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    NON-DISRUPTIVE GENE TARGETING
    1.
    发明申请
    NON-DISRUPTIVE GENE TARGETING 审中-公开
    非破坏性基因定位

    公开(公告)号：WO2013158309A2

    公开(公告)日：2013-10-24

    申请号：PCT/US2013/032443

    申请日：2013-03-15

    Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY ,  BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM

    Inventor： KAY, Mark A. ,  PORTEUS, Matthew ,  BARKER, Jenny ,  CHECKETTS, Josh ,  VOIT, Richard ,  BARZEL, Adi

    IPC: C12N15/85

    CPC classification number: C12N15/907 ,  A01K2207/12 ,  C07K2319/80 ,  C07K2319/81 ,  C12N9/22 ,  C12N15/85 ,  C12N2840/20

    Abstract: Compositions and methods are provided for integrating one or more genes of interest into cellular DNA without substantially disrupting the expression of the gene at the locus of integration, i.e., the target locus. These compositions and methods are useful in any in vitro or in vivo application in which it is desirable to express a gene of interest in the same spatially and temporally restricted pattern as that of a gene at a target locus while maintaining the expression of the gene at the target locus, for example, to treat disease, in the production of genetically modified organisms in agriculture, in the large scale production of proteins by cells for therapeutic, diagnostic, or research purposes, in the induction of iPS cells for therapeutic, diagnostic, or research purposes, in biological research, etc. Reagents, devices and kits thereof that find use in practicing the subject methods are also provided.

    Abstract translation: 提供组合物和方法用于将一种或多种感兴趣的基因整合到细胞DNA中，而基本上不破坏整合位点即靶基因座上基因的表达。 这些组合物和方法可用于任何体外或体内应用，其中期望在与靶基因的基因相同的空间和时间限制性模式下表达感兴趣的基因，同时将基因的表达保持在 目标基因座，例如在农业中生产转基因生物，在用于治疗，诊断或研究目的的细胞大规模生产蛋白质中治疗疾病，诱导iPS细胞用于治疗，诊断， 或研究目的，生物研究等。还提供了在实践主题方法中使用的试剂，装置和试剂盒。

    ADENO-ASSOCIATED VIRAL VECTORS FOR CROSSING THE HUMAN BLOOD BRAIN BARRIER
    2.
    发明申请
    ADENO-ASSOCIATED VIRAL VECTORS FOR CROSSING THE HUMAN BLOOD BRAIN BARRIER 审中-公开

    公开(公告)号：WO2021041489A1

    公开(公告)日：2021-03-04

    申请号：PCT/US2020/047917

    申请日：2020-08-26

    Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY

    Inventor： KAY, Mark A. ,  SONG, Ren

    IPC: C12N15/86

    Abstract: The present disclosure provides variant adeno-associated virus (AAV) capsid polypeptides that provide an AAV particle with the ability to traverse the human blood brain barrier (BBB) and transduce cells of the CNS. In some embodiment, a subject variant AAV capsid protein includes an amino acid sequence having 95% or more sequence identity (e.g., 96% or more, 97% or more, 98% or more, 99% or more, 99.5% or more, or 100% sequence identity) with the amino acid sequence set forth in any one of SEQ ID NOs: 1-27. Also provided are nucleic acids, AAV vectors, viral particles, cells, kits, and methods.

    MINI-INTRONIC PLASMID VECTORS
    4.
    发明申请
    MINI-INTRONIC PLASMID VECTORS 审中-公开

    公开(公告)号：WO2013119371A2

    公开(公告)日：2013-08-15

    申请号：PCT/US2013/021984

    申请日：2013-01-17

    Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY

    Inventor： LU, Jiamiao ,  KAY, Mark A.

    IPC: C12N15/85

    CPC classification number: C12N15/85 ,  C12N2830/42 ,  C12N2840/445

    Abstract: Compositions and methods are provided for achieving persistent, high level expression of transgenes in vitro and in vivo. Aspects of the invention include vectors comprising an intronic cassette that comprises plasmid elements, and methods that rely on the use of vectors comprising an intronic cassette that comprises plasmid elements. These compositions and methods find use in many applications, including therapeutic applications such as in gene therapy; synthesis applications such as in the synthesis of peptides, proteins, and RNAs, e.g. for research or therapeutic purposes; and research applications, such as in the production of transgenic cells and animals. In addition, reagents, devices and kits thereof that find use in making the subject compositions and practicing the subject methods are provided.

    Abstract translation: 提供组合物和方法用于在体外和体内实现转基因的持续高水平表达。 本发明的方面包括含有包含质粒元件的内含子盒的载体，以及依赖于使用包含质粒元件的内含子盒的载体的方法。 这些组合物和方法可用于许多应用，包括治疗应用如基因治疗; 合成应用例如在肽，蛋白质和RNA的合成中，例如。 用于研究或治疗目的; 和研究应用，如转基因细胞和动物的生产。 此外，提供了用于制备主题组合物和实践本发明方法的试剂，装置和试剂盒。

    METHODS AND APPARATUS FOR NUCLEIC ACID SYNTHESIS USING OLIGO-TEMPLATED POLYMERIZATION
    6.
    发明申请
    METHODS AND APPARATUS FOR NUCLEIC ACID SYNTHESIS USING OLIGO-TEMPLATED POLYMERIZATION 审中-公开
    使用低聚合聚合的核酸合成方法和装置

    公开(公告)号：WO2014021938A1

    公开(公告)日：2014-02-06

    申请号：PCT/US2013/027536

    申请日：2013-02-24

    Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY

    Inventor： KAY, Mark A. ,  BARZEL, Adi

    IPC: C12N15/11 ,  C07H21/04 ,  C12Q1/68 ,  C12M1/38

    CPC classification number: B01L7/525 ,  B01L3/50851 ,  B01L7/52 ,  B01L2200/0647 ,  B01L2200/18 ,  B01L2300/0829 ,  C07H21/04 ,  C12Q1/6834 ,  C12Q1/6844 ,  C12Q2533/101 ,  C12Q2537/155 ,  C12Q2537/157 ,  C12Q2565/518

    Abstract: A method and apparatus for synthesizing nucleic acid (NA) by sequential addition of nucleotide triphosphates to a single stranded NA molecule (growing-chain) is described. The growing-chain is annealed to an oligonucleotide, wherein the 3' end of the oligonucleotide is complementary to the 3' end of the growing-chain and wherein the protruding 5' end of the oligonucleotide has the correct sequence to serve as a template for the growing-chain to produce a NA product. The growing-chain is then brought into contact with a polymerase in the presence of one or more nucleotide triphosphates under conditions permitting the polymerase to catalyze addition of one or more nucleotide triphosphates to the 3' end of the growing-chain using the annealed oligonucleotide as a template. After polymerization has occurred, the annealed growing-chain and oligonucleotide are denatured so that the steps can be repeated until the NA product of desired length is synthesized.

    Abstract translation: 描述了通过向单链NA分子（生长链）顺序加入核苷酸三磷酸合成核酸（NA）的方法和装置。 生长链与寡核苷酸退火，其中寡核苷酸的3'末端与生长链的3'末端互补，其中寡核苷酸的突出的5'末端具有正确的序列，用作 生产NA产品的生长链。 然后在一个或多个核苷酸三磷酸存在的情况下使生长链与聚合酶接触，条件是允许聚合酶催化一个或多个核苷酸三磷酸酯加入生长链的3'末端，使用退火寡核苷酸作为 一个模板 发生聚合后，退火的生长链和寡核苷酸被变性，使得可以重复这些步骤，直到合成所需长度的NA产物。

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