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    Targeted introduction of DNA into primary or secondary cells and their use for gene therapy and protein production
    3.
    发明申请
    Targeted introduction of DNA into primary or secondary cells and their use for gene therapy and protein production 审中-公开
    将DNA引入原代细胞或次要细胞，并将其用于基因治疗和蛋白质生产

    公开(公告)号：US20050032215A1

    公开(公告)日：2005-02-10

    申请号：US10704322

    申请日：2003-11-06

    Applicant: Douglas Treco ,  Michael Heartlein ,  Richard Selden

    Inventor： Douglas Treco ,  Michael Heartlein ,  Richard Selden

    IPC: A61K48/00 ,  C12N15/00 ,  C12N15/09 ,  C12N15/64 ,  C12N15/85 ,  C12N15/87

    CPC classification number: A61K48/005 ,  A61K48/0058

    Abstract: The present invention relates to a method of gene or DNA targeting in cells of vertebrate, particularly mammalian, origin. That is, it relates to a method of introducing DNA into primary or secondary cells of vertebrate origin through homologous recombination or targeting of the DNA, which is introduced into genomic DNA of the primary or secondary cells at a preselected site. The present invention further relates to primary or secondary cells, referred to as homologously recombinant (HR) primary or secondary cells, produced by the present method and to uses of the homologously recombinant primary or secondary cells. The present invention also relates to a method of turning on a gene present in primary cells, secondary cells or immortalized cells of vertebrate origin, which is normally not expressed in the cells or is not expressed at significant levels in the cells.

    Abstract translation: 本发明涉及一种在脊椎动物，特别是哺乳动物来源的细胞中靶向的基因或DNA的方法。 也就是说，它涉及通过DNA的同源重组或靶向将DNA导入脊椎动物来源的一次或二次细胞的方法，该DNA在预先选择的位点被引入到初级细胞或次级细胞的基因组DNA中。 本发明还涉及通过本发明方法产生的称为同源重组（HR）初级或次级细胞的原代细胞或二次细胞，以及同源重组的初级细胞或次级细胞的用途。 本发明还涉及一种开放存在于原代细胞，二次细胞或脊椎动物来源的永生化细胞中的基因的方法，其通常不在细胞中表达或不在细胞中以显着水平表达。

    MITOCHONDRIAL TARGETING AND THERAPEUTIC USE THEREOF
    5.
    发明申请
    MITOCHONDRIAL TARGETING AND THERAPEUTIC USE THEREOF 有权
    麻醉指导和治疗方法

    公开(公告)号：US20140135275A1

    公开(公告)日：2014-05-15

    申请号：US14127074

    申请日：2012-06-15

    Applicant: Dennis Keefe ,  Michael Concino ,  Michael Heartlein ,  Serene Josiah ,  Bettina Strack-Logue

    Inventor： Dennis Keefe ,  Michael Concino ,  Michael Heartlein ,  Serene Josiah ,  Bettina Strack-Logue

    IPC: C07K14/47 ,  C07K7/08

    CPC classification number: C07K14/47 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0306 ,  A61K38/00 ,  A61K38/1709 ,  A61K47/645 ,  A61K49/0008 ,  C07K7/08 ,  C07K2319/07 ,  C07K2319/10

    Abstract: The present invention provides, among other things, compositions and methods for treatment of Friedrich's Ataxia based on effective targeting of a therapeutic moiety to mitochondria that can substitute for natural FXN protein activity or rescue one or more phenotypes or symptoms associated with frataxin-deficiency. In some embodiments, the present invention provides a targeted therapeutic comprising a therapeutic moiety, which is a polypeptide having an N-terminus and a C-terminus, a mitochondrial targeting sequence associated with the therapeutic moiety at the N-terminus, and a mitochondrial membrane-penetrating peptide associated with the therapeutic moiety at the C-terminus, wherein the therapeutic moiety is targeted to mitochondria upon cellular entry.

    Abstract translation: 本发明尤其提供了用于治疗弗里德里希共济失调的组合物和方法，其基于可以替代天然FXN蛋白活性或拯救一种或多种与frataxin缺陷相关的表型或症状的线粒体的治疗性部分的有效靶向。 在一些实施方案中，本发明提供靶向治疗剂，其包含治疗部分，其为具有N末端和C末端的多肽，与N末端的治疗部分相关的线粒体靶向序列，以及线粒体膜 与C-末端的治疗部分相关的穿透肽，其中治疗部分在细胞进入时靶向线粒体。

    In vivo production and delivery of erythropoietin or insulinotropin for gene therapy
    9.
    发明申请
    In vivo production and delivery of erythropoietin or insulinotropin for gene therapy 有权
    红细胞生成素或促胰岛素的体内产生和递送用于基因治疗

    公开(公告)号：US20050136041A1

    公开(公告)日：2005-06-23

    申请号：US10885899

    申请日：2004-07-07

    Applicant: Richard Selden ,  Douglas Treco ,  Michael Heartlein

    Inventor： Richard Selden ,  Douglas Treco ,  Michael Heartlein

    IPC: A61K31/70 ,  A61K35/36 ,  A61K38/22 ,  A61K38/27 ,  A61K47/48 ,  A61K48/00 ,  A61P3/06 ,  A61P3/10 ,  A61P5/06 ,  A61P5/18 ,  A61P7/02 ,  A61P7/04 ,  A61P37/00 ,  A61P37/02 ,  A61P39/06 ,  A61P43/00 ,  C07K14/505 ,  C07K14/605 ,  C07K14/61 ,  C12N5/077 ,  C12N5/10 ,  C12N15/09 ,  C12N15/85 ,  C12N15/90 ,  C12N5/08

    CPC classification number: C12N15/907 ,  A61K47/6901 ,  A61K48/00 ,  C07K14/505 ,  C07K14/605 ,  C07K14/61 ,  C07K2319/02 ,  C12N15/85 ,  C12N2510/02 ,  C12N2800/108 ,  C12N2830/002 ,  C12N2830/85 ,  C12N2840/20 ,  C12N2840/44

    Abstract: The present invention relates to transfected primary and secondary somatic cells of vertebrate origin, particularly mammalian origin, transfected with exogenous genetic material (DNA) which encodes erythropoietin or an insulinotropin [e.g., derivatives of glucagon-like peptide 1 (GLP-1)], methods by which primary and secondary cells are transfected to include exogenous genetic material encoding erythropoietin or an insulinotropin, methods of producing clonal cell strains or heterogenous cell strains which express erythropoietin or an insulinotropin, methods of gene therapy in which the transfected primary or secondary cells are used, and methods of producing antibodies using the transfected primary or secondary cells. The present invention also includes primary and secondary somatic cells, such as fibroblasts, keratinocytes, epithelial cells, endothelial cells, glial cells, neural cells, formed elements of the blood, muscle cells, other somatic cells, which can be cultured and somatic cell precursors, which have been transfected with exogenous DNA encoding EPO or an insulinotropin, which is stably integrated into their genomes or is expressed in the cells episomally.

    Abstract translation: 本发明涉及用编码促红细胞生成素或促胰岛素（例如，胰高血糖素样肽1（GLP-1）的衍生物）的外源遗传物质（DNA）转染的脊椎动物来源的转基因和次要体细胞，特别是哺乳动物来源， 转染初级细胞和次级细胞的方法包括编码促红细胞生成素或促胰岛激素的外源性遗传物质，产生克隆细胞株或表达促红细胞生成素或促胰岛激素的异源细胞株的方法，转染的原代细胞或次要细胞是基因治疗方法 使用，以及使用转染的一次或二次细胞产生抗体的方法。 本发明还包括初级和次级体细胞，例如成纤维细胞，角质形成细胞，上皮细胞，内皮细胞，神经胶质细胞，神经细胞，形成的血液元素，肌肉细胞，可培养的其他体细胞和体细胞前体 其已经用编码EPO或促胰岛素的外源DNA转染，其稳定整合到其基因组中或者在细胞中以表观形式表达。

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