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    METHODS AND COMPOSITIONS RELATING TO IMPROVED LENTIVIRAL VECTOR PRODUCTION SYSTEMS
    4.
    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。

    Packaging systems for human recombinant adenovirus to be used in gene therapy
    6.
    发明申请
    Packaging systems for human recombinant adenovirus to be used in gene therapy 失效
    用于基因治疗的人重组腺病毒的包装系统

    公开(公告)号：US20050074885A1

    公开(公告)日：2005-04-07

    申请号：US10396548

    申请日：2003-03-25

    申请人： Ronald Vogels ,  Abraham Bout

    发明人： Ronald Vogels ,  Abraham Bout

    IPC分类号： C12N15/09 ,  A61K48/00 ,  C07K14/075 ,  C12N5/10 ,  C12N7/00 ,  C12N7/01 ,  C12N15/861 ,  C12R1/01 ,  C12N5/06

    CPC分类号： C12N7/00 ,  A61K48/00 ,  C07K14/005 ,  C12N15/86 ,  C12N2710/10322 ,  C12N2710/10343 ,  C12N2710/10352 ,  C12N2830/00 ,  C12N2830/15 ,  C12N2830/38 ,  C12N2830/60

    摘要： The invention provides improved methods and products based on adenoviral materials which can advantageously be used in gene therapy. In one aspect, an adenoviral vector is provided having no overlap with a suitable packaging cell line. The suitable packaging cell line is another aspect. The combination excludes the possibility of homologous recombination, thereby excluding the possibility of the formation of replication competent adenovirus. Another aspect embodies an adenovirus based helper construct which by its size is incapable of being encapsidated. This helper virus can be transferred into any suitable host cell making it a packaging cell. Furthermore, a number of useful mutations to adenoviral based materials and combinations of such mutations are disclosed, all of which have in common the safety of the methods and the products, in particular avoiding the production of replication competent adenovirus and/or interference with the immune system.

    摘要翻译： 本发明提供了可以有利地用于基因治疗的基于腺病毒材料的改进的方法和产品。 在一个方面，提供了与合适的包装细胞系不重叠的腺病毒载体。 合适的包装细胞系是另一个方面。 该组合不包括同源重组的可能性，从而排除形成复制型腺病毒的可能性。 另一方面体现了一种基于腺病毒的辅助构建体，其大小不能被包裹。 这种辅助病毒可以转移到任何合适的宿主细胞中，使其成为包装细胞。 此外，公开了对基于腺病毒的材料和这种突变的组合的许多有用的突变，所有这些都具有方法和产品的安全性，特别是避免生产复制有关的腺病毒和/或免疫的干扰 系统。

    Methods of inhibiting gene expression by RNA interference
    7.
    发明申请
    Methods of inhibiting gene expression by RNA interference 审中-公开
    通过RNA干扰抑制基因表达的方法

    公开(公告)号：US20040234504A1

    公开(公告)日：2004-11-25

    申请号：US10742740

    申请日：2003-12-18

    发明人： Inder M. Verma ,  Gustavo Tiscornia ,  Oded Singer

    IPC分类号： A61K048/00 ,  C12N015/867

    CPC分类号： C12N15/111 ,  A61K48/00 ,  C12N15/86 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2330/30 ,  C12N2740/16043 ,  C12N2800/30 ,  C12N2830/002 ,  C12N2830/15 ,  C12N2830/38 ,  C12N2830/48 ,  C12N2830/50 ,  C12N2840/20

    摘要： The invention provides a lentiviral vector capable of inhibiting the expression of at least one target gene. A lentiviral vector of the invention encompasses a first nucleic acid sequence derived from a target gene transcript and a second nucleic acid sequence corresponding to the reverse complement of said first nucleic acid sequence. A lentiviral vector of the invention capable of inhibiting the expression of a target gene is useful in therapeutic applications to inactivate disease-associated transcripts and thereby reduce the severity of inherited metabolic, infectious or malignant conditions. Methods for inhibiting one or more target genes in a cell as well as methods for producing a non-human mammal in which the expression of one or more target genes is inhibited also are provided by the present invention.

    摘要翻译： 本发明提供能够抑制至少一种靶基因表达的慢病毒载体。 本发明的慢病毒载体包括衍生自靶基因转录物的第一核酸序列和对应于所述第一核酸序列的反向互补序列的第二核酸序列。 能够抑制靶基因表达的本发明的慢病毒载体可用于灭活疾病相关转录物的治疗应用，从而降低遗传性代谢，感染性或恶性病症的严重性。 本发明还提供了抑制细胞中一种或多种靶基因的方法以及抑制一种或多种靶基因表达的非人哺乳动物的制备方法。

    Helper dependent adenoviral vector system and methods for using the same
    8.
    发明申请
    Helper dependent adenoviral vector system and methods for using the same 审中-公开
    辅助依赖性腺病毒载体系统及其使用方法

    公开(公告)号：US20040203133A1

    公开(公告)日：2004-10-14

    申请号：US10753962

    申请日：2004-01-07

    发明人： Anja Ehrhardt ,  Mark A. Kay

    IPC分类号： C12N015/86 ,  C12N007/00

    CPC分类号： C12N15/86 ,  C12N2710/10343 ,  C12N2800/108 ,  C12N2800/30 ,  C12N2800/80 ,  C12N2800/90 ,  C12N2830/002 ,  C12N2830/008 ,  C12N2830/38 ,  C12N2830/42 ,  C12N2830/46 ,  C12N2830/85

    摘要： A helper dependent adenoviral vector system is provided. The subject helper dependent adenoviral vector system is made up of: (1) a nullgutlessnull adenoviral vector, which in certain embodiments includes cis-acting human stuffer DNA that provides for in vivo long term, high level expression of a coding sequence present on the vector, where in certain embodiments the vector includes an integrating domain; (2) an adenoviral helper vector that is characterized by having an adenoviral genome region flanked by recombinase recognition sites, where the helper vectors further include a non-mammalian endonuclease recognition site positioned outside of the adenoviral genome region and in certain embodiments a third adenoviral inverted terminal repeat (ITR) sequence positioned between first and second terminal ITRs; and (3) a mammalian cell that expresses the corresponding recombinase and endonuclease, as well as the adenoviral preterminal and polymerase proteins. Also provided are methods of using the subject systems to produce virions having the subject helper dependent adenoviral vectors encapsulated in an adenoviral capsid. In addition, kits for use in practicing the subject methods are provided.

    摘要翻译： 提供辅助性依赖性腺病毒载体系统。 受试者辅助因子依赖性腺病毒载体系统由以下部分组成：（1）“无味”腺病毒载体，其在某些实施方案中包括顺式作用人填充DNA，其提供体内长期，高水平表达存在于 矢量，其中在某些实施例中，矢量包括积分域; （2）腺病毒辅助载体，其特征在于具有侧翼为重组酶识别位点的腺病毒基因组区域，其中辅助载体还包括位于腺病毒基因组区域外的非哺乳动物内切核酸酶识别位点，在某些实施方案中，第三腺病毒反向 终端重复（ITR）序列位于第一和第二终端ITR之间; 和（3）表达相应的重组酶和内切核酸酶的哺乳动物细胞，以及腺病毒早产和聚合酶蛋白。 还提供了使用受试系统产生具有包封在腺病毒衣壳中的受试者辅助性依赖性腺病毒载体的病毒粒子的方法。 此外，还提供了用于实践主题方法的套件。

    Methods of infection with hepatitis c virus
    10.
    发明申请
    Methods of infection with hepatitis c virus 审中-公开
    丙型肝炎病毒感染方法

    公开(公告)号：US20040101827A1

    公开(公告)日：2004-05-27

    申请号：US10398076

    申请日：2003-04-02

    发明人： Michinori Kohara ,  Asao Katsume

    IPC分类号： C12Q001/70

    CPC分类号： C12N15/8509 ,  A01K2227/106 ,  A01K2267/0337 ,  C12N15/86 ,  C12N2770/24211 ,  C12N2770/24243 ,  C12N2830/38

    摘要： The present invention relates to a method for efficiently infecting a small animal with hepatitis C virus. Specifically, the present invention relates to a method for infecting a small animal with hepatitis C virus, which comprises a step of administering RNA containing the genomic RNA of hepatitis C virus into the liver, or a step of administering to a small animal the culture supernatant of an animal cell having a vector that contains cDNA corresponding to the genomic RNA of hepatitis C virus introduced therein, and relates to a small animal infected with hepatitis C virus by these methods. The present invention further relates to a method for screening for remedies against hepatitis C virus-associated diseases, or substances that inhibit the growth of hepatitis C virus using the yield of the virus as an indicator.

    摘要翻译： 本发明涉及一种有效感染丙型肝炎病毒的小动物的方法。 具体地说，本发明涉及用于感染丙型肝炎病毒的小动物的方法，其包括将含有丙型肝炎病毒的基因组RNA的RNA施用于肝脏的步骤，或向小动物施用培养上清液的步骤 的具有载体的动物细胞，该载体含有对应于其中引入的丙型肝炎病毒的基因组RNA的cDNA，涉及通过这些方法感染丙型肝炎病毒的小动物。 本发明还涉及一种用于筛选针对丙型肝炎病毒相关疾病的药物的方法，或使用病毒产量作为指标抑制丙型肝炎病毒生长的物质。