脏来实现。 异源基因的示例性实例包括编码凝血因子的那些。
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公开(公告)号：US20050276787A1
公开(公告)日：2005-12-15
申请号：US10632645
申请日：2003-08-01
申请人： Linda Couto , Peter Colosi , Xiaobing Qian
发明人： Linda Couto , Peter Colosi , Xiaobing Qian
IPC分类号： C12N15/09 , A61K35/76 , A61K38/27 , A61K38/37 , A61K38/43 , A61K48/00 , A61P7/04 , C07K14/755 , C12N5/10 , C12N15/861 , C12N15/864
CPC分类号： C12N15/86 , A61K38/37 , A61K48/00 , C07K14/755 , C12N2750/14143 , C12N2799/025 , C12N2830/008 , C12N2830/42 , C12N2830/85
摘要： One form of a composition has two types of recombinant adeno-associated virus. The first type encodes a portion of Factor VIII operably linked to an expression control element; and the second type encodes a different portion of Factor VIII operably linked to an expression control element. The first and second nucleotide sequences collectively encode a functional Factor VIII protein. Another form of the composition is a recombinant adeno-associated virus containing a nucleotide sequence encoding functional Factor VIII light or heavy chain operably linked to a tissue-specific promoter.
摘要翻译： 一种形式的组合物具有两种类型的重组腺相关病毒。 第一类型编码可操作地连接到表达控制元件的因子VIII的一部分; 并且第二类型编码可操作地连接到表达控制元件的因子VIII的不同部分。 第一和第二核苷酸序列共同编码功能性因子VIII蛋白。 该组合物的另一种形式是含有编码与组织特异性启动子可操作地连接的功能性因子VIII轻链或重链的核苷酸序列的重组腺相关病毒。
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公开(公告)号：US08993530B2
公开(公告)日：2015-03-31
申请号：US13293359
申请日：2011-11-10
IPC分类号： C12N15/113 , C12N15/11
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1131 , C12N2310/111 , C12N2310/14 , C12N2310/51 , C12N2310/53 , C12N2310/531 , C12N2330/30 , C12N2770/24211
摘要： The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.
摘要翻译： 本发明提供适用于在体外和体内表达1-x RNAi试剂以针对感兴趣的细胞，组织或器官中的基因或基因的组合物和方法，以便治疗疾病或病症。
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公开(公告)号：US08076471B2
公开(公告)日：2011-12-13
申请号：US11883645
申请日：2006-02-03
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1131 , C12N2310/111 , C12N2310/14 , C12N2310/51 , C12N2310/53 , C12N2310/531 , C12N2330/30 , C12N2770/24211
摘要： The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.
摘要翻译： 本发明提供适用于在体外和体内表达1-x RNAi试剂以针对感兴趣的细胞，组织或器官中的基因或基因的组合物和方法，以便治疗疾病或病症。
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公开(公告)号：US20060115454A1
公开(公告)日：2006-06-01
申请号：US11251076
申请日：2005-10-14
申请人： Linda Couto , Sarah Brashears , Sara Cunningham
发明人： Linda Couto , Sarah Brashears , Sara Cunningham
CPC分类号： A61L31/16 , A61K31/7105 , A61L27/54 , A61L2300/258 , A61L2300/416 , C12N15/113 , C12N15/1136 , C12N2310/14
摘要： The present invention provides compositions and methods suitable for delivering RNAi agents against genetic targets in vascular and adjacent tissue in vivo so as to treat restenosis.
摘要翻译： 本发明提供了适合于在体内在血管和相邻组织中递送RNAi剂以抵抗血管和相邻组织中的遗传靶点的组合物和方法，以便治疗再狭窄。
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公开(公告)号：US20120058555A1
公开(公告)日：2012-03-08
申请号：US13293359
申请日：2011-11-10
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1131 , C12N2310/111 , C12N2310/14 , C12N2310/51 , C12N2310/53 , C12N2310/531 , C12N2330/30 , C12N2770/24211
摘要： The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.
摘要翻译： 本发明提供适用于在体外和体内表达1-x RNAi试剂以针对感兴趣的细胞，组织或器官中的基因或基因的组合物和方法，以便治疗疾病或病症。
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公开(公告)号：US07803611B2
公开(公告)日：2010-09-28
申请号：US11347028
申请日：2006-02-03
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1131 , C12N2310/111 , C12N2310/14 , C12N2310/51 , C12N2310/53 , C12N2310/531 , C12N2330/30 , C12N2770/24211
摘要： The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.
摘要翻译： 本发明提供适用于在体外和体内表达1-x RNAi试剂以针对感兴趣的细胞，组织或器官中的基因或基因的组合物和方法，以便治疗疾病或病症。
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公开(公告)号：US20100028998A1
公开(公告)日：2010-02-04
申请号：US11883645
申请日：2006-02-03
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1131 , C12N2310/111 , C12N2310/14 , C12N2310/51 , C12N2310/53 , C12N2310/531 , C12N2330/30 , C12N2770/24211
摘要： The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.
摘要翻译： 本发明提供适用于在体外和体内表达1-x RNAi试剂以针对感兴趣的细胞，组织或器官中的基因或基因的组合物和方法，以便治疗疾病或病症。
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公开(公告)号：US20070025969A1
公开(公告)日：2007-02-01
申请号：US11347028
申请日：2006-02-03
申请人： Petrus Roelvink , David Suhy , Alexander Kolykhalov , Linda Couto
发明人： Petrus Roelvink , David Suhy , Alexander Kolykhalov , Linda Couto
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1131 , C12N2310/111 , C12N2310/14 , C12N2310/51 , C12N2310/53 , C12N2310/531 , C12N2330/30 , C12N2770/24211
摘要： The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.
摘要翻译： 本发明提供适用于在体外和体内表达1-x RNAi试剂以针对感兴趣的细胞，组织或器官中的基因或基因的组合物和方法，以便治疗疾病或病症。
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