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公开(公告)号：US11884930B2
公开(公告)日：2024-01-30
申请号：US17127210
申请日：2020-12-18
CPC分类号： C12N15/86 , C07K14/721 , C12N9/22 , C12N15/62 , G01N33/743 , C12N2710/10043 , C12N2740/15043 , G01N2333/723
摘要： Disclosed herein are methods and compositions for inactivation of the human glucocorticoid receptor (GR) gene by targeted cleavage of genomic DNA encoding the GR. Such methods and compositions are useful, for example, in therapeutic applications which require retention of immune function during glucocorticoid treatment.
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公开(公告)号：US11352631B2
公开(公告)日：2022-06-07
申请号：US15380643
申请日：2016-12-15
发明人： Gary K. Lee , David Paschon , Lei Zhang
IPC分类号： C12N15/62 , C12N15/63 , A61K48/00 , C12N15/52 , C12N5/073 , C12N5/0735 , C12N5/0783 , C12N5/074 , C12N9/16
摘要： Disclosed herein are methods and compositions for inactivating TCR genes, using engineered nucleases comprising at least one DNA binding domain and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding nucleases, vectors comprising polynucleotides encoding nucleases and cells comprising polynucleotides encoding nucleases and/or cells comprising nucleases are also provided. Disclosed herein are also methods and compositions for expressing a functional exogenous TCR in the absence of endogenous TCR expression in T lymphocytes, including lymphocytes with a central memory phenotype. Polynucleotides encoding exogenous TCR, vectors comprising polynucleotides encoding exogenous TCR and cells comprising polynucleotides encoding exogenous TCR and/or cells comprising exogenous TCR are also provided.
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公开(公告)号：US11110154B2
公开(公告)日：2021-09-07
申请号：US14706747
申请日：2015-05-07
IPC分类号： A61K38/00 , A61K39/00 , A61K48/00 , A61P25/00 , A61P25/14 , A61P25/28 , C07K19/00 , C12N15/62 , C12N15/86 , A61K38/54 , A61K38/46 , A61K38/18 , A61K38/17 , C12N15/864 , C12N15/63
摘要： Disclosed herein are methods and compositions for treating or preventing Huntington's Disease.
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公开(公告)号：US09650648B2
公开(公告)日：2017-05-16
申请号：US14013250
申请日：2013-08-29
发明人： Gregory J. Cost , Philip D. Gregory , Dmitry Guschin , Michael C. Holmes , Jeffrey C. Miller , David Paschon , Edward J. Rebar , Andreas Reik , Fyodor Urnov , Lei Zhang
CPC分类号： C12N15/85 , C07K14/4702 , C07K14/805 , C07K2319/81 , C12N9/22 , C12N15/52
摘要： Methods and compositions for a genetic disease are provided.
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公开(公告)号：US09629930B2
公开(公告)日：2017-04-25
申请号：US14878703
申请日：2015-10-08
IPC分类号： A01N63/00 , C12P21/06 , A01N65/00 , C12N5/00 , A61K48/00 , A61K31/7088 , A61K45/06 , C12N9/64 , A61N5/10 , C12N15/10 , A61N5/06
CPC分类号： A61K48/005 , A61K31/7088 , A61K45/06 , A61N5/10 , A61N2005/0661 , C12N9/644 , C12N15/1024 , C12Y304/21022 , A61K2300/00
摘要： Disclosed herein are methods and compositions for insertion of Factor IX (FIX) sequences into the genome of a cell for treating hemophilia B.
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公开(公告)号：US09597357B2
公开(公告)日：2017-03-21
申请号：US14051244
申请日：2013-10-10
CPC分类号： C07K16/32 , A61K35/17 , A61K35/26 , C07K16/2863 , C07K2317/622 , C07K2317/64 , Y02A50/463
摘要： Methods and compositions for modifying T-cells in which PD1 and/or CTLA-4 is repressed and/or inactivated using fusion proteins such as artificial transcription factors and nucleases.
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公开(公告)号：US09567573B2
公开(公告)日：2017-02-14
申请号：US13066801
申请日：2011-04-25
IPC分类号： C12N9/22 , A01K67/027 , C12N15/10 , C12N15/90 , A61K38/00
CPC分类号： C12N9/22 , A01K67/0275 , A01K2207/05 , A01K2217/07 , A01K2227/105 , A01K2267/03 , A61K38/00 , C07K2319/81 , C12N15/102 , C12N15/907
摘要： Disclosed herein are methods and compositions for genome editing of a Rosa locus, using fusion proteins comprising a DNA binding domain and a cleavage domain or cleavage half-domain. Polynucleotides encoding said fusion proteins are also provided, as are cells comprising said polynucleotides and fusion proteins.
摘要翻译： 本文公开了使用包含DNA结合结构域和切割结构域或裂解半结构域的融合蛋白的罗莎基因座的基因组编辑的方法和组合物。 还提供了编码所述融合蛋白的多核苷酸，以及包含所述多核苷酸和融合蛋白的细胞。
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公开(公告)号：US09506120B2
公开(公告)日：2016-11-29
申请号：US12284887
申请日：2008-09-25
申请人： Yannick Doyon , Fyodor Urnov
发明人： Yannick Doyon , Fyodor Urnov
IPC分类号： C12Q1/34 , C12Q1/68 , C12N9/22 , G01N33/573
CPC分类号： C12Q1/6897 , C12N9/22 , C12Q1/34 , G01N33/573 , G01N2333/922
摘要： Disclosed herein are methods and compositions for rapidly identifying and ranking nucleases for specific cleavage of a target sequence.
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公开(公告)号：US09428756B2
公开(公告)日：2016-08-30
申请号：US12376871
申请日：2007-08-09
申请人： Qihua Cai , Jeffrey C. Miller , William M. Ainley , Robbi J. Garrison , Joseph F. Petolino , Beth C. Rubin-Wilson , Lisa L. Schulenberg , Andrew F. Worden
发明人： Qihua Cai , Jeffrey C. Miller , William M. Ainley , Robbi J. Garrison , Joseph F. Petolino , Beth C. Rubin-Wilson , Lisa L. Schulenberg , Andrew F. Worden
IPC分类号： C12N15/82
CPC分类号： C12N15/8213 , C07K14/00 , C07K2319/81 , C12N9/22
摘要： Disclosed herein are methods and compositions for targeted integration of an exogenous sequence into a predetermined target site in a plant genome.
摘要翻译： 本文公开了用于将外源序列靶向整合到植物基因组中的预定靶位点的方法和组合物。
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公开(公告)号：US09376689B2
公开(公告)日：2016-06-28
申请号：US14627812
申请日：2015-02-20
发明人： Yannick Doyon , Jeffrey C. Miller
CPC分类号： C12N15/907 , C07K7/06 , C07K14/47 , C07K2319/50 , C07K2319/80 , C07K2319/81 , C12N9/22 , C12N15/85 , C12Y301/21004
摘要： Disclosed herein are engineered cleavage half-domains; fusion polypeptides comprising these engineered cleavage half-domains; polynucleotides encoding the engineered cleavage half-domains and fusion proteins; and cells comprising said polynucleotides and/or fusion proteins. Also described are methods of using these polypeptides and polynucleotides, for example for targeted cleavage of a genomic sequence.
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