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1.发明公开COMPOSITIONS FOR LINKING DNA-BINDING DOMAINS AND CLEAVAGE DOMAINS 审中-公开ZUSAMMENSETZUNGEN ZUR VERNETZUNG VON DNA-BINDUNGSDOMÄNENUND-SPALTUNGSDOMÄNEN
公开(公告)号：EP3039136A1
公开(公告)日：2016-07-06
申请号：EP14840079.9
申请日：2014-08-28
发明人： PASCHON, David , ZHANG, Lei
IPC分类号： C12N9/16
摘要： Disclosed herein are compositions for linking DNA-binding domains and cleavage domains to form nucleases, for example nucleases with altered target site separation (gap) preferences as compared to conventional linkers. Also described are fusion proteins comprising these linkers. The disclosure also provides methods of using these fusion proteins and compositions thereof for targeted cleavage of cellular chromatin in a region of interest and/or homologous recombination at a predetermined region of interest in cells. Thus, in one aspect, described herein are amino acid sequences linking a DNA-binding domain to a cleavage domain. The linker may replace one or more residues of the DNA-binding domain and/or cleavage domain.
摘要翻译： 本文公开了用于连接DNA结合结构域和切割结构域（或切割半结构域）以形成非天然存在的核酸酶的组合物。 还描述了制备和使用包含这些接头的组合物的方法。
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2.发明授权METHODS AND COMPOSITIONS FOR ALTERATION OF A CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE 有权用于改善的囊肿的方法和组合物纤维化跨膜LINE调节基因
公开(公告)号：EP2737063B1
公开(公告)日：2016-06-01
申请号：EP12817382.0
申请日：2012-07-25
CPC分类号： A61K48/00 , A61K38/00 , A61K48/005 , C07K2319/81 , C12N5/0662 , C12N9/22 , C12N15/85 , C12N15/907 , C12N2015/8536 , C12N2800/30 , C12N2800/80
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公开(公告)号：EP2571512A1
公开(公告)日：2013-03-27
申请号：EP11783865.6
申请日：2011-05-17
发明人： GREGORY, Philip, D. , MILLER, Jeffrey, C. , PASCHON, David , REBAR, Edward, J. , TAN, Siyuan , URNOV, Fyodor , ZHANG, Lei
IPC分类号： A61K38/16
CPC分类号： C12N15/907 , A61K38/00 , C07K14/195 , C07K2319/80 , C12N9/22 , C12N15/52 , C12N15/62 , C12N15/8213 , C12N15/85 , C12Y301/21004
摘要： Disclosed herein are polypeptides, polynucleotides encoding, cells and organisms comprising novel DNA-binding domains, including TALE DNA-binding domains. Also disclosed are methods of using these novel DNA-binding domains for modulation of gene expression and/or genomic editing of endogenous cellular sequences.
摘要翻译： 本文公开了编码包含新的DNA结合结构域（包括TALE DNA结合域）的细胞和生物体的多肽，多核苷酸。 还公开了使用这些新的DNA结合结构域来调节内源细胞序列的基因表达和/或基因组编辑的方法。
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公开(公告)号：EP2393506A1
公开(公告)日：2011-12-14
申请号：EP10738861.3
申请日：2010-02-04
发明人： DENT, Carolyn , LAGANIERE, Josee , MENG, Xiangdong , PASCHON, David , TAN, Siyuan , ZHANG, Lei , ZHANG, Steve, H.
IPC分类号： A61K38/08 , C07K7/06 , A61K31/7088
CPC分类号： C07K14/4702 , A61K38/00
摘要： Disclosed herein are methods and compositions for treating neuropathies by modulating endogenous
NT-3 of
GDNF gene expression.-
公开(公告)号：EP2137310A2
公开(公告)日：2009-12-30
申请号：EP08743239.9
申请日：2008-04-24
CPC分类号： C12N15/907 , C07K14/435 , C07K14/43595 , C07K2319/81 , C12N9/22 , C12N15/1082 , C12N15/113 , C12N2800/10 , C12N2800/30 , C12Y301/21004
摘要： Disclosed herein are methods and compositions for targeted integration of an exogenous sequence into the human PPP1R12C locus, for example, for expression of a polypeptide of interest.
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6.发明公开METHODS AND COMPOSITIONS FOR GENOME ENGINEERING 审中-公开VERFAHREN UND ZUSAMMENSETZUNGENFÜRGENOM-ENGINEERING
公开(公告)号：EP3080274A2
公开(公告)日：2016-10-19
申请号：EP14869719.6
申请日：2014-12-09
摘要： Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins involved in clotting into the genome of a cell for treating conditions including hemophilias.
摘要翻译： 本文公开了用于将编码参与凝血的蛋白质的转基因序列插入细胞的基因组中用于治疗包括血友病的病症的方法和组合物。
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公开(公告)号：EP3080143A1
公开(公告)日：2016-10-19
申请号：EP14869305.4
申请日：2014-12-09
IPC分类号： C07K14/00
CPC分类号： C07K14/47 , A61K48/00 , C07K14/4702 , C07K2319/81 , C12N9/22 , C12N9/644 , C12N15/86 , C12N2750/14133 , C12N2750/14142 , C12N2750/14143
摘要： Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins involved in clotting into the genome of a cell for treating conditions including hemophilias. In one aspect, described herein is a non-naturally occurring zinc-finger protein (ZFP) that binds to a target site in a region of interest (e.g., an albumin gene) in a genome, wherein the ZFP comprises one or more engineered zinc-finger binding domains. In certain embodiments, the zinc finger domain recognizes a target site in an albumin gene. In some embodiments, the zinc finger protein comprises five or six zinc finger domains designated and ordered F1 to F5 or F1 to F6, as shown in a single row of Table 1.
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8.发明公开METHODS AND COMPOSITIONS FOR MODULATING PD1 审中-公开VERFAHREN UND ZUSAMMENSETZUNGEN ZUR PD1-MODULIERUNG
公开(公告)号：EP3034084A1
公开(公告)日：2016-06-22
申请号：EP16150673.8
申请日：2011-11-17
发明人： GREGORY, Philip D. , HOLMES, Michael C. , MENDEL, Matthew C. , MENG, Xiangdong , PASCHON, David , REIK, Andreas , URNOV, Fyodor
IPC分类号： A61K31/7088 , C07K14/435 , C07H21/00 , C12N5/0783 , A61P35/00 , A61P31/12 , C12N9/22 , A61K38/17 , A61K35/17
CPC分类号： A61K38/1709 , A61K35/17 , A61P31/12 , A61P35/00 , C07K2319/71 , C12N9/22
摘要： Disclosed herein are methods and compositions for modulating expression of a PD1 gene.
摘要翻译： 本文公开了调节PD1基因表达的方法和组合物。
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9.发明公开METHODS AND COMPOSITIONS FOR ALTERATION OF A CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE 有权用于改善的囊肿的方法和组合物纤维化跨膜LINE调节基因
公开(公告)号：EP2737063A2
公开(公告)日：2014-06-04
申请号：EP12817382.0
申请日：2012-07-25
IPC分类号： C12N15/00
CPC分类号： A61K48/00 , A61K38/00 , A61K48/005 , C07K2319/81 , C12N5/0662 , C12N9/22 , C12N15/85 , C12N15/907 , C12N2015/8536 , C12N2800/30 , C12N2800/80
摘要： Nucleases and methods of using these nucleases for alteration of a CFTR gene and generation of cells and animal models.
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公开(公告)号：EP2640399A2
公开(公告)日：2013-09-25
申请号：EP11840913.5
申请日：2011-11-17
发明人： GREGORY, Philip D. , HOLMES, Michael C. , MENDEL, Matthew C. , MENG, Xiangdong , PASCHON, David , REIK, Andreas , URNOV, Fyodor
IPC分类号： A61K31/7088 , C07K14/435 , C07H21/00 , C12N5/0783 , A61P35/00 , A61P31/12
CPC分类号： A61K38/1709 , A61K35/17 , A61P31/12 , A61P35/00 , C07K2319/71 , C12N9/22
摘要： Disclosed herein are methods and compositions for modulating expression of a PD1 gene.
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