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    GENE THERAPY FOR OCULAR IMPROVEMENT
    1.
    发明申请
    GENE THERAPY FOR OCULAR IMPROVEMENT 审中-公开

    公开(公告)号：WO2019108570A2

    公开(公告)日：2019-06-06

    申请号：PCT/US2018/062712

    申请日：2018-11-28

    Applicant: COPERNICUS THERAPEUTICS, INC. ,  CLEARSIDE BIOMEDICAL

    Inventor： TARABORELLI, Donna ,  YOO, Jesse ,  NORONHA, Glenn ,  COOPER, Mark J. ,  MOEN, Robert C. ,  WHITE, Daniel

    CPC classification number: A61K48/0075 ,  A61K48/0008

    Abstract: Targeted non-surgical administration of a nucleic acid formulation to the suprachoroidal space (SCS) of the eye of a human subject permits effective treatment of ocular disorders, including posterior ocular or choroidal maladies. In one embodiment, the method comprises inserting a hollow microneedle into the eye at an insertion site and infusing a nucleic acid formulation through the inserted microneedle and into the suprachoroidal space of the eye. The infused nucleic acid formulation flows within the suprachoroidal space away from the insertion site. In one embodiment, the fluid nucleic acid formulation comprises nucleic acid nanoparticles consisting of one molecule of nucleic acid.

    COMPOSITIONS AND METHODS FOR ADENO-ASSOCIATED VIRUS MEDIATED GENE EXPRESSION IN MYOFIBROBLAST-LIKE CELLS
    2.
    发明申请
    COMPOSITIONS AND METHODS FOR ADENO-ASSOCIATED VIRUS MEDIATED GENE EXPRESSION IN MYOFIBROBLAST-LIKE CELLS 审中-公开
    腺样结合病毒介导的类软骨细胞基因表达的组合物和方法

    公开(公告)号：WO2017151717A1

    公开(公告)日：2017-09-08

    申请号：PCT/US2017/020113

    申请日：2017-03-01

    Applicant: FRENCH, Brent A.

    Inventor： FRENCH, Brent A.

    IPC: A61K35/34 ,  A61K48/00 ,  A61P9/10 ,  C12N15/63

    CPC classification number: A61K48/0058 ,  A61K35/34 ,  A61K48/0008 ,  A61K48/0066 ,  A61P9/10 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/008

    Abstract: The present application discloses a recombinant AAV vector comprising a promoter that targets gene expression to myofibroblast-like lineage of cells in the heart. The present application, also discloses the preparation and use of AAV expression cassettes using a modified periostin promoter that successfully drives gene expression in cardiac myofibroblast-like cells. The present invention encompasses compositions and methods useful for treating myocardial infarction. Further comprising compositions and methods for preparing and using AAV vectors for targeting cells and inducing gene expression. The compositions and methods of the invention are useful for efficiently targeting cardiac myofibroblasts following a cardiac injury, disease, or disorder. Further comprising a kit for effecting alleviation of the various diseases or disorders recited herein.

    Abstract translation: 本申请公开了包含靶向基因表达的启动子的重组AAV载体，其在心脏中的细胞的肌成纤维细胞样谱系中。 本申请还公开了使用修饰的骨膜素启动子制备和使用AAV表达盒，所述修饰的骨膜素启动子成功驱动心肌成肌细胞样细胞中的基因表达。 本发明包括可用于治疗心肌梗塞的组合物和方法。 进一步包括用于制备和使用AAV载体用于靶向细胞和诱导基因表达的组合物和方法。 本发明的组合物和方法可用于在心脏损伤，疾病或病症之后有效地靶向心肌成纤维细胞。 还包括用于减轻本文所述各种疾病或病症的试剂盒。

    COMPOSITIONS COMPRISING AAV EXPRESSING DUAL ANTIBODY CONSTRUCTS AND USES THEREOF
    5.
    发明申请
    COMPOSITIONS COMPRISING AAV EXPRESSING DUAL ANTIBODY CONSTRUCTS AND USES THEREOF 审中-公开
    包含AAV表达双抗体构建体的组合物及其用途

    公开(公告)号：WO2015175639A1

    公开(公告)日：2015-11-19

    申请号：PCT/US2015/030533

    申请日：2015-05-13

    Applicant: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

    Inventor： WILSON, James, M. ,  TRETIAKOVA, Anna

    IPC: A61P31/12 ,  C12N15/861

    CPC classification number: C07K16/1018 ,  A61K48/0008 ,  A61K2039/507 ,  C07K2317/14 ,  C07K2317/31 ,  C07K2317/51 ,  C07K2317/515 ,  C07K2317/52 ,  C07K2317/622 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14143 ,  C12N2830/205 ,  C12N2830/30 ,  C12N2830/50 ,  C12N2840/203

    Abstract: A recombinant adeno-associated virus (AAV) having an AAV capsid and packaged therein a heterologous nucleic acid which expresses two functional antibody constructs in a cell is described. Also described are antibodies comprising a heavy chain and a light chain from a heterologous antibody. In one embodiment, the antibodies are co-expressed from a vector containing: a first expression cassette which encodes at least a first open reading frame (ORF) for a first immunoglobulin under the control of regulatory control sequences which direct expression thereof; and a second expression cassette which comprises a second ORF, a linker, and a third ORF under the control of regulatory control sequences which direct expression thereof, wherein the second and third ORF for a second and third immunoglobulin construct. The vector co-expressing these two antibody constructs is in one embodiment an AAV, in which the 5 ' and 3 ' ITRs flank the expression cassettes and regulatory sequences.

    Abstract translation: 描述了具有AAV衣壳并包装有在细胞中表达两种功能性抗体构建体的异源核酸的重组腺相关病毒（AAV）。 还描述了包含来自异源抗体的重链和轻链的抗体。 在一个实施方案中，抗体由含有下列基因的载体共表达：含有第一表达盒，该第一表达盒在其指导其表达的调控控制序列的控制下编码第一免疫球蛋白的至少第一开放阅读框（ORF） 以及第二表达盒，其包含第二ORF，接头和第三ORF，所述第二ORF在其指导其表达的调节控制序列的控制下，其中所述第二和第三免疫球蛋白构建体的第二和第三ORF。 在一个实施方案中，共表达这两种抗体构建体的载体是AAV，其中5'和3'ITR位于表达盒和调节序列的两侧。

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