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1. 发明授权

US06808923B2 Episomally replicating lentiviral vectors 有权
标题翻译： Episomally复制慢病毒载体
公开(公告)号：US06808923B2
公开(公告)日：2004-10-26
申请号：US09990879
申请日：2001-11-21
申请人： Alan Engelman , Wolfgang Hofmann , Joseph G. Sodroski , Richard Lu
发明人： Alan Engelman , Wolfgang Hofmann , Joseph G. Sodroski , Richard Lu
IPC分类号： C12N15867
CPC分类号： C12N15/86 , A61K48/00 , C12N2740/16043 , C12N2740/16052 , C12N2800/108
摘要： The invention is directed to a lentiviral vector system that can be used for episomal replication of a desired gene. The vector system contains a first vector containing a lentiviral gag gene encoding a lentiviral gag protein, a second vector containing an env gene encoding a functional envelope protein, and a lentiviral pol gene encoding a lentiviral pol protein. The pol protein is at least integrase, but that the integrase has been modified so that it is not capable of integration. This pol gene can be on the first or second vectors or on a third vector. The lentiviral gag and pol and the envelope protein are not on a single vector, and these vectors do not contain nucleotides to effectively package lentiviral RNA. The system also contains another vector having a nucleic acid sequence encoding a target molecule operably linked to a component of an episomal replicon and a lentiviral packaging sequence.
摘要翻译：本发明涉及可用于所需基因的附加型复制的慢病毒载体系统。载体系统含有含有编码慢病毒gag蛋白的慢病毒gag基因的第一载体，含有编码功能性包膜蛋白的env基因的第二载体和编码慢病毒pol蛋白的慢病毒pol基因。 pol蛋白至少是整合酶，但是整合酶已经被修饰，使得它不能整合。该pol基因可以在第一或第二载体上或第三载体上。慢病毒gag和pol和包膜蛋白不在单个载体上，并且这些载体不含有有效包装慢病毒RNA的核苷酸。该系统还含有另一载体，其具有编码与游离复制子的组分和慢病毒包装序列可操作地连接的靶分子的核酸序列。

2. 发明授权

US06521457B2 Lentivirus-based gene transfer vectors 有权
标题翻译：基于慢病毒的基因转移载体
公开(公告)号：US06521457B2
公开(公告)日：2003-02-18
申请号：US09900419
申请日：2001-07-06
申请人： John C. Olsen
发明人： John C. Olsen
IPC分类号： C12N15867
CPC分类号： C12N15/86 , A61K48/00 , C12N7/00 , C12N2740/15043 , C12N2740/15052
摘要： A recombinant lentiviral vector expression system comprises a first vector that comprises a nucleic acid sequence of at least part of the Equine Infectious Anemia Virus (EIAV) genome. The vector contains at least one defect in at least one gene encoding an EIAV structural protein, but is preferably a gag/pol expression vector. The expression system further comprises a second vector, also comprising a nucleic acid sequence of at least part of the Equine Infectious Anemia Virus (EIAV) genome, and additionally containing a multiple cloning site wherein a heterologous gene may be inserted. The expression system also comprises a third vector which expresses a viral envelope protein. The first and third vectors are packaging signal-defective. When the expression system is transfected into a lentivirus-permissive cell, replication-defective EIAV particles may be produced, which particles are useful in delivering heterologous genes to a broad range of both dividing and non-dividing cells.
摘要翻译：重组慢病毒载体表达系统包含第一载体，其包含至少部分马传染性贫血病毒（EIAV）基因组的核酸序列。该载体在编码EIAV结构蛋白的至少一个基因中含有至少一个缺陷，但优选为gag / pol表达载体。表达系统还包含第二载体，其还包含至少部分马传染性贫血病毒（EIAV）基因组的核酸序列，并另外含有可以插入异源基因的多克隆位点。表达系统还包含表达病毒包膜蛋白的第三载体。第一和第三载体是包装信号有缺陷的。当将表达系统转染入慢病毒允许细胞时，可以产生复制缺陷型EIAV颗粒，该颗粒可用于将异源基因递送到分裂细胞和非分裂细胞的广泛范围。

3. 发明授权

US06495349B1 Chimeric gene constructs 失效
标题翻译：嵌合基因构建体
公开(公告)号：US06495349B1
公开(公告)日：2002-12-17
申请号：US08462512
申请日：1995-06-05
申请人： Harry E. Gruber , Douglas J. Jolly , James G. Respess , Paul K. Laikind
发明人： Harry E. Gruber , Douglas J. Jolly , James G. Respess , Paul K. Laikind
IPC分类号： C12N15867
CPC分类号： A01K67/0275 , A01K2217/05 , A61K39/0011 , A61K39/12 , A61K39/21 , A61K48/00 , A61K2039/5256 , A61K2039/53 , A61K2039/57 , C07K14/005 , C07K14/70514 , C12N7/00 , C12N9/1211 , C12N15/86 , C12N2740/13022 , C12N2740/13023 , C12N2740/13032 , C12N2740/13043 , C12N2740/13052 , C12N2740/15022 , C12N2740/16023 , C12N2740/16134 , C12N2740/16222
摘要： Recombinant retroviruses carrying a vector construct capable of preventing, inhibiting, stabilizing or reversing infectious, cancerous or auto-immune diseases are disclosed. More specifically, the recombinant retroviruses of the present invention are useful for (a) stimulating a specific immune response to an antigen or a pathogenic antigen; (b) inhibiting a function of a pathogenic agent, such as a virus; and (c) inhibiting the interaction of an agent with a host cell receptor. In addition, eucaryotic cells infected with, and pharmaceutical compositions containing such a recombinant retrovirus are disclosed. Various methods for producing recombinant retroviruses having unique characteristics, and methods for producing transgenic packaging animals or insects are also disclosed.
摘要翻译：公开了携带能够预防，抑制，稳定或逆转感染性，癌性或自身免疫性疾病的载体构建体的重组逆转录病毒。更具体地，本发明的重组逆转录病毒可用于（a）刺激对抗原或病原性抗原的特异性免疫应答; （b）抑制病原体如病毒的功能; 和（c）抑制药剂与宿主细胞受体的相互作用。此外，公开了感染的真核细胞和含有这种重组逆转录病毒的药物组合物。还公开了用于生产具有独特特征的重组逆转录病毒的各种方法，以及用于生产转基因包装动物或昆虫的方法。

4. 发明授权

US06790657B1 Lentivirus vector system 失效
标题翻译：慢病毒载体系统
公开(公告)号：US06790657B1
公开(公告)日：2004-09-14
申请号：US09869588
申请日：2001-06-28
申请人： Suresh K. Arya
发明人： Suresh K. Arya
IPC分类号： C12N15867
CPC分类号： C12N15/86 , C12N2740/15043 , C12N2740/15052 , C12N2740/16043 , C12N2740/16052 , C12N2840/20 , C12N2840/203
摘要： A method is disclosed for improving encapsidation of transgene RNA using retroviral packaging and transfer vectors. An HIV-2 transfer vector, which includes the transgene, is introduced into a packaging cell that is also transfected with (or stably expresses) an HIV-2 derived packaging vector or a combination of packaging vectors. The packaging vector has mutations in packaging signal sequences that are both upstream and downstream of the 5′ splice donor site. It can also be composed of a combination of two or more partial vectors. A transfer vector, which is introduced into the packaging cell line, has a mutation that renders its splice donor site non-functional. Transgene RNA expression and encapsidation from these cells is markedly increased, but with little or no levels of infectious viral RNA encapsidation.
摘要翻译：公开了一种使用逆转录病毒包装和转移载体来改善转基因RNA的壳化的方法。将包含转基因的HIV-2转移载体导入也用（或稳定表达）HIV-2衍生的包装载体或包装载体的组合的包装细胞中。包装载体在5'剪接供体位点的上游和下游的包装信号序列具有突变。它也可以由两个或更多个部分向量的组合组成。引入到包装细胞系中的转移载体具有使其剪接供体位点无功能的突变。来自这些细胞的转基因RNA表达和壳化显着增加，但具有很少或没有感染性病毒RNA包被水平。

5. 发明授权

US06541219B1 Therapeutic Gene 有权
标题翻译：治疗基因
公开(公告)号：US06541219B1
公开(公告)日：2003-04-01
申请号：US09254832
申请日：1999-06-21
申请人： Alan John Kingsman , Susan Mary Kingsman
发明人： Alan John Kingsman , Susan Mary Kingsman
IPC分类号： C12N15867
CPC分类号： C12N9/0071 , A61K48/00 , C07K2319/00 , C12N9/88 , C12N15/62 , C12N2799/027
摘要： Polynucleotide sequences and vectors containing them, for use in gene therapy, the polynucleotide sequences comprising two or more therapeutic genes operably linked to a promoter and encoding a fusion protein product of the therapeutic genes. The fusion protein may be for example a tyrosine hydroxylase (TH)-DOPA decarboxylase (DD) fusion in either TH-DD or DD-TH order, useful for treating Parkinson's disease.
摘要翻译：含有它们的多核苷酸序列和载体用于基因治疗，所述多核苷酸序列包含与启动子可操作地连接并编码治疗基因的融合蛋白产物的两个或更多个治疗基因。融合蛋白可以是例如TH-DD或DD-TH序列中用于治疗帕金森病的酪氨酸羟化酶（TH）-DOPA脱羧酶（DD）融合物。

6. 发明授权

US06506604B2 Method for production of high titer virus and high efficiency retroviral mediated transduction of mammalian cells 失效
标题翻译：生产高滴度病毒和高效逆转录病毒介导的哺乳动物细胞转导的方法
公开(公告)号：US06506604B2
公开(公告)日：2003-01-14
申请号：US09944411
申请日：2001-09-04
申请人： Mitchell H. Finer , Thomas J. Dull , Krisztina M. Zsebo , Keegan Cooke , Deborah A. Farson
发明人： Mitchell H. Finer , Thomas J. Dull , Krisztina M. Zsebo , Keegan Cooke , Deborah A. Farson
IPC分类号： C12N15867
CPC分类号： C12N7/00 , A61K48/00 , C07K14/005 , C07K14/7051 , C07K14/70514 , C07K14/721 , C07K16/10 , C07K2319/00 , C12N15/86 , C12N15/87 , C12N2740/13022 , C12N2740/13043 , C12N2740/13052 , C12N2740/13062
摘要： The invention provides a novel retroviral packaging system, in which retroviral packaging plasmids and packagable vector transcripts are produced from high expression plasmids after stable or transient transfection in mammalian cells. High titers of recombinant retrovirus are produced in these transfected mammalian cells and can then transduce a mammalian target cell by cocultivation or supernatant infection. The methods of the invention include the use of the novel retroviral packaging plasmids and vectors to transduce primary human cells, including T cells and, human hematopoietic stem cells, with foreign genes by cocultivation or supernatant infection at high efficiencies. The invention is is useful for the rapid production of high titer viral supernatants, and to transduce with high efficiency cells that are refractory to transduction by conventional means.
摘要翻译：本发明提供了一种新型的逆转录病毒包装系统，其中在哺乳动物细胞中稳定或瞬时转染之后，由高表达质粒产生逆转录病毒包装质粒和可包装载体转录物。在这些转染的哺乳动物细胞中产生高滴度的重组逆转录病毒，然后可以通过共培养或上清液感染来转导哺乳动物靶细胞。本发明的方法包括使用新的逆转录病毒包装质粒和载体以高效率通过共培养或上清液感染来转导包含T细胞和人造血干细胞的原代人细胞与外来基因。本发明可用于快速生产高滴度病毒上清液，并且可以通过常规方法转导难以转导的高效细胞。

7. 发明授权

US06218187B1 Method for production of high titer virus and high efficiency retroviral mediated transduction of mammalian cells 有权
标题翻译：生产高滴度病毒和高效逆转录病毒介导的哺乳动物细胞转导的方法
公开(公告)号：US06218187B1
公开(公告)日：2001-04-17
申请号：US09266596
申请日：1999-03-11
申请人： Mitchell H. Finer , Thomas J. Dull , Krisztina M. Zsebo , Keegan Cooke , Deborah A. Farson
发明人： Mitchell H. Finer , Thomas J. Dull , Krisztina M. Zsebo , Keegan Cooke , Deborah A. Farson
IPC分类号： C12N15867
CPC分类号： C12N15/86 , A61K48/00 , C07K14/005 , C07K14/7051 , C07K14/70514 , C07K14/721 , C07K16/10 , C07K2319/00 , C12N7/00 , C12N15/87 , C12N2740/13022 , C12N2740/13023 , C12N2740/13043 , C12N2740/13052 , C12N2740/13062
摘要： The invention provides a novel retroviral packaging system, in which retroviral packaging plasmids and packagable vector transcripts are produced from high expression plasmids after stable or transient transfection in mammalian cells. High titers of recombinant retrovirus are produced in these transfected mammalian cells and can then transduce a mammalian target cell by cocultivation or supernatant infection. The methods of the invention include the use of the novel retroviral packaging plasmids and vectors to transduce primary human cells, including T cells and human hematopoietic stem cells, with foreign genes by cocultivation or supernatant infection at high efficiencies. The invention is useful for the rapid production of high titer viral supernatants, and to transduce with high efficiency cells that are refractory to transduction by conventional means.
摘要翻译：本发明提供了一种新型的逆转录病毒包装系统，其中在哺乳动物细胞中稳定或瞬时转染之后，由高表达质粒产生逆转录病毒包装质粒和可包装载体转录物。在这些转染的哺乳动物细胞中产生高滴度的重组逆转录病毒，然后可以通过共培养或上清液感染来转导哺乳动物靶细胞。本发明的方法包括使用新型逆转录病毒包装质粒和载体以高效率通过共培养或上清液感染将原代人细胞（包括T细胞和人造血干细胞）转染外来基因。本发明可用于快速生产高滴度病毒上清液，并且可以通过常规方法转导难以转导的高效细胞。

8. 发明授权

US06818439B1 Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders 失效
标题翻译：用于治疗血友病和其他疾病的重组基因递送载体的给药方法
公开(公告)号：US06818439B1
公开(公告)日：2004-11-16
申请号：US09001039
申请日：1997-12-30
申请人： Douglas J. Jolly , Stephen Chang , James G. Respess , Nicholas J. DePolo , David Chi-Tang Hsu , Carlos E. Ibanez , Judith Greengard , Lee Will
发明人： Douglas J. Jolly , Stephen Chang , James G. Respess , Nicholas J. DePolo , David Chi-Tang Hsu , Carlos E. Ibanez , Judith Greengard , Lee Will
IPC分类号： C12N15867
CPC分类号： C12N15/86 , A61K38/00 , A61K48/00 , C07K14/005 , C07K14/56 , C07K14/61 , C07K14/705 , C07K14/7455 , C07K14/755 , C07K14/8125 , C07K14/8128 , C12N9/1211 , C12N9/644 , C12N9/6464 , C12N9/647 , C12N2710/10322 , C12N2710/10343 , C12N2740/13043 , C12Y304/21022 , C12Y304/21069
摘要： Methods are provided for obtaining measurable levels of a protein, nucleic acid molecule, or enzymatic product in a bodily fluid or cells of a human, comprising the step of administering to a human a recombinant retroviral preparation having a titer on HT1080 cells of greater than 105 cfu/ml, wherein the recombinant retroviral preparation is capable of directing the expression of a protein, nucleic acid molecule, or enzyme which generates an enzymatic product, such that measurable levels of the protein, nucleic acid molecule, or enzymatic product may be obtained in the bodily fluid or cells of the human.
摘要翻译：提供了用于在体液或人体细胞中获得可测量水平的蛋白质，核酸分子或酶产物的方法，其包括以下步骤：将具有大于10的HT1080细胞滴度的重组逆转录病毒制剂给予人其中重组逆转录病毒制剂能够引导产生酶产物的蛋白质，核酸分子或酶的表达，使得蛋白质，核酸分子或酶促产物的可测量水平可以可以在人的体液或细胞中获得。

9. 发明授权

US06485977B1 Recombinant constructs and techniques for delivering to eucaryotic cells bacterial proteins that are secreted via type III secretion systems 失效
标题翻译：用于递送至真核细胞的重组构建体和技术通过III型分泌系统分泌的细菌蛋白质
公开(公告)号：US06485977B1
公开(公告)日：2002-11-26
申请号：US09660742
申请日：2000-09-13
申请人： Alan Collmer , Steven V. Beer
发明人： Alan Collmer , Steven V. Beer
IPC分类号： C12N15867
CPC分类号： C07K14/005 , A61K38/00 , A61K48/00 , C07K14/195 , C07K14/21 , C07K14/4702 , C07K2319/00 , C07K2319/02 , C07K2319/10 , C07K2319/31 , C07K2319/55 , C07K2319/75 , C07K2319/80 , C12N15/62 , C12N2740/16322 , C12P21/02
摘要： The present invention relates to a method for delivering effector proteins into a target cell. This method involves introducing into the target cell an effector protein fused to a protein transduction domain of a human immunodeficiency virus TAT protein or derivatives or functional analogs thereof. The present invention also relates to a fusion protein including an effector protein fused to a protein transduction domain of a human immunodeficiency virus TAT protein or derivatives or functional analogs thereof. Another aspect of the present invention relates to a DNA construct including a first DNA molecule encoding an effector protein and a second DNA molecule operatively associated with the first DNA molecule and encoding a protein transduction domain of a human immunodeficiency virus TAT protein or derivatives or functional analogs thereof and its use in a method for delivering effector proteins into a target cell.
摘要翻译：本发明涉及一种将效应蛋白递送到靶细胞中的方法。该方法包括向靶细胞引入与人免疫缺陷病毒TAT蛋白的蛋白转导结构域融合的效应蛋白或其衍生物或功能类似物。本发明还涉及包含与人免疫缺陷病毒TAT蛋白的蛋白转导结构域融合的效应子蛋白或其衍生物或功能类似物的融合蛋白。本发明的另一方面涉及包含编码效应蛋白的第一DNA分子和与第一DNA分子可操作地相关联并编码人免疫缺陷病毒TAT蛋白或其衍生物或功能类似物的蛋白转导结构域的第二DNA分子的DNA构建体并且其用于将效应蛋白递送至靶细胞的方法中的用途。

10. 发明授权

US06451595B1 High efficiency retroviral vectors that contain none of viral coding sequences 有权
标题翻译：不含病毒编码序列的高效率逆转录病毒载体
公开(公告)号：US06451595B1
公开(公告)日：2002-09-17
申请号：US09463067
申请日：2000-01-14
申请人： Sunyoung Kim , Seung Shin Yu , Jong-mook Kim
发明人： Sunyoung Kim , Seung Shin Yu , Jong-mook Kim
IPC分类号： C12N15867
CPC分类号： C12N15/86 , C12N2740/13043 , C12N2830/42 , C12N2830/50 , C12N2840/203 , C12N2840/44
摘要： The present invention relates to improved retroviral vectors for gene therapy. In this invention, retroviral vectors with higher safety and efficiency are constructed from MLV-based starting vectors, MON and MIN. The improved vectors have the following features: 1) sequences corresponding to MLV-derived pol gene are completely deleted in the vectors, avoiding homologous recombination which has been a baffling problem in conventional retroviral vectors, 2) a heterologous intron, splicing acceptor and/or non-coding sequence are/is inserted into the upstream position of a cloning site, maximizing the expression of a foreign gene through efficient splicing, 3) the vectors contain either the full-length U3 sequence of 5′ LTR or a strong heterologous promoter instead, permitting the abundant production of RNA, 4) either IRES (Internal Ribosomal Entry Site) or internal SV40 minimal promoter is inserted into the downstream position of cloning site, enabling the simultaneous expression of two or more foreign genes. Since the improved retroviral vectors of this invention turn out to be safe and to express the foreign gene efficiently, they are useful for gene therapy and the like.
摘要翻译：本发明涉及用于基因治疗的改良逆转录病毒载体。在本发明中，具有更高安全性和效率的逆转录病毒载体由基于MLV的起始载体MON和MIN构建。改进的载体具有以下特征：1）对应于MLV衍生的pol基因的序列在载体中完全缺失，避免了在常规逆转录病毒载体中已经成为障碍问题的同源重组，2）异源内含子，剪接受体和/或非编码序列被插入到克隆位点的上游位置，通过有效剪接使外源基因的表达最大化，3）载体含有5'LTR的全长U3序列或者强异源启动子，允许大量产生RNA，4）将IRES（内部核糖体进入位点）或内部SV40最小启动子插入到克隆位点的下游位置，使得能够同时表达两个或多个外源基因。由于本发明改良的逆转录病毒载体是安全的并有效表达外源基因，所以它们可用于基因治疗等。

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