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1. 发明申请

WO2012001527A2 METHOD FOR IMPROVING CLEAVAGE OF DNA BY ENDONUCLEASE SENSITIVE TO METHYLATION 审中-公开
标题翻译：提高对甲基化敏感的核酸内切酶对DNA的切割的方法
公开(公告)号：WO2012001527A2
公开(公告)日：2012-01-05
申请号：PCT/IB2011/002196
申请日：2011-06-15
申请人： CELLECTIS S.A. , DUCHATEAU, Philippe , VALTON, Julien , DABOUSSI, Fayza
发明人： DUCHATEAU, Philippe , VALTON, Julien , DABOUSSI, Fayza
IPC分类号： C12Q1/68
CPC分类号： C12P19/34 , C12Q1/683 , C12Q2521/331
摘要： The present invention concerns novel methods for improving cleavage of DNA by rare-cutting endonucleases, overcoming DNA modification constraints, particularly DNA methylation, thereby giving new tools for genome engineering, particularly to increase the integration efficiency of a transgene into a genome at a predetermined location, including therapeutic applications and cell line engineering.
摘要翻译：本发明涉及通过稀有切割内切核酸酶改善DNA切割的新方法，克服DNA修饰约束，特别是DNA甲基化，从而为基因组工程提供新的工具，特别是提高基因工程的整合效率转基因在预定位置转化为基因组，包括治疗应用和细胞系工程。

2. 发明申请

WO2012138939A1 NEW TALE-PROTEIN SCAFFOLDS AND USES THEREOF 审中-公开
标题翻译：新蛋白质蛋白质及其用途
公开(公告)号：WO2012138939A1
公开(公告)日：2012-10-11
申请号：PCT/US2012/032439
申请日：2012-04-05
申请人： DUCHATEAU, Philippe , JUILLERAT, Alexandre , VALTON, Julien , BERTONATI, Claudia , EPINAT, Jean-charles , SILVA, George, H.
发明人： DUCHATEAU, Philippe , JUILLERAT, Alexandre , VALTON, Julien , BERTONATI, Claudia , EPINAT, Jean-charles , SILVA, George, H.
IPC分类号： A61K38/16 , C12N9/22 , C12N15/10
CPC分类号： C12N9/22 , A61K38/00 , C07K14/4703 , C07K2319/00 , C07K2319/80 , C12N9/16 , C12P19/34
摘要： The present invention relates to new Transcription Activator-Like Effector proteins and more particularly new Transcription Activator-Like Effector Nucleases (TALENs) that can efficiently target and process nucleic acids. The present invention also concerns methods to use these new Transcription Activator-Like Effector proteins. The present invention also relates to vectors, compositions and kits in which Transcription Activator-Like Effector proteins of the present invention are used.
摘要翻译：本发明涉及可以有效靶向和加工核酸的新的转录激活子样效应蛋白，更特别是新的转录激活剂样效应核酸酶（TALEN）。本发明还涉及使用这些新的转录激活子样效应蛋白的方法。本发明还涉及其中使用本发明的转录激活子样效应蛋白的载体，组合物和试剂盒。

3. 发明申请

WO2012138927A2 METHOD FOR THE GENERATION OF COMPACT TALE-NUCLEASES AND USES THEREOF 审中-公开
标题翻译：用于生成紧凑型陶瓷核的方法及其用途
公开(公告)号：WO2012138927A2
公开(公告)日：2012-10-11
申请号：PCT/US2012/032426
申请日：2012-04-05
申请人： DUCHATEAU, Philippe , JUILLERAT, Alexandre , VALTON, Julien , BERTONATI, Claudia , EPINAT, Jean-Charles , SILVA, George, H.
发明人： DUCHATEAU, Philippe , JUILLERAT, Alexandre , VALTON, Julien , BERTONATI, Claudia , EPINAT, Jean-Charles , SILVA, George, H.
IPC分类号： C12Q1/68
CPC分类号： C12N9/22 , A61K38/00 , C07K14/4703 , C07K2319/00 , C07K2319/80 , C12N9/16 , C12P19/34
摘要： The present invention relates to a method for the generation of compact Transcription Activator-Like Effector Nucleases (TALENs) that can efficiently target and process double-stranded DNA. More specifically, the present invention concerns a method for the creation of TALENs that consist of a single TALE DNA binding domain fused to at least one catalytic domain such that the active entity is composed of a single polypeptide chain for simple and efficient vectorization and does not require dimerization to target a specific single double-stranded DNA target sequence of interest and process DNA nearby said DNA target sequence. The present invention also relates to compact TALENs, vectors, compositions and kits used to implement the method.
摘要翻译：本发明涉及能够有效靶向和加工双链DNA的致密转录激活子样效应核酸酶（TALEN）的生成方法。更具体地，本发明涉及一种用于产生TALEN的方法，其由与至少一个催化结构域融合的单个TALE DNA结合结构域组成，使得活性实体由单个多肽链组成，用于简单且有效的载体化，并且不需要二聚化来靶向特定的单个双链DNA靶序列，并在DNA靶序列附近处理DNA。本发明还涉及用于实施该方法的紧凑型TALEN，载体，组合物和试剂盒。

4. 发明申请

WO2013017950A1 HIGH THROUGHPUT METHOD FOR ASSEMBLY AND CLONING POLYNUCLEOTIDES COMPRISING HIGHLY SIMILAR POLYNUCLEOTIDIC MODULES 审中-公开
标题翻译：用于组装和克隆包含高类似多核模块的多核苷酸的高通量方法
公开(公告)号：WO2013017950A1
公开(公告)日：2013-02-07
申请号：PCT/IB2012/001861
申请日：2012-07-20
申请人： CELLECTIS , VALTON, Julien , JULLERAT, Alexandre , DUCHATEAU, Philippe
发明人： VALTON, Julien , JULLERAT, Alexandre , DUCHATEAU, Philippe
IPC分类号： C12N15/66 , C12P19/34
CPC分类号： C12N15/1093 , C07K14/195 , C12N15/66 , C12P19/34 , G01N33/6845
摘要： The present invention relates to a method for the assembly and cloning of polynucleotides comprising highly similar polynucleotidic modules, that is highly versatile, does not require intermediate amplification step and can be easily automated for high throughput production of customized polynucleotidic modules.
摘要翻译：本发明涉及用于组装和克隆多核苷酸的方法，所述多核苷酸包含高度相似的多核苷酸模块，其是高通用性的，不需要中间扩增步骤，并且可以容易地自动化以用于定制多核苷酸模块的高通量生产。

5. 发明申请

WO2009074842A1 IMPROVED CHIMERIC MEGANUCLEASE ENZYMES AND USES THEREOF 审中-公开
标题翻译：改进的重组质粒酶及其用途
公开(公告)号：WO2009074842A1
公开(公告)日：2009-06-18
申请号：PCT/IB2007/004418
申请日：2007-12-13
申请人： CELLECTIS , GRIZOT, Sylvestre , DUCHATEAU, Philippe
发明人： GRIZOT, Sylvestre , DUCHATEAU, Philippe
IPC分类号： C07K14/195 , C12N15/62
CPC分类号： C12N9/22
摘要： The current invention relates to polypeptides encoding mutant I-DmoI derivatives with enhanced cleavage activity and altered sequence specificity and uses of these polypeptides. These polypeptides comprise at least the first I-DmoI domain, and the peptide sequence comprises the substitution of at least one of residues 15, 19 and/or 20 as well as at least one of the residues in positions 29, 33, 35, 75, 76, 77 of the first I-DmoI domain.
摘要翻译：本发明涉及编码具有增强的切割活性和改变的序列特异性和这些多肽的用途的突变体I-DmoI衍生物的多肽。这些多肽包含至少第一个I-DmoI结构域，并且肽序列包含位置29,33,35,75的残基15,19和/或20中的至少一个以及至少一个残基的取代，76，77的第一个I-DmoI域。

6. 发明申请

WO2004067753A2 USE OF MEGANUCLEASES FOR INDUCING HOMOLOGOUS RECOMBINATION EX VIVO AND IN TOTO IN VERTEBRATE SOMATIC TISSUES AND APPLICATION THEREOF. 审中-公开
标题翻译：用于诱导异体重建和血管内皮细胞的体外组织和应用的研究。
公开(公告)号：WO2004067753A2
公开(公告)日：2004-08-12
申请号：PCT/IB2004/000848
申请日：2004-01-28
申请人： CELLECTIS , ARNOULD, Sylvain , BRUNEAU, Sylvia , CABANIOLS, Jean-Pierre , CHAMES, Patrick , CHOULIKA, André , DUCHATEAU, Philippe , EPINAT, Jean-Charles , GOUBLE, Agnès , LACROIX, Emmanuel , PAQUES, Frédéric , PEREZ-MICHAUT, Christophe , SMITH, Julianne , SOURDIVE, David
发明人： ARNOULD, Sylvain , BRUNEAU, Sylvia , CABANIOLS, Jean-Pierre , CHAMES, Patrick , CHOULIKA, André , DUCHATEAU, Philippe , EPINAT, Jean-Charles , GOUBLE, Agnès , LACROIX, Emmanuel , PAQUES, Frédéric , PEREZ-MICHAUT, Christophe , SMITH, Julianne , SOURDIVE, David
IPC分类号： C12N15/90
CPC分类号： C12N15/907 , A01K67/0275 , A01K67/0278 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2227/105 , A01K2267/03 , A01K2267/0337 , A61K38/00 , A61K38/1709 , A61K38/465 , A61K48/00 , A61K48/0058 , C07K14/435 , C07K2319/81 , C12N7/00 , C12N9/22 , C12N15/1058 , C12N15/8509 , C12N15/86 , C12N15/90 , C12N2730/10143 , C12N2799/022 , C12N2800/80 , C12N2830/002 , C12N2830/55 , C12N2840/20 , C12N2840/44 , C12Y301/00 , C12Y301/21004
摘要： Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and to its application for genome engineering and gene therapy.
摘要翻译：使用大范围核酸酶从体外诱导同源重组，并在脊椎动物体细胞组织中诱导同源重组，并用于基因组工程和基因治疗。

7. 发明申请

WO2004067736A2 CUSTOM-MADE MEGANUCLEASE AND USE THEREOF 审中-公开
标题翻译：定制的大核糖核酸酶及其用途
公开(公告)号：WO2004067736A2
公开(公告)日：2004-08-12
申请号：PCT/IB2004/000827
申请日：2004-01-28
申请人： CELLECTIS , ARNOULD, Sylvain , BRUNEAU, Sylvia , CABANIOLS, Jean-Pierre , CHAMES, Patrick , CHOULIKA, André , DUCHATEAU, Philippe , EPINAT, Jean-Charles , GOUBLE, Agnès , LACROIX, Emmanuel , PAQUES, Frédéric , PEREZ-MICHAUT, Christophe , SMITH, Julianne , SOURDIVE, David
发明人： ARNOULD, Sylvain , BRUNEAU, Sylvia , CABANIOLS, Jean-Pierre , CHAMES, Patrick , CHOULIKA, André , DUCHATEAU, Philippe , EPINAT, Jean-Charles , GOUBLE, Agnès , LACROIX, Emmanuel , PAQUES, Frédéric , PEREZ-MICHAUT, Christophe , SMITH, Julianne , SOURDIVE, David
IPC分类号： C12N9/22
CPC分类号： C12N15/907 , A01K67/0275 , A01K67/0278 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2227/105 , A01K2267/03 , A01K2267/0337 , A61K38/00 , A61K38/1709 , A61K38/465 , A61K48/00 , A61K48/0058 , C07K14/435 , C07K2319/81 , C12N7/00 , C12N9/22 , C12N15/1058 , C12N15/8509 , C12N15/86 , C12N15/90 , C12N2730/10143 , C12N2799/022 , C12N2800/80 , C12N2830/002 , C12N2830/55 , C12N2840/20 , C12N2840/44 , C12Y301/00 , C12Y301/21004
摘要： New rare-cutting endonucleases, also called custom-made meganucleases, which recognize and cleave a specific nucleotide sequence, derived polynucleotide sequences, recombinant vector cell, animal, or plant comprising said polynucleotide sequences, process for producing said rare-cutting endonucleases and any use thereof, more particularly, for genetic engineering, antiviral therapy and gene therapy.
摘要翻译：新的罕见切割核酸内切酶，也称为定制的大范围核酸酶，其识别和切割特定的核苷酸序列，衍生的多核苷酸序列，重组载体细胞，动物或包含所述多核苷酸序列的植物，产生所述稀有切割内切核酸酶及其任何用途，更具体地，用于基因工程，抗病毒疗法和基因疗法。

8. 发明申请

WO2013182910A2 NEW TRANSCRIPTION ACTIVATOR-LIKE EFFECTOR (TALE) FUSION PROTEIN 审中-公开
标题翻译：新的转录激活子效应（TALE）融合蛋白
公开(公告)号：WO2013182910A2
公开(公告)日：2013-12-12
申请号：PCT/IB2013/001741
申请日：2013-06-05
申请人： CELLECTIS , JUILLERAT, Alexandre , DUCHATEAU, Philippe
发明人： JUILLERAT, Alexandre , DUCHATEAU, Philippe
CPC分类号： C12N15/907 , C07K2319/80 , C12N9/22 , C12N15/10
摘要： The present invention relates to Transcription Activator-Like Effector (TALE) derived proteins that allow to efficiently target and/or process double stranded nucleic acid sequences. The proteins of the invention are typically chimeric protein monomers composed of a core scaffold comprising Repeat Variable Dipeptide regions (RVDs) having binding specificity to a DNA target sequence, to which is fused a catalytic domain to its N-terminal. This later catalytic domain, which can be a monomer of a nuclease, is placed at this position to possibly interact with another catalytic domain fused to another TAL monomer, such that, when said monomers are binding to their respective target DNA sequences, both catalytic domains form a catalytic entity likely to process DNA in the proximity of these target sequences. This new TAL architecture makes it possible to target only one DNA strand, which is not the case, for instance, with classical TALEN architectures. The present invention also relates to vectors encoding such proteins and compositions or kits in which Transcription Activator-Like Effector (TALE) proteins of the present invention are used.
摘要翻译：本发明涉及允许有效靶向和/或加工双链核酸序列的转录激活子样效应子（TALE）衍生蛋白。本发明的蛋白质通常是由包含对DNA靶序列具有结合特异性的重复可变二肽区域（RVD）的核心支架构成的嵌合蛋白质单体，将催化结构域融合到其N-末端。该后续的催化结构域（其可以是核酸酶的单体）置于该位置以可能与另一个与另一个TAL单体融合的另一催化结构域相互作用，使得当所述单体与其各自的靶DNA序列结合时，两个催化结构域形成可能在这些靶序列附近处理DNA的催化实体。这种新的TAL架构使得可以仅靶向一条DNA链，而不是例如经典的TALEN架构。本发明还涉及编码这样的蛋白质的载体，其中使用本发明的转录激活剂样效应子（TALE）蛋白质的组合物或试剂盒。

9. 发明申请

WO2009074873A1 IMPROVED CHIMERIC MEGANUCLEASE ENZYMES AND USES THEREOF 审中-公开
标题翻译：改进的重组质粒酶及其用途
公开(公告)号：WO2009074873A1
公开(公告)日：2009-06-18
申请号：PCT/IB2008/003744
申请日：2008-12-12
申请人： CELLECTIS , GRIZOT, Sylvestre , DUCHATEAU, Philippe
发明人： GRIZOT, Sylvestre , DUCHATEAU, Philippe
IPC分类号： C07K14/195 , C12N15/62
CPC分类号： C12N9/22
摘要： The current invention relates to polypeptides encoding mutant I-DmoI derivatives with enhanced cleavage activity and altered sequence specificity and uses of these polypeptides. These polypeptides comprise at least the first I-DmoI domain, and the peptide sequence comprises the substitution of at least one of residues 15, 19 and/or 20 as well as at least one of the residues in positions 27, 29, 33, 35, 37, 75, 76, 77, 81 of the first I-DmoI domain.
摘要翻译：本发明涉及编码具有增强的切割活性和改变的这些多肽的序列特异性和改变的突变体I-DmoI衍生物的多肽。这些多肽包含至少第一个I-DmoI结构域，并且肽序列包含位置27,29,33,35中的残基15,19和/或20中的至少一个以及至少一个残基的取代，第一个I-DmoI域的37,75,76,77,81。

10. 发明申请

WO2011135396A1 METHOD FOR MODULATING DOUBLE-STRAND BREAK-INDUCED HOMOLOGOUS RECOMBINATION 审中-公开
标题翻译：用于调制双链断裂诱导的同源重建的方法
公开(公告)号：WO2011135396A1
公开(公告)日：2011-11-03
申请号：PCT/IB2010/001286
申请日：2010-04-30
申请人： CELLECTIS , DUCHATEAU, Philippe , PAQUES, Frédéric , PEREZ-MICHAUT, Christophe , DELACOTE, Fabien
发明人： DUCHATEAU, Philippe , PAQUES, Frédéric , PEREZ-MICHAUT, Christophe , DELACOTE, Fabien
IPC分类号： C12N15/113 , C12N15/11
CPC分类号： C12N15/1137 , C12N15/1034 , C12N15/1086 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2320/12
摘要： The present invention concerns a method for modulating double-strand break- induced homologous recombination through the identification of effectors that modulate said double-strand break-induced homologous recombination by uses of interfering agents; these agents are capable of modulating double-strand break-induced homologous recombination through their respective actions on said effectors. The present invention also concerns the uses of these effectors and interfering agents and derivatives, respectively, by introducing them in an eukaryotic cell in order to modulate and more particularly to increase double-strand break-induced homologous recombination and gene targeting efficiency. The present invention also relates to specific derivatives of identified effectors and interfering agents, vectors encoding them, compositions and kits comprising such derivatives in order to modulate and more particularly to increase double-strand break-induced homologous recombination and gene targeting efficiency.
摘要翻译：本发明涉及通过鉴定通过使用干扰剂调节所述双链断裂诱导的同源重组的效应子来调节双链断裂诱导的同源重组的方法; 这些试剂能够通过其对所述效应物的相应作用来调节双链断裂诱导的同源重组。本发明还分别通过将它们引入真核细胞中来调节和更特别地增加双链断裂诱导的同源重组和基因靶向效率，分别涉及这些效应子和干扰剂和衍生物的用途。本发明还涉及鉴定的效应物和干扰剂的特异性衍生物，编码它们的载体，包含这些衍生物的组合物和试剂盒，以便调节并更具体地增加双链断裂诱导的同源重组和基因靶向效率。

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