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    METHODS AND COMPOSITIONS FOR TREATMENT OF A GENETIC CONDITION
    2.
    发明公开
    METHODS AND COMPOSITIONS FOR TREATMENT OF A GENETIC CONDITION 审中-公开
    治疗遗传病症的方法和组合物

    公开(公告)号：EP2997146A2

    公开(公告)日：2016-03-23

    申请号：EP14797155.0

    申请日：2014-05-15

    申请人： Sangamo BioSciences, Inc.

    发明人： CONWAY, Anthony ,  COST, Gregory J. ,  DEKELVER, Russell ,  REBAR, Edward J. ,  REIK, Andreas ,  URNOV, Fyodor ,  WANG, Jianbin ,  ZHANG, H. Steve

    IPC分类号： C12N15/11

    CPC分类号： C12N15/907 ,  C07K2/00 ,  C07K2319/81 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90 ,  C12N15/902 ,  C12N2320/30 ,  C12Y301/21004

    摘要： Methods and compositions comprising single guide RNAs targeted to an endogenous mammalian β globin gene (HBB), gamma globin gene (HBG1), B-cell lymphoma/leukemia 11A (BCL11A) gene, Kruppel-like factor 1 (KLF1) gene, CCR5 gene, CXCR4 gene, PPP1R12C (AAVS1) gene, hypoxanthine phosphoribosyltransferase (HPRT) gene, albumin gene, Factor VIII gene, Factor IX gene, Leucine-rich repeat kinase 2 (LRRK2) gene, Hungtingin (Htt) gene, rhodopsin (RHO) gene, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, surfactant protein B gene (SFTPB), T-cell receptor alpha (TRAC) gene, T-cell receptor beta (TRBC) gene, programmed cell death 1 (PD1) gene, Cytotoxic T-Lymphocyte Antigen 4 (CTLA-4) gene, human leukocyte antigen (HLA) A gene, a LMP7 gene, a Transporter associated with Antigen Processing (TAP) gene, tapasin gene (TAPBP), a class II major histocompatibility complex transactivator (CIITA) gene, a dystrophin gene (DMD), a glucocorticoid receptor gene (GR), IL2RG gene or RFX5 for genetic alteration of cells are provided.

    摘要翻译： 提供了用于细胞遗传改变的方法和组合物。

    METHODS AND COMPOSITIONS FOR TARGETED CLEAVAGE AND RECOMBINATION
    7.
    发明公开
    METHODS AND COMPOSITIONS FOR TARGETED CLEAVAGE AND RECOMBINATION 审中-公开
    用于靶向切割和重组的方法和组合物

    公开(公告)号：EP3222715A1

    公开(公告)日：2017-09-27

    申请号：EP17165881.8

    申请日：2004-08-06

    申请人： Sangamo BioSciences, Inc.

    发明人： PABO, Carl, O. ,  URNOV, Fyodor ,  HOLMES, Michael, C. ,  MILLER, Jeffrey, C. ,  GUSCHIN, Dmitry

    IPC分类号： C12N9/22 ,  C12N15/62 ,  C12N15/01 ,  A61K35/12 ,  C07K14/715 ,  C07K14/435 ,  C07K14/47 ,  C12N9/14 ,  C12N15/85 ,  C12N15/90

    CPC分类号： C12N15/10 ,  C07K14/4702 ,  C07K2319/00 ,  C07K2319/81 ,  C12N9/22 ,  C12N15/902

    摘要： Disclosed herein are methods and compositions for targeted cleavage of a genomic sequence, targeted alteration of a genomic sequence, and targeted recombination between a genomic region and an exogenous polynucleotide homologous to M the genomic region. The compositions include fusion proteins comprising a cleavage domain (or cleavage half-domain) and an engineered zinc finger domain and polynucleotides encoding same. Methods for targeted cleavage include introduction of such fusion proteins, or polynucleotides encoding same, into a cell. Methods for targeted recombination additionally include introduction of an exogenous polynucleotide homologous to a genomic region into cells comprising the disclosed fusion proteins.

    摘要翻译： 本文公开了用于基因组序列的靶向切割，基因组序列的靶向改变以及基因组区域和与基因组区域同源的外源多核苷酸之间的靶向重组的方法和组合物。 该组合物包括含有切割结构域（或切割半结构域）和工程化锌指结构域的融合蛋白以及编码它们的多核苷酸。 用于靶向切割的方法包括将此类融合蛋白或编码它们的多核苷酸引入细胞中。 用于靶向重组的方法另外包括将与基因组区域同源的外源多核苷酸引入包含所公开的融合蛋白的细胞中。