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    ADENO-ASSOCIATED VIRUS VECTOR VARIANTS FOR HIGH EFFICIENCY GENOME EDITING AND METHODS THEREOF
    15.
    发明公开
    ADENO-ASSOCIATED VIRUS VECTOR VARIANTS FOR HIGH EFFICIENCY GENOME EDITING AND METHODS THEREOF 审中-公开
    用于高效基因组编辑的腺伴随病毒载体变体及其方法

    公开(公告)号：EP3198018A1

    公开(公告)日：2017-08-02

    申请号：EP15775884.8

    申请日：2015-09-23

    申请人： City of Hope

    发明人： CHATTERJEE, Saswati ,  SMITH, Laura ,  WONG, Kamehameha

    IPC分类号： C12N15/864 ,  C12N15/90 ,  A61K48/00

    CPC分类号： C12N15/907 ,  A61K48/00 ,  A61K48/0008 ,  A61K48/005 ,  C07K14/005 ,  C12N7/00 ,  C12N15/102 ,  C12N15/86 ,  C12N2710/16622 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14152 ,  C12N2750/14171 ,  C12N2800/70 ,  C12N2840/44

    摘要： Adeno-associated virus (AAV) Clade F vectors or AAV vector variants (relative to AAV9) for precise editing of the genome of a cell and methods and kits thereof are provided. Targeted genome editing using the AAV Clade F vectors or AAV vector variants provided herein occurred at frequencies that were shown to be 1,000 to 100,000 fold more efficient than has previously been reported. Also provided are methods of treating a disease or disorder in a subject by editing the genome of a cell of the subject via transducing the cell with an AAV Clade F vector or AAV vector variant as described herein and further transplanting the transduced cell into the subject to treat the disease or disorder of the subject. Also provided herein are methods of treating a disease or disorder in a subject by in vivo genome editing by directly administering the AAV Clade F vector or AAV vector variant as described herein to the subject.

    摘要翻译： 提供了用于精确编辑细胞基因组的腺伴随病毒（AAV）进化枝F载体或AAV载体变体（相对于AAV9）以及其方法和试剂盒。 使用本文提供的AAV Clade F载体或AAV载体变体的靶向基因组编辑发生在比之前已经报道的频率显示出更高的1,000至100,000倍的频率。 还提供了通过如本文所述通过用AAV进化枝F载体或AAV载体变体转导细胞来编辑受试者的细胞的基因组并且进一步将转导的细胞移植入受试者以治疗受试者中的疾病或病症的方法， 治疗受试者的疾病或病症。 本文还提供通过体内基因组编辑通过将本文所述的AAV Clade F载体或AAV载体变体直接施用于受试者来治疗受试者的疾病或病症的方法。

    GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS
    16.
    发明公开
    GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS 审中-公开
    GENTHERAPIEFÜRAMYOTROPHE LATERALSKLEROSE UND ANDERERüCKENMARKSERKRANKUNGEN

    公开(公告)号：EP3146982A1

    公开(公告)日：2017-03-29

    申请号：EP16184966.6

    申请日：2007-10-03

    申请人： Genzyme Corporation

    发明人： O'Riordan, Catherine ,  Wadsworth, Samuel

    IPC分类号： A61K48/00 ,  C12N15/864 ,  A61P21/00

    CPC分类号： C12N15/86 ,  A61K38/1709 ,  A61K48/005 ,  A61K48/0075 ,  C07K2319/00 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/6027 ,  C12N2830/002 ,  C12N2830/15

    摘要： This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant viral vector containing the transgene to the spinal cord. The viral vector delivers the transgene which expresses the encoded recombinant viral gene product. The viral gene product comprises HiF1 -alpha. Also provided are compositions for delivery of a transgene product to a subject's spinal cord.

    摘要翻译： 本公开提供了用于治疗影响受试者的运动功能和控制的疾病或损伤的方法和组合物。 一方面，本发明通过向脊髓施用含有转基因的重组病毒载体将转基因产物递送至受试者的脊髓。 病毒载体递送表达编码的重组病毒基因产物的转基因。 病毒基因产物包含HiF1-α。 还提供了用于将转基因产物递送至受试者的脊髓的组合物。