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    AAV vectors for gene therapy
    1.
    发明授权
    AAV vectors for gene therapy 有权
    用于基因治疗的AAV载体

    公开(公告)号：US06632670B1

    公开(公告)日：2003-10-14

    申请号：US09029705

    申请日：1998-11-16

    申请人： Samuel C. Wadsworth ,  Karen Vincent ,  Susan Piraino ,  Sirkka Kyostio

    发明人： Samuel C. Wadsworth ,  Karen Vincent ,  Susan Piraino ,  Sirkka Kyostio

    IPC分类号： C12N15864

    CPC分类号： C12N7/00 ,  A01K2217/05 ,  A61K48/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14152

    摘要： The present invention is directed to methods for generating high titer, contaminant free, recombinant AAV vectors, methods and genetic constructs for producing recombinant AAV vectors conveniently and in large quantities, methods for the delivery of all essential viral proteins required in trans for high yields of recombinant AAV, recombinant AAV vectors for use in gene therapy, novel packaging cell lines which obviate the need for cotransfection of vector and helper plasmids, helper plasmids and vector plasmid backbone constructs, a reporter assay for determining AAV vector yield. Further provided are recombinant AAV vectors in a pharmaceutically acceptable carrier, methods of delivering a transgene of interest to a cell, compositions and methods for delivering a DNA sequence encoding a desired polypeptide to a cell, and transgenic non-human mammals that express a human chromosome 19 AAV integration locus.

    摘要翻译： 本发明涉及用于产生高效价，无污染的重组AAV载体的方法，用于方便和大量生产重组AAV载体的方法和遗传构建体，用于递送反式所需的所有必需病毒蛋白的高产率的方法 重组AAV，用于基因治疗的重组AAV载体，新型包装细胞系，其不需要共转染载体和辅助质粒，辅助质粒和载体质粒骨架构建体，用于测定AAV载体产量的报道分析。 还提供了药学上可接受的载体中的重组AAV载体，将感兴趣的转基因递送至细胞的方法，用于将编码所需多肽的DNA序列递送至细胞的组合物和方法，以及表达人染色体的转基因非人哺乳动物 19 AAV整合轨迹。

    Genetic modification of primate hemopoietic repopulating stem cells
    3.
    发明授权
    Genetic modification of primate hemopoietic repopulating stem cells 失效
    灵长类动物造血干细胞的遗传修饰

    公开(公告)号：US06312957B1

    公开(公告)日：2001-11-06

    申请号：US09326032

    申请日：1999-06-04

    申请人： Markus Peter Wilhelmus Einerhand ,  Domenico Valerio

    发明人： Markus Peter Wilhelmus Einerhand ,  Domenico Valerio

    IPC分类号： C12N15864

    CPC分类号： C12N15/86 ,  A61K38/00 ,  A61K48/00 ,  C07K14/805 ,  C12N2750/14143

    摘要： Genetic modification of pluripotent hemopoietic stem cells of primates (P-PHSC) by transduction of P-PHSC with a recombinant adeno-associated virus (AAV). Tile genomc of the recombinant AAV comprises a DNA sequence flanked by the inverted terminal repeats (ITR) of AAV. The DNA sequence will normally comprise regulatory sequences which are functional in hemopoictic cells and, controlled by these regulatory sequences, a sequence coding for a protein or RNA with a therapeutic property when introduced into hemopoietic cells. Preferred examples of DNA sequences are the human lysosomal glococerebrosidase gene, a globin gene from the human &bgr;-globin gene cluster, a DNA sequence encoding an RNA or protein with anti-viral activity, the &agr;1-antitrypsin gene and the human multidrug resistance gene I (MDRI). The invention provides for effective gene therapy with PHSC of primates, in particular humans.

    摘要翻译： 通过用重组腺相关病毒（AAV）转导P-PHSC，灵长类动物多能造血干细胞（P-PHSC）的遗传修饰。 重组AAV的平铺基因组包含侧翼为AAV的反向末端重复序列（ITR）的DNA序列。 DNA序列通常包含在血液细胞中有功能的调控序列，并且由这些调节序列控制，当引入造血细胞时，编码具有治疗性质的蛋白质或RNA的序列。 DNA序列的优选实例是人溶酶体glococerebrosidase基因，来自人β珠蛋白基因簇的珠蛋白基因，编码具有抗病毒活性的RNA或蛋白质的DNA序列，α1-抗胰蛋白酶基因和人多药耐药基因I （MDRI）。 本发明提供用灵长类动物，特别是人的PHSC进行有效的基因治疗。

    Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
    5.
    发明授权
    Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV 失效
    用于介导腺相关病毒（AAV）附着和感染的方法和制剂以及用于纯化AAV的方法

    公开(公告)号：US06703237B2

    公开(公告)日：2004-03-09

    申请号：US10102314

    申请日：2002-03-20

    申请人： Richard Jude Samulski ,  Candace Summerford

    发明人： Richard Jude Samulski ,  Candace Summerford

    IPC分类号： C12N15864

    CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N7/00 ,  C12N2750/14143 ,  C12N2750/14151

    摘要： Primary receptors and co-receptors for adeno-associated virus (AAV) attachment to and infection of target cells are described. Such receptors can be used to facilitate AAV attachment to and infection of cells, e.g., for gene therapy. Methods for purification and/or concentration of AAV are also described. Methods of facilitating or enhancing AAV infection of a cell are also provided. Also described are methods of inhibiting or preventing infection of AAV into a cell. Cell samples may be screened for permissiveness for AAV attachment and infection by detecting the presence or abundance of cellular receptors that mediate attachment and/or infection of AAV into the cell. Formulations and kits for mediating AAV attachment to, and infection of, cells are also provided herein.

    摘要翻译： 描述了腺相关病毒（AAV）附着和靶细胞感染的主要受体和辅助受体。 这样的受体可用于促进AAV附着和感染细胞，例如用于基因治疗。 还描述了AAV的纯化和/或浓缩方法。 还提供了促进或增强细胞的AAV感染的方法。 还描述了抑制或预防AAV感染细胞的方法。 可以通过检测介导AAV附着和/或感染细胞的细胞受体的存在或丰度来筛选细胞样品的AAV附着和感染的允许性。 本文还提供了用于介导AAV附着和细胞感染的制剂和试剂盒。