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公开(公告)号:US20130123339A1
公开(公告)日:2013-05-16
申请号:US13697000
申请日:2011-05-12
申请人: James Heyes , Mark Wood , Alan Martin , Amy C.H. Lee , Adam Judge , Marjorie Robbins , Ian MacLachlan
发明人: James Heyes , Mark Wood , Alan Martin , Amy C.H. Lee , Adam Judge , Marjorie Robbins , Ian MacLachlan
IPC分类号: A61K31/713
CPC分类号: A61K31/713 , C12N15/111 , C12N15/113 , C12N15/88 , C12N2310/14 , C12N2320/32
摘要: The present invention provides compositions and methods for the delivery of interfering RNAs such as siRNAs that silence APOB expression in cells such as liver cells. In particular, the nucleic acid-lipid particles provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells such as liver cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of APOB at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
摘要翻译: 本发明提供用于递送干扰RNA的组合物和方法,例如在细胞如肝细胞中沉默APOB表达的siRNA。 特别地,核酸 - 脂质颗粒提供了核酸的有效包封,并且将体内核酸的活性有效地递送到细胞例如肝细胞。 本发明的组合物是非常有效的,从而允许以相对低的剂量有效地击倒APOB。 此外,与本领域已知的组合物和方法相比,本发明的组合物和方法毒性低,并提供更大的治疗指数。
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公开(公告)号:US08865675B2
公开(公告)日:2014-10-21
申请号:US13697000
申请日:2011-05-12
申请人: James Heyes , Mark Wood , Alan Martin , Amy C. H. Lee , Adam Judge , Marjorie Robbins , Ian MacLachlan
发明人: James Heyes , Mark Wood , Alan Martin , Amy C. H. Lee , Adam Judge , Marjorie Robbins , Ian MacLachlan
IPC分类号: C12N15/11 , C12Q1/68 , A61K31/713 , C12N15/88 , C12N15/113
CPC分类号: A61K31/713 , C12N15/111 , C12N15/113 , C12N15/88 , C12N2310/14 , C12N2320/32
摘要: The present invention provides compositions and methods for the delivery of interfering RNAs such as siRNAs that silence APOB expression in cells such as liver cells. In particular, the nucleic acid-lipid particles provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells such as liver cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of APOB at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
摘要翻译: 本发明提供用于递送干扰RNA的组合物和方法,例如在细胞如肝细胞中沉默APOB表达的siRNA。 特别地,核酸 - 脂质颗粒提供了核酸的有效包封,并且将体内核酸的活性有效地递送到细胞例如肝细胞。 本发明的组合物是非常有效的,从而允许以相对低的剂量有效地击倒APOB。 此外,与本领域已知的组合物和方法相比,本发明的组合物和方法毒性低,并提供更大的治疗指数。
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公开(公告)号:US20130064894A1
公开(公告)日:2013-03-14
申请号:US13599220
申请日:2012-08-30
申请人: Alan Martin , James Heyes , Mark Wood
发明人: Alan Martin , James Heyes , Mark Wood
IPC分类号: A61K48/00 , A61K47/28 , A61K9/14 , A61K47/22 , A61K31/713 , C07D319/06
CPC分类号: C07D319/06 , A61K9/127 , A61K9/1272 , A61K9/14 , A61K9/5123 , A61K31/713 , A61K47/22 , A61K47/28 , A61K48/0033 , B82Y5/00 , C12N15/88 , Y10S977/773 , Y10S977/906 , Y10T428/2982
摘要: The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
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公开(公告)号:US09126966B2
公开(公告)日:2015-09-08
申请号:US13599220
申请日:2012-08-30
申请人: Alan Martin , James Heyes , Mark Wood
发明人: Alan Martin , James Heyes , Mark Wood
IPC分类号: A61K9/14 , A61K9/127 , C07D319/06 , A61K47/28 , A61K47/22 , A61K31/713 , A61K9/51 , C12N15/88 , B82Y5/00 , A61K48/00
CPC分类号: C07D319/06 , A61K9/127 , A61K9/1272 , A61K9/14 , A61K9/5123 , A61K31/713 , A61K47/22 , A61K47/28 , A61K48/0033 , B82Y5/00 , C12N15/88 , Y10S977/773 , Y10S977/906 , Y10T428/2982
摘要: The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
摘要翻译: 本发明提供了将治疗剂递送至细胞的组合物和方法。 特别地,这些包括提供核酸的有效包封的新型阳离子脂质和核酸 - 脂质颗粒,并将包封的核酸在体内有效递送至细胞。 本发明的组合物是高度有效的,从而允许以相对低的剂量有效击倒特异性靶蛋白。 此外,与本领域已知的组合物和方法相比,本发明的组合物和方法毒性低,并提供更大的治疗指数。
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公开(公告)号:US08466122B2
公开(公告)日:2013-06-18
申请号:US13235253
申请日:2011-09-16
申请人: James Heyes , Mark Wood , Alan Martin
发明人: James Heyes , Mark Wood , Alan Martin
CPC分类号: A61K47/44 , A61K9/1272 , A61K31/7088 , A61K31/7105 , A61K31/713 , A61K47/34 , C07C229/12 , C07C271/20
摘要: The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
摘要翻译: 本发明提供了将治疗剂递送至细胞的组合物和方法。 特别地,这些包括提供核酸的有效包封的新型阳离子脂质和核酸 - 脂质颗粒,并将包封的核酸在体内有效递送至细胞。 本发明的组合物是高度有效的,从而允许以相对低的剂量有效击倒特异性靶蛋白。 此外,与本领域已知的组合物和方法相比,本发明的组合物和方法毒性低,并提供更大的治疗指数。
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公开(公告)号:US10077232B2
公开(公告)日:2018-09-18
申请号:US13696999
申请日:2011-05-12
申请人: James Heyes , Mark Wood , Alan Martin
发明人: James Heyes , Mark Wood , Alan Martin
IPC分类号: A61K47/18 , C07C217/28 , C07C229/12 , C07D317/28 , C12N15/11 , A61K9/127 , A61K9/51 , A61K31/7105 , A61K31/713
CPC分类号: C07C217/28 , A61K9/1272 , A61K9/5123 , A61K31/7105 , A61K31/713 , A61K47/18 , C07C229/12 , C07C2601/02 , C07D317/28 , C12N15/111 , C12N2310/14 , C12N2310/3515 , C12N2320/32
摘要: The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
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公开(公告)号:US20130123338A1
公开(公告)日:2013-05-16
申请号:US13696801
申请日:2011-05-12
申请人: James Heyes , Mark Wood , Alan Martin
发明人: James Heyes , Mark Wood , Alan Martin
IPC分类号: A61K48/00 , C07C217/08 , C07C271/20 , A61K47/14 , C07C237/06 , C07D233/60 , C07D249/04 , A61K47/18 , C07C229/12 , C07C327/22
CPC分类号: A61K47/18 , A61K31/713 , A61K47/14 , A61K47/183 , A61K47/186 , A61K47/22 , A61K48/0033 , C07C217/08 , C07C217/46 , C07C229/12 , C07C229/30 , C07C237/06 , C07C271/20 , C07C327/06 , C07C327/22 , C07D233/60 , C07D249/04 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2320/32
摘要: The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
摘要翻译: 本发明提供了将治疗剂递送至细胞的组合物和方法。 特别地,这些包括提供核酸的有效包封的新型阳离子脂质和核酸 - 脂质颗粒,并将包封的核酸在体内有效递送至细胞。 本发明的组合物是高度有效的,从而允许以相对低的剂量有效击倒特异性靶蛋白。 此外,与本领域已知的组合物和方法相比,本发明的组合物和方法毒性低,并提供更大的治疗指数。
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公开(公告)号:US20130116307A1
公开(公告)日:2013-05-09
申请号:US13696999
申请日:2011-05-12
申请人: James Heyes , Mark Wood , Alan Martin
发明人: James Heyes , Mark Wood , Alan Martin
IPC分类号: A61K48/00 , C07C229/06 , A61K38/17
CPC分类号: C07C217/28 , A61K9/1272 , A61K9/5123 , A61K31/7105 , A61K31/713 , A61K47/18 , C07C229/12 , C07C2601/02 , C07D317/28 , C12N15/111 , C12N2310/14 , C12N2310/3515 , C12N2320/32
摘要: The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
摘要翻译: 本发明提供了将治疗剂递送至细胞的组合物和方法。 特别地,这些包括提供核酸的有效包封的新型阳离子脂质和核酸 - 脂质颗粒,并将包封的核酸在体内有效递送至细胞。 本发明的组合物是高度有效的,从而允许以相对低的剂量有效击倒特异性靶蛋白。 此外,与本领域已知的组合物和方法相比,本发明的组合物和方法毒性低,并提供更大的治疗指数。
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公开(公告)号:US20120172411A1
公开(公告)日:2012-07-05
申请号:US13235253
申请日:2011-09-16
申请人: James Heyes , Mark Wood , Alan Martin
发明人: James Heyes , Mark Wood , Alan Martin
IPC分类号: A61K47/44 , C07C271/20 , C12N5/071 , A61K31/7105 , A61K31/713 , C07C229/12 , A61K31/7088
CPC分类号: A61K47/44 , A61K9/1272 , A61K31/7088 , A61K31/7105 , A61K31/713 , A61K47/34 , C07C229/12 , C07C271/20
摘要: The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
摘要翻译: 本发明提供了将治疗剂递送至细胞的组合物和方法。 特别地,这些包括提供核酸的有效包封的新型阳离子脂质和核酸 - 脂质颗粒,并将包封的核酸在体内有效递送至细胞。 本发明的组合物是高度有效的,从而允许以相对低的剂量有效击倒特异性靶蛋白。 此外,与本领域已知的组合物和方法相比,本发明的组合物和方法毒性低,并提供更大的治疗指数。
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公开(公告)号:US20130197490A1
公开(公告)日:2013-08-01
申请号:US13755299
申请日:2013-01-31
申请人: Larry Stanton , John Golden , Gary Kappel , Mark Wood , Bernadette Durr , Brandon Zalewski , Kenneth Keene , Barry Weitzner
发明人: Larry Stanton , John Golden , Gary Kappel , Mark Wood , Bernadette Durr , Brandon Zalewski , Kenneth Keene , Barry Weitzner
IPC分类号: A61B17/00
CPC分类号: A61B17/00 , A61B17/00234 , A61B2017/00323
摘要: This invention is directed to a medical device having an elongate shaft having a distal end and a controller coupled to the shaft. A control member can extend through at least part of the elongate shaft and at least part of the controller, wherein the control member in a tensioned configuration can be moveable by the controller to control the distal end of the shaft and the control member can be slack in an untensioned configuration. A tensioning mechanism can be configured to move relative to at least one of the elongate shaft and the controller to tension the control member and automatically lock the control member in the tensioned configuration.
摘要翻译: 本发明涉及一种具有细长轴的医疗装置,其具有远端和耦合到该轴的控制器。 控制构件可延伸穿过细长轴的至少一部分和控制器的至少一部分,其中处于张紧构型的控制构件可由控制器移动以控制轴的远端,并且控制构件可以松弛 在未张紧的配置中。 张紧机构可以被构造成相对于细长轴和控制器中的至少一个移动以张紧控制构件并且将控制构件自动地锁定在张紧构型中。
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