利索-全球专利检索

  1. 登录
  2. 注册

搜索结果

22 条记录 (耗时 0.033 秒)

    Methods and compositions relating to restricted expression lentiviral vectors and their applications
    5.
    发明授权
    Methods and compositions relating to restricted expression lentiviral vectors and their applications 有权
    与限制性表达慢病毒载体相关的方法和组合及其应用

    公开(公告)号：US08748169B2

    公开(公告)日：2014-06-10

    申请号：US11680414

    申请日：2007-02-28

    申请人： Didier Trono ,  Maciej Wiznerowicz

    发明人： Didier Trono ,  Maciej Wiznerowicz

    IPC分类号： C12N15/63 ,  C12N15/85

    CPC分类号： C12N15/86 ,  A61K48/00 ,  C07K14/55 ,  C07K14/70503 ,  C07K16/00 ,  C07K2317/622 ,  C07K2319/30 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2800/30 ,  C12N2830/00 ,  C12N2830/002 ,  C12N2830/008 ,  C12N2830/30 ,  C12N2830/48 ,  C12N2830/50 ,  C12N2830/85 ,  C12N2840/20 ,  C12N2840/44

    摘要： The present invention provides HIV-derived lentivectors which are safe, highly efficient, and very potent for expressing transgenes for human gene therapy, especially, in human hematopoietic progenitor cells as well as in all other blood cell derivatives. The lentiviral vectors comprise promoters active to promote expression specific to cell types or tissues. Further, promoters are providing that are amenable to control by activators, enhancers, or repressors. These vectors are in a self-inactivating configuration for biosaftey. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element, without any decrease in the specificity or control exerted by the promoters. These vectors therefore provide useful tools for genetic treatments such as inherited and acquired lympho-hematological disorders, gene-therapies for cancers especially the hematological cancers, as well as for the study of hematopoiesis via lentivector-mediated modification of human HSCs.

    摘要翻译： 本发明提供了用于人基因治疗特别是人类造血祖细胞以及所有其他血液细胞衍生物中表达转基因的安全，高效和非常有效的HIV衍生的慢病毒药物。 慢病毒载体包含促进对细胞类型或组织特异性表达的活性的启动子。 此外，提供可由活化剂，增强剂或阻遏物控制的启动子。 这些载体是生物燃料的自灭活配置。 还描述了另外的启动子。 载体还可以包含额外的转录增强元件，例如木卡因肝炎病毒转录后调节元件，而启动子所引起的特异性或对照的任何降低。 因此，这些载体为遗传治疗提供了有用的工具，例如遗传和获得性淋巴 - 血液学障碍，癌症特别是血液癌症的基因治疗，以及通过慢性介导的人类HSC修饰研究造血功能。

    Chimeric glycoproteins and pseudotyped lentiviral vectors
    6.
    发明授权
    Chimeric glycoproteins and pseudotyped lentiviral vectors 有权
    嵌合糖蛋白和假型慢病毒载体

    公开(公告)号：US08338168B2

    公开(公告)日：2012-12-25

    申请号：US10512474

    申请日：2003-04-25

    申请人： Didier Trono ,  Francois-Loic Cosset ,  Virginie Sandrin ,  Bertrand Boson ,  Didier Negre ,  Patrick Salmon

    发明人： Didier Trono ,  Francois-Loic Cosset ,  Virginie Sandrin ,  Bertrand Boson ,  Didier Negre ,  Patrick Salmon

    IPC分类号： C12N15/00 ,  C12N15/64

    CPC分类号： C12N15/86 ,  A61K48/00 ,  C07K14/005 ,  C07K2319/00 ,  C07K2319/03 ,  C12N7/045 ,  C12N2510/02 ,  C12N2740/13022 ,  C12N2740/13043 ,  C12N2740/15043 ,  C12N2740/15045 ,  C12N2810/60 ,  C12N2830/50

    摘要： The present invention provides improved chimeric glycoproteins (GPs) and improved lentiviral vectors pseudotyped with those glycoproteins. Also provided are methods and compositions for making such glycoproteins and vectors, and improved methods of in vitro and in vivo transduction of cells with such vectors. Improved chimeric GPs encode the extracellular and transmembrane domains of GALV or RD114 GPs fused to the cytoplasmic tail of MLV-A GP. Vectors pseudotyped with these GAL V/TR and RD 114/TR GP chimeras have significantly higher titers than vectors coated with the parental GPs. Additionally, RD114/TR-pseudotyped vectors are efficiently concentrated and are resistant to inactivation induced by the complement of both human and macaque sera. RD114 GP-pseudotyped lentiviral vectors have particular utility for in vivo gene transfer applications.

    摘要翻译： 本发明提供改进的嵌合糖蛋白（GP）和用这些糖蛋白假型分类的改良的慢病毒载体。 还提供了用于制备这种糖蛋白和载体的方法和组合物，以及用这些载体体外和体内转导细胞的改进方法。 改进的嵌合GP编码与MLV-A GP的细胞质尾融合的GALV或RD114GP的细胞外和跨膜结构域。 与这些GAL V / TR和RD 114 / TR GP嵌合体假型分离的载体具有比用亲本GP包被的载体更高的滴度。 此外，RD114 / TR-假型载体有效浓缩，并且对由人和猕猴血清的补体诱导的失活具有抗性。 RD114 GP-假型慢病毒载体对于体内基因转移应用具有特殊的用途。

    METHODS AND COMPOSITIONS RELATING TO IMPROVED LENTIVIRAL VECTOR PRODUCTION SYSTEMS
    7.
    发明申请
    METHODS AND COMPOSITIONS RELATING TO IMPROVED LENTIVIRAL VECTOR PRODUCTION SYSTEMS 有权
    与改进的生物载体生产系统相关的方法和组合

    公开(公告)号：US20100062524A1

    公开(公告)日：2010-03-11

    申请号：US12578346

    申请日：2009-10-13

    申请人： Didier Trono ,  Romain N. Zufferey

    发明人： Didier Trono ,  Romain N. Zufferey

    IPC分类号： C12N15/63

    CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N7/00 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2740/16052 ,  C12N2740/16061 ,  C12N2830/38 ,  C12N2830/48 ,  C12N2830/50 ,  C12N2840/203 ,  C12N2840/44

    摘要： The present invention provides HIV-derived lentivectors which are multiply modified to create highly safe, efficient, and potent vectors for expressing transgenes for gene therapy. The lentiviral vectors comprise various combinations of an inactive central polypurine tract, a stuffer sequence, which may encode drug susceptibility genes, and a mutated hairpin in the 5′ leader sequence that substantially abolishes replication. These elements are provided in conjunction with other features of lentiviral vectors, such as a self-inactivating configuration for biosaftey and promoters such as the EF1α promoter as one example. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element. These vectors therefore provide useful tools for genetic treatments for inherited and acquired disorders, gene-therapies for cancers and other disease, the creation of industrial and experimental production systems utilizing transformed cells, as well as for the study of basic cellular and genetic processes.

    摘要翻译： 本发明提供了HIV衍生的慢病毒药物，其被多重修饰以产生用于表达用于基因治疗的转基因的高度安全，有效和有效的载体。 慢病毒载体包括不规则的中枢多巴胺，可编码药物敏感性基因的填充序列和基本上消除复制的5'前导序列中突变的发夹的各种组合。 这些元件与慢病毒载体的其它特征结合提供，例如用于生物卫生的自灭活构型和启动子如EF1α启动子作为一个实例。 还描述了另外的启动子。 载体还可以包含额外的转录增强元件，例如木吸虫肝炎病毒转录后调节元件。 因此，这些载体为继承和获得性疾病的遗传治疗，癌症和其他疾病的基因治疗，利用转化细胞的工业和实验生产系统的建立以及基础细胞和遗传过程的研究提供了有用的工具。

    Gene expression in mammalian cells using a cap independent 5' noncoding region
    9.
    发明授权
    Gene expression in mammalian cells using a cap independent 5' noncoding region 失效
    使用帽独立的5'非编码区在哺乳动物细胞中的基因表达

    公开(公告)号：US5358856A

    公开(公告)日：1994-10-25

    申请号：US887223

    申请日：1992-05-18

    申请人： David Baltimore ,  Didier Trono

    发明人： David Baltimore ,  Didier Trono

    IPC分类号： C12N15/85 ,  C12N5/10 ,  C12P21/02

    CPC分类号： C12N15/85 ,  C12N2840/203

    摘要： A construct which includes a cap independent 5' noncoding region of viral or cellular origin and a nucleotide sequence of interest, which is located downstream of the noncoding region. A method of producing a protein or a polypeptide of interest by introducing the construct, including a nucleotide sequence encoding the protein or the polypeptide of interest, into mammalian cells is also described. In one embodiment, the construct comprises all or a portion of the poliovirus cap independent 5' noncoding region and a nucleotide sequence encoding a protein or a polypeptide of interest.

    摘要翻译： 包括病毒或细胞来源的帽独立的5'非编码区和感兴趣的核苷酸序列的构建体，其位于非编码区的下游。 还描述了通过将编码所述蛋白质或目标多肽的核苷酸序列引入哺乳动物细胞的构建体来产生目的蛋白质或多肽的方法。 在一个实施方案中，构建体包含脊髓灰质炎病毒帽独立的5'非编码区的全部或一部分和编码感兴趣的蛋白质或多肽的核苷酸序列。

    Methods and compositions relating to improved lentiviral vector production systems
    10.
    发明授权
    Methods and compositions relating to improved lentiviral vector production systems 有权
    与改良的慢病毒载体生产系统相关的方法和组合物

    公开(公告)号：US08900858B2

    公开(公告)日：2014-12-02

    申请号：US12578346

    申请日：2009-10-13

    申请人： Didier Trono ,  Romain N. Zufferey

    发明人： Didier Trono ,  Romain N. Zufferey

    IPC分类号： C12N15/00 ,  C12N15/86 ,  C12N7/00

    CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N7/00 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2740/16052 ,  C12N2740/16061 ,  C12N2830/38 ,  C12N2830/48 ,  C12N2830/50 ,  C12N2840/203 ,  C12N2840/44

    摘要： The present invention provides HIV-derived lentivectors which are multiply modified to create highly safe, efficient, and potent vectors for expressing transgenes for gene therapy. The lentiviral vectors comprise various combinations of an inactive central polypurine tract, a stuffer sequence, which may encode drug susceptibility genes, and a mutated hairpin in the 5′ leader sequence that substantially abolishes replication. These elements are provided in conjunction with other features of lentiviral vectors, such as a self-inactivating configuration for biosaftey and promoters such as the EF1α promoter as one example. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element. These vectors therefore provide useful tools for genetic treatments for inherited and acquired disorders, gene-therapies for cancers and other disease, the creation of industrial and experimental production systems utilizing transformed cells, as well as for the study of basic cellular and genetic processes.

    摘要翻译： 本发明提供了HIV衍生的慢病毒药物，其被多重修饰以产生用于表达用于基因治疗的转基因的高度安全，有效和有效的载体。 慢病毒载体包括不规则的中枢多巴胺，可编码药物敏感性基因的填充序列和基本上消除复制的5'前导序列中突变的发夹的各种组合。 这些元件与慢病毒载体的其它特征结合提供，例如用于生物卫生的自灭活构型和启动子如EF1α启动子作为一个实例。 还描述了另外的启动子。 载体还可以包含额外的转录增强元件，例如木吸虫肝炎病毒转录后调节元件。 因此，这些载体为继承和获得性疾病的遗传治疗，癌症和其他疾病的基因治疗，利用转化细胞的工业和实验生产系统的建立以及基础细胞和遗传过程的研究提供了有用的工具。