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公开(公告)号：EP3964573A1
公开(公告)日：2022-03-09
申请号：EP21187367.4
申请日：2017-08-24
摘要： Described herein are engineered nucleases comprising mutations in the cleavage domain ( e.g., Fok I or homologue thereof) and/or DNA binding domain (zinc finger protein, TALE, single guide RNA) such that on-target specificity is increased.
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公开(公告)号：EP2780460B1
公开(公告)日：2018-07-11
申请号：EP12850371.1
申请日：2012-11-16
发明人： GREGORY, Philip D. , MILLER, Jeffrey C. , PASCHON, David , REBAR, Edward J. , TAN, Siyuan , URNOV, Fyodor , ZHANG, Lei
IPC分类号： C12N15/87 , C07K14/195 , C12N5/04 , C12N5/071 , C12N15/63 , C12N15/90 , C12P19/34 , C12N9/22
CPC分类号： C07K14/195 , C07K2319/80 , C12N1/16 , C12N5/04 , C12N5/0602 , C12N9/1007 , C12N9/22 , C12N15/63 , C12N15/902 , C12N15/907 , C12P19/34 , C12Y301/21004
摘要： Disclosed herein are enhanced polypeptides, polynucleotides encoding these polypeptides, 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.
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公开(公告)号：EP4328304A3
公开(公告)日：2024-08-28
申请号：EP23206260.4
申请日：2011-02-07
发明人： DOYON, Yannick , MILLER, Jeffrey C.
IPC分类号： C12N9/22 , C07K14/195
CPC分类号： C12N9/22 , C07K2319/5020130101 , C07K2319/8020130101 , C07K2319/8120130101 , 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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公开(公告)号：EP3769775A3
公开(公告)日：2021-03-17
申请号：EP20192119.4
申请日：2016-12-15
摘要： Disclosed herein are compositions for linking DNA binding domains and cleavage domains (or cleavage half-domains) to form non-naturally occurring nucleases with alternative configurations. Also described are methods of making and using compositions comprising these linkers.
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公开(公告)号：EP3080274B1
公开(公告)日：2020-06-03
申请号：EP14869719.6
申请日：2014-12-09
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公开(公告)号：EP4234570A2
公开(公告)日：2023-08-30
申请号：EP23162123.6
申请日：2019-09-18
发明人： MILLER, Jeffrey C. , REBAR, Edward J. , ZHANG, Lei
摘要： Described herein are engineered nucleases specific for PD1 gene target sites, the nucleases comprising mutations in the cleavage domain ( e.g., Fok I or homologue thereof) and/or DNA binding domain (zinc finger protein, TALE, single guide RNA) such that on-target specificity for PD1 gene target sites is increased.
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公开(公告)号：EP3504229A1
公开(公告)日：2019-07-03
申请号：EP17844404.8
申请日：2017-08-24
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公开(公告)号：EP3080143B1
公开(公告)日：2019-02-20
申请号：EP14869305.4
申请日：2014-12-09
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公开(公告)号：EP2892321B1
公开(公告)日：2018-10-24
申请号：EP13834776.0
申请日：2013-09-05
发明人： AINLEY, William Michael , WEBB, Steven R. , SAMUEL, Pon , GUSCHIN, Dmitry Y. , MILLER, Jeffrey C. , ZHANG, Lei
CPC分类号： C12N15/8216 , C07K14/4702 , C07K2319/81 , C12N9/22 , C12N15/102 , C12N15/66 , C12N15/822 , C12N15/8247 , C12N15/902 , C12Y301/00
摘要： A method of gene editing or gene stacking within a FAD2 loci by cleaving, in a site directed manner, a location in a FAD2 gene in a cell, to generate a break in the FAD2 gene and then ligating into the break a nucleic acid molecule associated with one or more traits of interest is disclosed.
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10.发明公开METHODS AND COMPOSITIONS FOR NUCLEASE-MEDIATED GENOME ENGINEERING AND CORRECTION IN HEMATOPOIETIC STEM CELLS 审中-公开核酶介导的基因工程方法和组合物以及造血干细胞的校正
公开(公告)号：EP3194570A1
公开(公告)日：2017-07-26
申请号：EP15841477.1
申请日：2015-09-16
发明人： COST, Gregory J. , MILLER, Jeffrey C. , ZHANG, Lei
IPC分类号： C12N5/071
CPC分类号： C12N9/22 , A61K35/28 , A61K48/005 , C07K14/805 , C12N5/0645 , C12N15/11 , C12N15/111 , C12N15/907 , C12N2310/20 , C12N2510/00 , C12N2800/80
摘要： The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic stem cell.
摘要翻译： 本公开内容处于基因组工程领域，特别是造血干细胞基因组的靶向修饰。 本文公开了用于改变表达或校正编码参与遗传疾病的蛋白质（例如产生疾病中缺乏，缺乏或异常的蛋白质和/或调节这些蛋白质的蛋白质）的一种或多种基因的方法和组合物，所述基因如镰状细胞 疾病。 本发明描述了用于基因治疗和基因组工程的组合物和方法。
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