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公开(公告)号:US08617876B2
公开(公告)日:2013-12-31
申请号:US13539835
申请日:2012-07-02
申请人: Gwyneth Jane Farrar , Sophia Millington-Ward , Naomi Chadderton , Arpad Palfi , Mary O'Reilly , Paul Kenna , Peter Humphries
发明人: Gwyneth Jane Farrar , Sophia Millington-Ward , Naomi Chadderton , Arpad Palfi , Mary O'Reilly , Paul Kenna , Peter Humphries
CPC分类号: A61K48/0066 , A61K31/7105 , C12N15/86 , C12N2310/14 , C12N2750/14143
摘要: The invention relates to gene suppression and replacement. In particular, the invention relates to enhanced expression of suppression agents for suppressing gene expression in a cell and in vivo and replacement nucleic acids that are not inhibited by the suppression agent. Regulatory elements are included in expression vectors to optimize expression of the suppression agent and/or replacement nucleic acid.
摘要翻译: 本发明涉及基因抑制和替代。 特别地,本发明涉及用于抑制细胞和体内基因表达的抑制剂的增强表达和不被抑制剂抑制的替代性核酸。 表达载体中包含调节元件以优化抑制剂和/或置换核酸的表达。
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公开(公告)号:US20120278912A1
公开(公告)日:2012-11-01
申请号:US13539835
申请日:2012-07-02
申请人: Gwyneth Jane Farrar , Sophia Millington-Ward , Naomi Chadderton , Arpad Palfi , Mary O'Reilly , Paul Kenna , Peter Humphries
发明人: Gwyneth Jane Farrar , Sophia Millington-Ward , Naomi Chadderton , Arpad Palfi , Mary O'Reilly , Paul Kenna , Peter Humphries
IPC分类号: C12N15/85 , A61K31/7088 , A01K67/027 , C07H21/04 , C12N5/10
CPC分类号: A61K48/0066 , A61K31/7105 , C12N15/86 , C12N2310/14 , C12N2750/14143
摘要: The invention relates to gene suppression and replacement. In particular, the invention relates to enhanced expression of suppression agents for suppressing gene expression in a cell and in vivo and replacement nucleic acids that are not inhibited by the suppression agent. Regulatory elements are included in expression vectors to optimize expression of the suppression agent and/or replacement nucleic acid.
摘要翻译: 本发明涉及基因抑制和替代。 特别地,本发明涉及用于抑制细胞和体内基因表达的抑制剂的增强表达和不被抑制剂抑制的替代性核酸。 表达载体中包含调节元件以优化抑制剂和/或置换核酸的表达。
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公开(公告)号:US08257969B2
公开(公告)日:2012-09-04
申请号:US12595080
申请日:2008-04-14
申请人: Gwyneth Jane Farrar , Sophia Millington-Ward , Naomi Chadderton , Arpad Palfi , Mary O'Reilly , Paul Kenna , Peter Humphries
发明人: Gwyneth Jane Farrar , Sophia Millington-Ward , Naomi Chadderton , Arpad Palfi , Mary O'Reilly , Paul Kenna , Peter Humphries
CPC分类号: A61K48/0066 , A61K31/7105 , C12N15/86 , C12N2310/14 , C12N2750/14143
摘要: The invention relates to gene suppression and replacement. In particular, the invention relates to enhanced expression of suppression agents for suppressing gene expression in a cell and in vivo and replacement nucleic acids that are not inhibited by the suppression agent. Regulatory elements are included in expression vectors to optimize expression of the suppression agent and/or replacement nucleic acid.
摘要翻译: 本发明涉及基因抑制和替代。 特别地,本发明涉及用于抑制细胞和体内基因表达的抑制剂的增强表达和不被抑制剂抑制的替代性核酸。 表达载体中包含调节元件以优化抑制剂和/或置换核酸的表达。
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公开(公告)号:US20100190841A1
公开(公告)日:2010-07-29
申请号:US12595080
申请日:2008-04-14
申请人: Gwyneth Jane Farrar , Sophia Millington-Ward , Naomi Chadderton , Arpad Palfi , Mary O'Reilly , Paul Kenna , Peter Humphries
发明人: Gwyneth Jane Farrar , Sophia Millington-Ward , Naomi Chadderton , Arpad Palfi , Mary O'Reilly , Paul Kenna , Peter Humphries
IPC分类号: A61K31/7052 , C12N15/63 , C07H21/00 , C12N5/00 , A01K67/027 , A61P27/02
CPC分类号: A61K48/0066 , A61K31/7105 , C12N15/86 , C12N2310/14 , C12N2750/14143
摘要: The invention relates to gene suppression and replacement. In particular, the invention relates to enhanced expression of suppression agents for suppressing gene expression in a cell and in vivo and replacement nucleic acids that are not inhibited by the suppression agent. Regulatory elements are included in expression vectors to optimize expression of the suppression agent and/or replacement nucleic acid.
摘要翻译: 本发明涉及基因抑制和替代。 特别地,本发明涉及用于抑制细胞和体内基因表达的抑制剂的增强表达和不被抑制剂抑制的替代性核酸。 表达载体中包含调节元件以优化抑制剂和/或置换核酸的表达。
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公开(公告)号:US20090233368A1
公开(公告)日:2009-09-17
申请号:US12188304
申请日:2008-08-08
CPC分类号: C12N15/113 , A61K38/00 , A61K48/00 , C12N2310/111 , C12N2310/121 , C12N2310/15 , C12N2310/3181
摘要: A strategy for suppressing expression of one allele of an endogenous gene is provided comprising providing suppression effectors such as antisense nucleic acids able to bind to polymorphisms within or adjacent to a gene such that one allele of a gene is exclusively or preferentially suppressed and if required of a replacement gene can be introduced.The invention has the advantage that the same suppression strategy when directed to polymorphisms could be used to suppress, in principle, many mutations in a gene. This is particularly relevant when large numbers of mutations within a single gene cause disease pathology.
摘要翻译: 提供了抑制内源基因的一个等位基因表达的策略,其包括提供抑制效应子,例如能够结合基因内部或与基因相邻的多态性的反义核酸,使得基因的一个等位基因被单独或优先抑制, 可以引入替代基因。 本发明的优点在于,当针对多态性时,相同的抑制策略原则上可用于抑制基因中的许多突变。 当单个基因内的大量突变导致疾病病理学时,这尤其重要。
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公开(公告)号:US20090197336A1
公开(公告)日:2009-08-06
申请号:US12188405
申请日:2008-08-08
CPC分类号: C12N15/113 , A61K38/00 , A61K48/00 , A61K48/005 , C12N2310/111 , C12N2310/121 , C12N2310/15 , C12N2310/3181
摘要: Methods and agents for suppressing expression of a mutant allele of a gene having a polymorphism are provided. The methods of the invention provide suppression effectors such as antisense nucleic acids or ribozymes, that bind to nucleic acid regions having a polymorphism within a gene such that one allele of a gene is exclusively or preferentially suppressed. The method also provides the administration of a replacement nucleic acid, if required. The invention has the advantage that the same suppression strategy, when directed to polymorphisms, could be used to suppress, in principle, many mutations in a polymorphic gene. This is particularly relevant when large numbers of mutations within a single gene cause disease pathology.
摘要翻译: 提供了用于抑制具有多态性的基因的突变等位基因表达的方法和试剂。 本发明的方法提供诸如反义核酸或核酶之类的抑制效应物,其与基因内具有多态性的核酸区域结合,使得基因的一个等位基因被专门或优先地抑制。 如果需要,该方法还提供了置换核酸的施用。 本发明的优点在于,当针对多态性时,相同的抑制策略原则上可用于抑制多态性基因中的许多突变。 当单个基因内的大量突变导致疾病病理学时,这尤其重要。
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公开(公告)号:US20060128648A1
公开(公告)日:2006-06-15
申请号:US11224524
申请日:2005-09-12
申请人: Gwenyth Farrar , Peter Humphries , Paul Kenna
发明人: Gwenyth Farrar , Peter Humphries , Paul Kenna
IPC分类号: A61K48/00
CPC分类号: C12N15/113 , A61K38/00 , A61K48/00 , A61K48/005 , C12N2310/111 , C12N2310/121 , C12N2310/15 , C12N2310/3181
摘要: A strategy for suppressing specifically or partially specifically an endogenous gene and introducing a replacement gene, said strategy comprising the steps of: 1. providing suppressing nucleic acids or other suppression effectors able to bind to an endogenous gene, gene transcript or gene product to be suppressed and 2. providing genomic DNA or cDNA (complete or partial) encoding a replacement gene wherein the suppressing nucleic acids are unable to bind to equivalent regions in the genomic DNA or cDNA to prevent expression of the replacement gene. The replacement nucleic acids have modifications in one or more third base (wobble) positions such that replacement nucleic acids still code for the wild type or equivalent amino acids.
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公开(公告)号:US20110229439A1
公开(公告)日:2011-09-22
申请号:US13063604
申请日:2009-09-11
申请人: Peter Humphries , Marian Humphries , Matthew Campbell , Paul Kenna , Lawrence Chi Shing Tam , Gwenyth Jane Farrar , Anna-Sophia Kiang
发明人: Peter Humphries , Marian Humphries , Matthew Campbell , Paul Kenna , Lawrence Chi Shing Tam , Gwenyth Jane Farrar , Anna-Sophia Kiang
IPC分类号: A61K35/76 , C07K14/435 , A61K38/16 , G01N33/53 , A61P27/02
CPC分类号: A61K38/1725
摘要: The present invention relates to classical pathway complement proteins and their use in the prognosis and prevention of diseases involving cone photoreceptor degeneration. Specifically, the present invention is directed to the use of one or more classical pathway complement proteins, preferably involved in the recognition phase, in the maintenance of cone photoreceptor cell viability in a degenerating retina. The invention is also directed to a method for determining the susceptibility, risk of development and/or progression of diseases involving cone photoreceptor degeneration in a subject.
摘要翻译: 本发明涉及经典途径补体蛋白及其在预后和预防涉及锥体感光器变性的疾病中的应用。 具体而言,本发明涉及一种或多种经典途径补体蛋白(优选参与识别阶段)在维持视网膜退行性视网膜中的锥体感光细胞活力的用途。 本发明还涉及用于确定受试者中涉及锥体感光器变性的疾病的易感性,发展风险和/或进展的方法。
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公开(公告)号:US4356474A
公开(公告)日:1982-10-26
申请号:US241268
申请日:1981-03-06
申请人: Peter Humphries , Paul Aeberli
发明人: Peter Humphries , Paul Aeberli
摘要: A digital alarm system of the type having first and second electrically conductive loops connected by an alarm switch. The alarm switch is normally open, but can be closed when an alarm state exists to electrically short one loop to the other. Currents are generated in each of the loops for use in detecting either the closing of the alarm switch or the occurrence of a fault in one of the loops. Resistors are connected to each of the loops for use in detecting the presence or absence of the currents. A first pair of transistor switches alternatingly short-circuit one resistor then the other; and a second pair of transistor switches, synchronized with the first pair, alternatingly short the ends of one loop then those of the other so that when the alarm switch is closed the first pair of transistor switches simultaneously short-circuit both resistors even if a fault occurs anywhere in one of the loops. Recording devices, such as flip-flops, coupled to the resistors, record signals indicative of a magnitude of the resistor voltage drops during intervals in which the resistors are not short-circuited. Logic circuitry is then used to process this stored information to generate signals indicating whether the alarm switch has been tripped or whether a fault has occurred.
摘要翻译: 一种具有通过报警开关连接的第一和第二导电回路的数字报警系统。 报警开关是常开的,但是当有一个报警状态存在电一个回路到另一个回路时可以关闭。 在每个回路中产生电流,用于检测报警开关的关闭或其中一个回路中发生故障。 电阻器连接到每个回路以用于检测电流的存在或不存在。 第一对晶体管开关交替地使一个电阻器短路另一个; 以及与第一对同步的第二对晶体管开关交替地将一个环路的端部交替地短路,使得当报警开关闭合时,第一对晶体管开关同时使两个电阻器短路,即使是故障 发生在其中一个循环中的任何位置。 耦合到电阻器的诸如触发器的记录装置在电阻器不短路的间隔期间记录指示电阻器电压下降幅度的信号。 然后使用逻辑电路来处理该存储的信息以产生指示报警开关是否已跳闸或是否发生故障的信号。
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公开(公告)号:US20110064792A1
公开(公告)日:2011-03-17
申请号:US12682331
申请日:2008-10-13
IPC分类号: A61K31/7088 , A61K9/127 , A61K38/00 , A61K38/43 , A61P25/28 , A61P25/00 , A61P35/00 , A61P27/02 , C12N5/071
CPC分类号: C12N15/1138 , A61K31/713 , A61K45/06 , C12N15/111 , C12N2310/14 , C12N2320/31 , C12N2320/32
摘要: The present invention is directed to a method and use of RNA interference (RNAi) for the transient, reversible and controlled opening of the tight junctions of the blood brain barrier and/or the blood retinal barrier. This method may be used in the treatment of many diseases and disorders which require the opening of the blood brain barrier and/or blood retinal barrier. Such methods generally involve the use of an RNAi-inducing agent, such as siRNA, miRNA, shRNA or an RNAi-inducing vector whose presence within a cell results in production of an siRNA or shRNA, targeting tight junction proteins to open the blood brain barrier and/or blood retinal barrier.
摘要翻译: 本发明涉及RNA干扰(RNAi)用于血脑屏障和/或血液视网膜屏障的紧密连接的瞬时,可逆和受控打开的方法和用途。 该方法可用于治疗需要开启血脑屏障和/或血液视网膜屏障的许多疾病和病症。 这样的方法通常涉及使用RNAi诱导剂,例如siRNA,miRNA,shRNA或RNAi诱导载体,其在细胞内的存在导致产生siRNA或shRNA,靶向紧密连接蛋白以打开血脑屏障 和/或血液视网膜屏障。
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