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    Expression of secreted and cell-surface polypeptides
    3.
    发明授权
    Expression of secreted and cell-surface polypeptides 有权
    分泌和细胞表面多肽的表达

    公开(公告)号：US09540657B2

    公开(公告)日：2017-01-10

    申请号：US13901940

    申请日：2013-05-24

    Applicant: California Institute of Technology

    Inventor： Kenneth Yu ,  David Baltimore ,  Lili Yang

    IPC: C12N15/85 ,  C12N15/86 ,  C12N15/90 ,  G01N33/58 ,  G01N33/68

    CPC classification number: C12N15/85 ,  C07K2319/00 ,  C12N15/86 ,  C12N15/907 ,  C12N2740/15043 ,  G01N33/582 ,  G01N33/68

    Abstract: Some embodiments herein provide compositions and methods for expressing secreted and cell-surface-bound polypeptides in a single cell. In some embodiments, secreted and cell-surface polypeptide are produced from a single polynucleotide. The polynucleotide can comprise a sequence (or sequence encoding a polypeptide) that mediates separation of a membrane anchor from the polypeptide. In some embodiments, a desired ratio of secreted to surface-bound polypeptide is obtained by selecting a sequence that mediates a desired level of separation of the membrane anchor from the polypeptide.

    Abstract translation: 本文的一些实施方案提供了用于在单个细胞中表达分泌的和细胞表面结合的多肽的组合物和方法。 在一些实施方案中，分泌的和细胞表面多肽由单个多核苷酸产生。 多核苷酸可以包含介导膜锚定体与多肽分离的序列（或编码多肽的序列）。 在一些实施方案中，分泌到表面结合多肽的期望比例通过选择介导膜锚与多肽分离的期望水平的序列来获得。

    METHOD OF TARGETING GENE DELIVERY USING VIRAL VECTORS
    4.
    发明申请
    METHOD OF TARGETING GENE DELIVERY USING VIRAL VECTORS 有权
    使用病毒载体靶向基因传递的方法

    公开(公告)号：US20160222409A1

    公开(公告)日：2016-08-04

    申请号：US14338947

    申请日：2014-07-23

    Applicant: CALIFORNIA INSTITUTE OF TECHNOLOGY

    Inventor： David Baltimore ,  Pin Wang ,  Lili Yang

    IPC: C12N15/86

    CPC classification number: C12N15/86 ,  A61K2039/505 ,  C07K16/2887 ,  C07K2317/53 ,  C07K2317/622 ,  C07K2319/00 ,  C07K2319/03 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2740/16045 ,  C12N2799/027 ,  C12N2810/80 ,  C12N2810/851 ,  C12N2810/859 ,  Y02A50/386 ,  Y02A50/396

    Abstract: Methods and compositions are provided for delivering a polynucleotide encoding a gene of interest to a target cell using a virus. The virus envelope comprises a cell-specific binding determinant that recognizes and binds to a component on the target cell surface, leading to endocytosis of the virus. A separate fusogenic molecule is also present on the envelope and facilitates delivery of the polynucleotide across the membrane and into the cytosol of the target cell. The methods and related compositions can be used for treating patients having suffering from a wide range of conditions, including infection, such as HIV; cancers, such as non-Hodgkin's lymphoma and breast cancer; and hematological disorders, such as severe combined immunodeficiency.

    Abstract translation: 提供了使用病毒将编码目标基因的多核苷酸递送至靶细胞的方法和组合物。 病毒包膜包含识别和结合靶细胞表面上的组分的细胞特异性结合决定簇，导致病毒的内吞。 单独的融合分子也存在于包膜上并促进多核苷酸穿过膜并进入靶细胞的胞质溶胶。 方法和相关组合物可用于治疗患有广泛病症（包括HIV感染）的患者; 癌症，如非霍奇金淋巴瘤和乳腺癌; 和血液学疾病，如严重的联合免疫缺陷。

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