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21. 发明申请

US20140234361A1 NOVEL EXPRESSION VECTORS AND USES THEREOF 有权
标题翻译：新兴表达载体及其用途
公开(公告)号：US20140234361A1
公开(公告)日：2014-08-21
申请号：US14153576
申请日：2014-01-13
申请人： FIT Biotech Oy
发明人： Kai KROHN , Vesna Blazevic , Marja Tähtinen , Mart Ustav , Urve Toots , Andres Männik , Annamari Ranki , Ene Ustav
IPC分类号： C07K14/005 , C12N15/85
CPC分类号： C07K14/005 , A61K48/00 , A61K2039/53 , C12N15/85 , C12N2800/108 , C12N2830/42 , C12N2840/20 , C12N2840/203
摘要： A method for treating an HIV disease in a subject in need of said treatment, comprising administering to the subject a therapeutically effective amount of a DNA vaccine comprising an expression vector and a pharmaceutically acceptable excipient, where the expression vector comprises: (a) a heterologous promoter operatively linked to a DNA sequence encoding a nuclear-anchoring protein, where the nuclear-anchoring protein comprises: (i) a DNA binding domain which binds to a specific DNA binding sequence, and (ii) a functional domain of the Bovine Papilloma Virus Type 1 E2 protein, where the functional domain binds to a nuclear component; (b) a multimerized DNA sequence that forms a binding site for the nuclear anchoring protein; and (c) at least one expression cassette comprising a DNA sequence encoding a protein or peptide that stimulates an immune response specific to the protein or peptide; where the expression vector lacks an origin of replication functional in mammalian cells.
摘要翻译：一种用于治疗需要所述治疗的受试者中的HIV疾病的方法，包括给予受试者治疗有效量的包含表达载体和药学上可接受的赋形剂的DNA疫苗，其中所述表达载体包含：（a）异源启动子与编码核锚定蛋白的DNA序列可操作地连接，其中核锚定蛋白包含：（i）结合特异性DNA结合序列的DNA结合结构域，和（ii）牛乳头状瘤病毒的功能结构域 1型E2蛋白，其中功能结构域结合核成分; （b）形成核锚定蛋白的结合位点的多聚化DNA序列; 和（c）至少一个表达盒，其包含编码蛋白质或肽的DNA序列，所述DNA序列刺激对所述蛋白质或肽特异性的免疫应答; 其中表达载体缺乏在哺乳动物细胞中起作用的复制起点。

22. 发明申请

US20140220060A1 HIV-1 SOUTH AFRICAN SUBTYPE C ENV PROTEINS 有权
标题翻译： HIV-1南非非洲C型ENV蛋白
公开(公告)号：US20140220060A1
公开(公告)日：2014-08-07
申请号：US14141396
申请日：2013-12-26
申请人： Novartis Vaccines & Diagnostics, Inc.
发明人： Jan ZUR MEGEDE , Susan BARNETT , Ying LIAN , Susan ENGELBRECHT , Estrelita Janse VAN RENSBERG , Thomas J. SCRIBA
IPC分类号： C07K14/005
CPC分类号： C07K14/005 , A61K39/00 , A61K39/12 , A61K39/21 , A61K2039/53 , C12N15/86 , C12N2720/00034 , C12N2740/16043 , C12N2740/16052 , C12N2740/16122 , C12N2740/16222 , C12N2740/16322 , C12N2799/027 , C12N2800/108 , C12N2830/42 , C12N2840/203
摘要： The present invention relates to polynucleotides encoding immunogenic HIV polypeptides. Uses of the polypeptides in applications including immunization, generation of packaging cell lines, and production of HIV polypeptides are also described. Polynucleotides encoding antigenic HIV polypeptides are described, as are uses of these polynucleotides and polypeptide products therefrom, including formulations of immunogenic compositions and uses thereof.
摘要翻译：本发明涉及编码免疫原性HIV多肽的多核苷酸。还描述了多肽在包括免疫，产生包装细胞系以及产生HIV多肽的应用中的用途。描述了编码抗原性HIV多肽的多核苷酸，以及来自其的这些多核苷酸和多肽产物的用途，包括免疫原性组合物的制剂及其用途。

23. 发明申请

US20140170123A1 Hepatocyte Based Insulin Gene Therapy For Diabetes 有权
标题翻译：基于肝细胞的胰岛素基因治疗糖尿病
公开(公告)号：US20140170123A1
公开(公告)日：2014-06-19
申请号：US14133016
申请日：2013-12-18
申请人： Wisconsin Alumni Research Foundation
发明人： Tausif Alam , Hans Sollinger
IPC分类号： C12N15/85 , A61K35/407
CPC分类号： C12N15/85 , A61K35/407 , A61K38/00 , C07K14/62 , C12N15/86 , C12N2710/10042 , C12N2830/002 , C12N2830/15 , C12N2830/42 , C12N2840/105
摘要： A method and vectors for controlling blood glucose levels in a mammal are disclosed. In one embodiment, the method comprises the steps of: treating the hepatocyte cells of a patient with a first, second or third vector, wherein the first vector comprises a promoter enhancer, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase and an albumin 3′UTR and lacks an HGH intron, wherein the second vector comprises an HGH intron, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase site and an albumin 3′UTR and lacks a promoter enhancer, wherein the third vector comprises an HGH intron, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase site, an albumin 3′UTR and a promoter enhancer and observing the patient's insulin levels, wherein the patient's insulin levels are controlled.
摘要翻译：公开了一种用于控制哺乳动物血糖水平的方法和载体。在一个实施方案中，所述方法包括以下步骤：用第一，第二或第三载体处理患者的肝细胞细胞，其中所述第一载体包含启动子增强子，葡萄糖诱导型调节元件，肝特异性启动子，编码具有修饰的肽酶和白蛋白3'UTR的人胰岛素和缺乏HGH内含子，其中第二载体包含HGH内含子，葡萄糖诱导型调节元件，肝特异性启动子，编码具有修饰的肽酶位点的人胰岛素的基因和白蛋白3 'UTR并且缺乏启动子增强子，其中第三载体包含HGH内含子，葡萄糖诱导型调节元件，肝特异性启动子，编码具有修饰的肽酶位点的人胰岛素的基因，白蛋白3'UTR和启动子增强子，并观察患者的胰岛素水平，其中患者的胰岛素水平被控制。

24. 发明申请

US20140127162A1 REVERSIBLE GENE EXPRESSION 有权
标题翻译：可逆基因表达
公开(公告)号：US20140127162A1
公开(公告)日：2014-05-08
申请号：US14067786
申请日：2013-10-30
申请人： California Institute of Technology
发明人： Alejandro Benjamin Balazs , David Baltimore
IPC分类号： A61K48/00 , C12N15/86
CPC分类号： A61K48/0066 , C12N15/86 , C12N2750/14143 , C12N2800/30 , C12N2800/40 , C12N2830/001 , C12N2830/42 , C12N2840/44
摘要： According to some embodiments herein, expression systems and methods for reversible gene expression are provided. In some embodiments, adeno-associated viral vectors encoding a gene product of interest and comprising a plurality of recombinase target sites are provided. In some embodiments, a source of recombinase is provided. In some embodiments, the gene product of interest is expressed, and the recombinase then induces recombination events between the recombinase target sites, thus reducing or eliminating expression of the gene product of interest.
摘要翻译：根据本文的一些实施方案，提供了可逆基因表达的表达系统和方法。在一些实施方案中，提供编码感兴趣的基因产物并且包含多个重组酶靶位点的腺相关病毒载体。在一些实施方案中，提供了重组酶的来源。在一些实施方案中，表达目的基因产物，然后重组酶诱导重组酶靶位点之间的重组事件，从而减少或消除感兴趣的基因产物的表达。

25. 发明申请

US20120277285A1 CONTROL OF GENE EXPRESSION 审中-公开
标题翻译：基因表达控制
公开(公告)号：US20120277285A1
公开(公告)日：2012-11-01
申请号：US13458704
申请日：2012-04-27
申请人： Michael Wayne Graham , Robert Norman Rice
发明人： Michael Wayne Graham , Robert Norman Rice
IPC分类号： A61K31/7088 , C12N15/85 , C07H21/04 , C12N5/07
CPC分类号： C12N15/113 , A01K2217/05 , A61K48/00 , C12N9/1051 , C12N9/127 , C12N9/503 , C12N15/111 , C12N15/1131 , C12N15/63 , C12N15/69 , C12N15/8216 , C12N15/8218 , C12N15/8283 , C12N15/85 , C12N2310/111 , C12N2310/14 , C12N2310/531 , C12N2320/10 , C12N2320/30 , C12N2330/30 , C12N2330/50 , C12N2330/51 , C12N2800/108 , C12N2830/00 , C12N2830/002 , C12N2830/15 , C12N2830/38 , C12N2830/42 , C12N2830/55 , C12N2830/60 , C12N2840/20
摘要： The present invention relates generally to a method of modifying gene expression and to synthetic genes for modifying endogenous gene expression in a cell, tissue or organ of a transgenic organism, in particular a transgenic animal or plant. More particularly, the present invention utilizes recombinant DNA technology to post-transcriptionally modify or modulate the expression of a target gene in a cell, tissue organ or whole organism, thereby producing novel phenotypes. Novel synthetic genes and genetic constructs which are capable of repressing delaying or otherwise reducing the expression of an endogenous gene or target gene in an organism when introduced thereto are also provided.
摘要翻译：本发明一般涉及修饰基因表达的方法和用于修饰转基因生物体，特别是转基因动物或植物的细胞，组织或器官中的内源基因表达的合成基因。更具体地，本发明利用重组DNA技术转录后修饰或调节细胞，组织器官或整个生物体中靶基因的表达，从而产生新的表型。还提供了新的合成基因和遗传构建体，其能够抑制在引入生物体时延迟或以其它方式减少生物体中的内源基因或靶基因的表达。

26. 发明申请

US20120252882A1 CARDIAC-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF 有权
标题翻译： CARDIAC特异性核酸调节元素及其使用方法
公开(公告)号：US20120252882A1
公开(公告)日：2012-10-04
申请号：US13504614
申请日：2010-10-29
申请人： Lay Khim Chuah , Thierry Vandendriessche , Pieter De Bleser
发明人： Lay Khim Chuah , Thierry Vandendriessche , Pieter De Bleser
IPC分类号： A61K48/00 , A61P9/00 , C12N15/867 , C12N15/113 , C12N15/85 , C12N15/864
CPC分类号： C07K14/4702 , A61K48/00 , C07K14/4716 , C07K14/805 , C12N15/85 , C12N2799/025 , C12N2830/008 , C12N2830/15 , C12N2830/30 , C12N2830/42 , C12N2830/85
摘要： The present invention relates to nucleic acid regulatory elements that are able to enhance cardiac-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy.
摘要翻译：本发明涉及能够增强基因的心脏特异性表达的核酸调节元件，采用这些调控元件的方法和这些元件的用途。还公开了含有这些核酸调节元件的表达盒和载体。本发明对于使用基因治疗的应用特别有用。

27. 发明授权

US08114980B2 Characterisation of gene function using double stranded RNA inhibition 有权
标题翻译：使用双链RNA抑制表征基因功能
公开(公告)号：US08114980B2
公开(公告)日：2012-02-14
申请号：US10826522
申请日：2004-04-16
申请人： Geert Plaetinck , Christ Platteeuw , Katharine Mortier , Thierry Bogaert
发明人： Geert Plaetinck , Christ Platteeuw , Katharine Mortier , Thierry Bogaert
IPC分类号： C07H21/04 , C12N15/11 , G01N33/569 , C12Q1/68
CPC分类号： C12N15/1079 , C12N15/1093 , C12N15/8216 , C12N15/8218 , C12N15/8279 , C12N15/8285 , C12N15/86 , C12N2795/10243 , C12N2800/40 , C12N2830/002 , C12N2830/008 , C12N2830/42 , C12Q1/68 , Y02A40/164
摘要： There is provided a method of identifying DNA responsible for conferring a particular phenotype in a cell which method comprises a) constructing a cDNA or genomic library of the DNA of the cell in a suitable vector in an orientation relative to a promoter(s) capable of initiating transcription of the cDNA or DNA to double stranded (ds) RNA upon binding of an appropriate transcription factor to the promoter(s), b) introducing the library into one or more of the cells comprising the transcription factor, and c) identifying and isolating a particular phenotype of the cell comprising the library and identifying the DNA or cDNA fragment from the library responsible for conferring the phenotype. Using this technique it is also possible to assign function to a known DNA sequence by a) identifying a homologue(s) of the DNA sequence in a cell, b) isolating the relevant DNA homologue(s) or a fragment thereof from the cell, c) cloning the homologue or fragment thereof into an appropriate vector in an orientation relative to a suitable promoter(s) capable of initiating transcription of dsRNA from the DNA homologue or fragment upon binding of an appropriate transcription factor to the promoter(s) and d) introducing the vector into the cell from step a) comprising the transcription factor.
摘要翻译：提供了鉴定负责赋予细胞中特定表型的DNA的方法，该方法包括a）在合适的载体中构建相对于能够启动子的启动子的方向的细胞DNA的cDNA或基因组文库在将合适的转录因子与启动子结合时，启动cDNA或DNA转录为双链（ds）RNA; b）将文库引入至一个或多个包含转录因子的细胞中，c）鉴定和分离包含文库的细胞的特定表型，并从负责表型的文库鉴定DNA或cDNA片段。使用这种技术还可以通过以下方式将功能分配给已知的DNA序列：a）鉴定细胞中DNA序列的同系物，b）从细胞中分离相关的DNA同系物或其片段， c）相对于合适的启动子（其能够在合适的转录因子与启动子结合时能够启动dsDNA从DNA同系物或片段的转录）的方向，将其同源物或片段克隆到合适的载体中，并且d ）将载体从包含转录因子的步骤a）引入细胞。

28. 发明授权

US08076100B2 Molecular clones with mutated HIV gag/pol, SIV gag and SIV env genes 有权
标题翻译：具有突变的HIV gag / pol，SIV gag和SIV env基因的分子克隆
公开(公告)号：US08076100B2
公开(公告)日：2011-12-13
申请号：US10263020
申请日：2002-10-02
申请人： George N. Pavlakis
发明人： George N. Pavlakis
IPC分类号： C12P21/06 , C12N15/63
CPC分类号： C07K14/005 , A61K39/00 , A61K39/12 , A61K39/21 , A61K48/00 , A61K2039/5256 , A61K2039/53 , C12N7/00 , C12N15/86 , C12N2740/15022 , C12N2740/15034 , C12N2740/16021 , C12N2740/16043 , C12N2740/16122 , C12N2740/16234 , C12N2830/42 , C12N2830/48 , C12N2830/50 , C12N2840/107 , C12N2840/20
摘要： Nucleic acid constructs containing HIV-1 gag/pol and SIV gag or SIV env genes which have been mutated to remove or reduce inhibitory/instability sequences are disclosed. Viral particles and host cells containing these constructs and/or viral particles are also disclosed. The exemplified constructs and viral particles of the invention may be useful in gene therapy for numerous disorders, including HIV infection, or as a vaccine for HIV-1 immunotherapy and immunoprophylaxis.
摘要翻译：公开了含有突变以除去或减少抑制/不稳定序列的HIV-1 gag / pol和SIV gag或SIV env基因的核酸构建体。还公开了含有这些构建体和/或病毒颗粒的病毒颗粒和宿主细胞。本发明的示例性构建体和病毒颗粒可用于许多病症（包括HIV感染）的基因治疗或HIV-1免疫治疗和免疫预防的疫苗。

29. 发明授权

US08067383B2 Synthetic genes and genetic constructs comprising same I 有权
标题翻译：合成基因和包含I的遗传构建体
公开(公告)号：US08067383B2
公开(公告)日：2011-11-29
申请号：US10346853
申请日：2003-01-17
申请人： Michael Wayne Graham , Robert Norman Rice
发明人： Michael Wayne Graham , Robert Norman Rice
IPC分类号： A01N43/04 , A61K31/70
CPC分类号： C12N15/85 , C12N9/0071 , C12N9/1051 , C12N9/127 , C12N9/503 , C12N15/113 , C12N15/63 , C12N15/69 , C12N15/8216 , C12N15/8218 , C12N15/8283 , C12N2800/108 , C12N2830/00 , C12N2830/002 , C12N2830/15 , C12N2830/38 , C12N2830/42 , C12N2830/55 , C12N2830/60 , C12N2840/102 , C12N2840/20
摘要： The present invention relates generally to synthetic genes for modifying endogenous gene expression in a cell, tissue or organ of a transgenic organism, in particular a transgenic animal or plant. More particularly, the present invention provides novel synthetic genes and genetic constructs which are capable of repressing delaying or otherwise reducing the expression of an endogenous gene or a target gene in an organism when introduced thereto.
摘要翻译：本发明一般涉及用于修饰转基因生物体，特别是转基因动物或植物的细胞，组织或器官中的内源基因表达的合成基因。更具体地说，本发明提供了新的合成基因和遗传构建体，其能够在引入生物体时抑制延迟或以其他方式降低生物体中的内源基因或靶基因的表达。

30. 发明授权

US07932087B2 Method of expressing recombinant protein in CHO cells 有权
标题翻译：在CHO细胞中表达重组蛋白的方法
公开(公告)号：US07932087B2
公开(公告)日：2011-04-26
申请号：US11962261
申请日：2007-12-21
申请人： Robert Kallmeier , Robert Gay
发明人： Robert Kallmeier , Robert Gay
IPC分类号： C12N15/85 , C07H21/04
CPC分类号： C12N15/907 , C12N2800/108 , C12N2830/42 , C12N2840/20
摘要： Method of expressing recombinant protein in CHO cells, by using an expression vector comprising the murine IgG 2A gene locus.
摘要翻译：通过使用包含鼠IgG2A基因座的表达载体在CHO细胞中表达重组蛋白的方法。

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