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    ADENO-ASSOCIATED VIRUS VECTOR VARIANTS FOR HIGH EFFICIENCY GENOME EDITING AND METHODS THEREOF
    2.
    发明申请
    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ADENO相关病毒载体变体及其方法

    公开(公告)号：WO2016049230A1

    公开(公告)日：2016-03-31

    申请号：PCT/US2015/051785

    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： 提供腺相关病毒（AAV）Clade F载体或AAV载体变体（相对于AAV9）用于细胞基因组的精确编辑及其方法和试剂盒。 使用本文提供的AAV进化枝F载体或AAV载体变体的靶向基因组编辑的频率显示出比先前报道的效率高1000至100,000倍的频率。 还提供了通过如本文所述用AAV Clade 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来治疗受试者的疾病或病症的方法，并进一步将转导的细胞移植到受试者中 治疗受试者的疾病或病症。 本文还提供了通过体内基因组编辑来治疗受试者的疾病或病症的方法，其通过直接向受试者施用如本文所述的AAV进化枝F载体或AAV载体变体。

    HSV-1 EPITOPES AND METHODS FOR USING SAME
    5.
    发明申请
    HSV-1 EPITOPES AND METHODS FOR USING SAME 审中-公开
    HSV-1抗生素及其使用方法

    公开(公告)号：WO2012061637A2

    公开(公告)日：2012-05-10

    申请号：PCT/US2011/059214

    申请日：2011-11-03

    申请人： UNIVERSITY OF WASHINGTON ,  KOELLE, David, M. ,  JING, Lichen

    发明人： KOELLE, David, M. ,  JING, Lichen

    IPC分类号： A61K39/245 ,  A61K35/76 ,  C07K14/035 ,  C12N7/01 ,  C12N5/10 ,  C12N15/63 ,  C12P21/04

    CPC分类号： C07K14/035 ,  A61K38/00 ,  A61K39/12 ,  A61K39/245 ,  C07K14/005 ,  C12N2710/16622 ,  C12N2710/16634

    摘要： The invention provides HSV antigens and epitopes that are useful for the prevention and treatment of HSV infection. T-cells having specificity for antigens of the invention have demonstrated cytotoxic activity against cells loaded with virally-encoded peptide epitopes, and in many cases, against cells infected with HSV. The identification of immunogenic antigens responsible for T-cell specificity provides improved anti-viral therapeutic and prophylactic strategies. Compositions containing antigens or polynucleotides encoding antigens of the invention provide effectively targeted vaccines for prevention and treatment of HSV infection.

    摘要翻译： 本发明提供可用于预防和治疗HSV感染的HSV抗原和表位。 对本发明的抗原具有特异性的T细胞已经证明了对负载有病毒编码的肽表位的细胞的细胞毒性活性，并且在许多情况下，针对感染HSV的细胞。 负责T细胞特异性的免疫原性抗原的鉴定提供了改进的抗病毒治疗和预防策略。 含有编码本发明抗原的抗原或多核苷酸的组合物提供有效靶向的疫苗，用于预防和治疗HSV感染。

    MEGANUCLEASE VARIANTS CLEAVING THE GENOME OF A PATHOGENIC NON-INTEGRATING VIRUS AND USES THEREOF
    8.
    发明申请
    MEGANUCLEASE VARIANTS CLEAVING THE GENOME OF A PATHOGENIC NON-INTEGRATING VIRUS AND USES THEREOF 审中-公开
    破坏病原性非整合病毒的基因组及其用途的大麦芽孢杆菌变种

    公开(公告)号：WO2010136981A3

    公开(公告)日：2011-04-28

    申请号：PCT/IB2010052340

    申请日：2010-05-26

    申请人： CELLECTIS ,  CHOULIKA ANDRE ,  CEDRONE FREDERIC ,  SMITH JULIANNE

    发明人： CHOULIKA ANDRE ,  CEDRONE FREDERIC ,  SMITH JULIANNE

    IPC分类号： C12N9/22 ,  A61K38/46 ,  A61P31/22 ,  C12N15/00

    CPC分类号： C12N9/22 ,  A61K38/00 ,  C12N2710/16622 ,  C12N2730/10122

    摘要： An I-CreI variant, wherein at least one of the two l-Crel monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the genome of a non- integrating virus, inparticular herpes simplex virus (HSV) or Hepatitis B virus (HBV) for use in genome engineering and for in vivo and ex vivo (gene cell therapy) genome therapy as well as the treatment of a virus infection.

    摘要翻译： I-CreI变体，其中两个I-CreI单体中的至少一个具有至少两个取代，位于位于I-CreI的位置26至40和44至77的LAGLIDADG核心结构域的两个功能亚结构域中的每一个中的一个 所述变体能够从非整合病毒，特异性单纯疱疹病毒（HSV）或乙型肝炎病毒（HBV）的基因组中切割DNA靶序列，用于基因组工程和体内和离体（基因细胞 治疗）基因组治疗以及病毒感染的治疗。

    PROCESS, VECTORS AND ENGINEERED CELL LINES FOR ENHANCED LARGE-SCALE TRANSFECTION
    10.
    发明申请
    PROCESS, VECTORS AND ENGINEERED CELL LINES FOR ENHANCED LARGE-SCALE TRANSFECTION 审中-公开
    过程，向量和工程细胞系，用于增强大规模转移

    公开(公告)号：WO2009137911A1

    公开(公告)日：2009-11-19

    申请号：PCT/CA2009/000263

    申请日：2009-03-09

    申请人： NATIONAL RESEARCH COUNSIL OF CANADA ,  DUROCHER, Yves ,  LOIGNON, Martin

    发明人： DUROCHER, Yves ,  LOIGNON, Martin

    IPC分类号： C12N15/85 ,  C07K19/00 ,  C07K14/05 ,  C07K14/035

    CPC分类号： C12N15/85 ,  C07K14/005 ,  C07K2319/71 ,  C12N2710/16222 ,  C12N2710/16622 ,  C12N2800/108 ,  C12N2800/40

    摘要： Processes vectors and engineered cell lines for large-scale transfection and protein production in mammalian cells, especially Chinese Hamster Ovary (CHO) cells are described in which transfection efficiencies are realized through the use of a single vector system, the use of functional oriP sequences in all plasmids, the use of codon- optimized Epstein-Barr virus nuclear antigen-1 (EBNA1 ) constructs the use of a fusion protein between a truncated Epstein-Barr virus nuclear antigenen-1c (EBNA1c) protein and a herpes simplex virus protein VP16, the use of a 40 kDa fully deacetylated poly(ethylenimine) as a transfection reagent, the use of co-expression of a fibroblast growth factor (FGF) and/or the use of protein kinase B to potentiate heterologous gene expression enhancement by valproic acid (VPA).

    摘要翻译： 描述了用于哺乳动物细胞，特别是中国仓鼠卵巢（CHO）细胞中大规模转染和蛋白质生产的载体和工程细胞系，其中通过使用单一载体系统实现转染效率，使用功能性oriP序列 所有质粒，使用密码子优化的爱泼斯坦 - 巴尔病毒核抗原-1（EBNA1）构建了截短的爱泼斯坦 - 巴尔病毒核抗原-1（EBNA1c）蛋白和单纯疱疹病毒蛋白VP16之间的融合蛋白的使用， 使用40kDa完全脱乙酰化的聚（亚乙基亚胺）作为转染试剂，使用共表达成纤维细胞生长因子（FGF）和/或使用蛋白激酶B来加强丙戊酸的异源基因表达增强（ VPA）。