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13 条记录 (耗时 0.018 秒)

    IMPROVING THE EFFICACY OF MUSCLE-TARGETED GENE THERAPY
    1.
    发明申请
    IMPROVING THE EFFICACY OF MUSCLE-TARGETED GENE THERAPY 审中-公开

    公开(公告)号：WO2023006890A1

    公开(公告)日：2023-02-02

    申请号：PCT/EP2022/071225

    申请日：2022-07-28

    申请人： VRIJE UNIVERSITEIT BRUSSEL

    发明人： VANDENDRIESSCHE, Thierry ,  CHUAH, Lay Khim

    IPC分类号： A61K48/00 ,  C07K14/47 ,  C12N15/86

    摘要： The present invention relates to nucleic acid regulatory elements that are able to enhance diaphragm-specific expression of genes, in particular expression in diaphragm as such, or in combination with expression in cardiac muscle and/or skeletal muscle, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy, more particularly muscle and/or heart tissue directed gene therapy, and for vaccination purposes.

    ENDOTHELIUM-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF
    3.
    发明申请
    ENDOTHELIUM-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF 审中-公开
    内皮特异性核酸调节元件及其使用方法和用途

    公开(公告)号：WO2017109039A1

    公开(公告)日：2017-06-29

    申请号：PCT/EP2016/082314

    申请日：2016-12-22

    申请人： VRIJE UNIVERSITEIT BRUSSEL

    发明人： CHUAH, Marinee ,  VANDENDRIESSCHE, Thierry

    IPC分类号： A61K48/00 ,  C12N15/86 ,  C12N15/861 ,  C12N15/864

    CPC分类号： C12N15/86 ,  C12N2740/15043 ,  C12N2750/14143 ,  C12N2830/008

    摘要： The present invention relates to nucleic acid regulatory elements that are able to enhance endothelial cell-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy, more particularly endothelial cell-directed gene therapy, and for vaccination purposes.

    摘要翻译： 本发明涉及能够增强基因的内皮细胞特异性表达的核酸调节元件，使用这些调节元件的方法以及这些元件的用途。 还公开了含有这些核酸调控元件的表达盒和载体。 本发明对于使用基因疗法，更特别是内皮细胞导向基因疗法和用于接种目的的应用特别有用。

    CARDIOMYOCYTE-DERIVED NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF
    4.
    发明申请
    CARDIOMYOCYTE-DERIVED NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF 审中-公开

    公开(公告)号：WO2022136388A1

    公开(公告)日：2022-06-30

    申请号：PCT/EP2021/087020

    申请日：2021-12-21

    申请人： VRIJE UNIVERSITEIT BRUSSEL

    发明人： CHUAH, Lay Khim ,  VANDENDRIESSCHE, Thierry

    IPC分类号： C12N15/11 ,  A61K48/00

    摘要： The present invention relates to nucleic acid regulatory elements that are able to enhance heart- and/or muscle-targeted expression of genes, in particular heart- and muscle-targeted gene expression, more particularly gene expression in cardiomyocytes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy, more particularly heart- and/or muscle-directed gene therapy, e.g. for the treatment of cardiovascular diseases and disorders and muscle disorders, as well as other diseases and disorders that may benefit from high transgene expression in heart and/or muscle cells or tissue, and for vaccination purposes.

    DIAPHRAGM-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF
    5.
    发明申请
    DIAPHRAGM-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF 审中-公开

    公开(公告)号：WO2018178067A1

    公开(公告)日：2018-10-04

    申请号：PCT/EP2018/057753

    申请日：2018-03-27

    申请人： VRIJE UNIVERSITEIT BRUSSEL

    发明人： CHUAH, Lay Khim ,  VANDENDRIESSCHE, Thierry ,  TULALAMBA, Warut

    IPC分类号： A61K48/00 ,  C12N15/861

    摘要： The present invention relates to nucleic acid regulatory elements that are able to enhance diaphragm-specific expression of genes, in particular expression in diaphragm as such, or in combination with expression in cardiac muscle and/or skeletal muscle, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy, more particularly diaphragm-directed gene therapy, and for vaccination purposes.

    MUSCLE-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF
    6.
    发明申请
    MUSCLE-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF 审中-公开

    公开(公告)号：WO2021165353A1

    公开(公告)日：2021-08-26

    申请号：PCT/EP2021/053945

    申请日：2021-02-18

    申请人： VRIJE UNIVERSITEIT BRUSSEL

    发明人： VANDENDRIESSCHE, Thierry ,  CHUAH, Lay Khim

    IPC分类号： C07K14/47 ,  A61K48/00 ,  C12N15/86

    摘要： The present invention relates to nucleic acid regulatory elements that are able to enhance muscle-specific expression of genes, in particular expression in muscle cells and/or tissues such as in diaphragm, smooth muscle, heart and/or skeletal muscle, comprising at least two diaphragm-specific regulatory elements and a heart- and skeletal muscle-specific regulatory element. The present invention further relates to expression cassettes and vectors containing these nucleic acid regulatory elements, as well as uses thereof. The present invention is particularly useful for applications using gene therapy of muscle-related disorders, more particularly diaphragm, heart and/or skeletal muscle-directed gene therapy, and for vaccination purposes.

    NEW TOOLS FOR IMPROVING GENE THERAPY AND USE THEREOF
    7.
    发明申请
    NEW TOOLS FOR IMPROVING GENE THERAPY AND USE THEREOF 审中-公开

    公开(公告)号：WO2020084161A1

    公开(公告)日：2020-04-30

    申请号：PCT/EP2019/079351

    申请日：2019-10-28

    申请人： VRIJE UNIVERSITEIT BRUSSEL

    发明人： VANDENDRIESSCHE, Thierry ,  CHUAH, Lay Khim

    IPC分类号： A61K38/38 ,  C07K14/745

    摘要： The present invention relates to a nucleic acid molecule encoding human albumin for increasing the levels and/or activity of a protein or polypeptide encoded by a transgene, comprising a sequence defined by SEQ ID NO: 14 or a sequence having at least 80% sequence identity to said sequence, its use in nucleic acid expression cassettes and vectors containing liver-specific regulatory elements and codon- optimized factor IX, factor VIII, factor VII or factor VIla transgenes, methods employing these expression cassettes and vectors and uses thereof. The present invention is particularly useful for applications using liver-directed gene therapy, in particular for the treatment of hemophilia A, hemophilia B or factor VII deficiency.

    GENETIC CORRECTION OF MYOTONIC DYSTROPHY TYPE 1
    8.
    发明申请
    GENETIC CORRECTION OF MYOTONIC DYSTROPHY TYPE 1 审中-公开
    MYOTONIC DYSTROPYY型1的遗传修正

    公开(公告)号：WO2015173436A1

    公开(公告)日：2015-11-19

    申请号：PCT/EP2015/060922

    申请日：2015-05-18

    申请人： VRIJE UNIVERSITEIT BRUSSEL

    发明人： VANDENDRIESSCHE, Thierry ,  CHUAH, Marinee

    IPC分类号： A61K48/00 ,  C12N5/0775 ,  C12N5/074 ,  C12N9/12 ,  C12N9/22 ,  C12N15/90 ,  A61K35/34 ,  C12N5/077 ,  A61K39/00

    CPC分类号： C12N5/0696 ,  A61K35/34 ,  A61K48/00 ,  A61K2039/515 ,  A61K2039/53 ,  C12N5/0658 ,  C12N9/1205 ,  C12N15/113 ,  C12N15/907 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2800/80 ,  C12N2810/10

    摘要： The invention relates to polynucleotides suitable for reducing or eliminating the expression of expanded repeat RNA (CUGexp) of the dystrophy myotonic-protein kinase (DMPK) gene in a cell of a DM-1 patient. The polynucleotides are a combination of a polynucleotide for a site specific nuclease targeting the dystrophy myotonic- protein kinase (DMPK) gene locus, and a donor polynucleotide having 5' and 3' regions which are homologous with the sequence of DMPK gene which flank the target site of the nuclease. The invention further relate to in vivo and in vitro methods to reduce or eliminate CTG repeats in the DMPK gene. The invention further relates to the medical use of polynucleotides and cells for treating DM-1 patient.

    摘要翻译： 本发明涉及适合于减少或消除DM-1患者细胞中营养不良肌强直蛋白激酶（DMPK）基因的扩增重复RNA（CUGexp）的表达的多核苷酸。 多核苷酸是针对营养不良肌强直蛋白激酶（DMPK）基因座的位点特异性核酸酶的多核苷酸和与靶向侧翼的DMPK基因的序列同源的5'和3'区域的供体多核苷酸的组合 核酸酶的位点。 本发明还涉及减少或消除DMPK基因中的CTG重复的体内和体外方法。 本发明还涉及用于治疗DM-1患者的多核苷酸和细胞的医学用途。