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1. 发明公开

EP2855671A2 NEW TRANSCRIPTION ACTIVATOR-LIKE EFFECTOR (TALE) FUSION PROTEIN 有权转让
标题翻译：消除转录激活蛋白
公开(公告)号：EP2855671A2
公开(公告)日：2015-04-08
申请号：EP13767070.9
申请日：2013-06-05
申请人： Cellectis , Juillerat, Alexandre , Duchateau, Philippe
发明人： JUILLERAT, Alexandre , DUCHATEAU, Philippe
IPC分类号： C12N9/22 , C12N15/10
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）衍生的蛋白质。本发明的蛋白质通常是由包含与DNA靶序列具有结合特异性的重复可变二肽区域（RVD）的核心支架组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架构使得可以仅靶定一条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子样效应子（TALE）蛋白质的组合物或试剂盒。

2. 发明专利

IL228747D0 Method for the generation of compact tale-nucleases and uses thereof 未知
公开(公告)号：IL228747D0
公开(公告)日：2013-12-31
申请号：IL22874713
申请日：2013-10-06
申请人： CELLECTIS , DUCHATEAU PHILIPPE , VALTON JULIEN , EPINAT JEAN CHARLES , ALEXANDRE JUILLERAT , CLAUDIA BERTONATI , GEORGE H SILVA , MARINE BEURDELEY
IPC分类号： A61K20060101
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.

3. 发明专利

IL228747A Method for the generation of compact tale-nucleases and uses thereof 未知
公开(公告)号：IL228747A
公开(公告)日：2020-04-30
申请号：IL22874713
申请日：2013-10-06
申请人： CELLECTIS , DUCHATEAU PHILIPPE , VALTON JULIEN , EPINAT JEAN CHARLES , ALEXANDRE JUILLERAT , CLAUDIA BERTONATI , GEORGE H SILVA , MARINE BEURDELEY
发明人： DUCHATEAU PHILIPPE , VALTON JULIEN , EPINAT JEAN-CHARLES , ALEXANDRE JUILLERAT , CLAUDIA BERTONATI , GEORGE H SILVA , MARINE BEURDELEY
IPC分类号： A61K38/16 , C12N1/22 , C12N15/10 , C12Q1/68
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.

4. 发明专利

IL225860D0 Method for increasing the efficiency of double-strand break-induced mutagenesis 未知
公开(公告)号：IL225860D0
公开(公告)日：2013-06-27
申请号：IL22586013
申请日：2013-04-21
申请人： CELLECTIS , DUCHATEAU PHILIPPE , JUILLERAT ALEXANDRE , SILVA GEORGE H , EPINAT JEAN CHARLES
IPC分类号： C12N20060101
摘要： The present invention relates to a method for increasing double-strand break-induced mutagenesis at a genomic locus of interest in a cell, thereby giving new tools for genome engineering, including therapeutic applications and cell line engineering. More specifically, the present invention concerns a method for increasing double-strand break-induced mutagenesis at a genomic locus of interest, leading to a loss of genetic information and preventing any scarless re-ligation of said genomic locus of interest by NHEJ. The present invention also relates to engineered endonucleases, chimeric or not, vectors, compositions and kits used to implement this method.

5. 发明申请

WO2012058458A3 METHOD FOR INCREASING THE EFFICIENCY OF DOUBLE-STRAND BREAK-INDUCED MUTAGENESIS 审中-公开
标题翻译：增加双链断裂诱导突变效率的方法
公开(公告)号：WO2012058458A3
公开(公告)日：2013-01-10
申请号：PCT/US2011058133
申请日：2011-10-27
申请人： CELLECTIS SA , DUCHATEAU PHILIPPE , JUILLERAT ALEXANDRE , SILVA GEORGE H , EPINAT JEAN-CHARLES
发明人： DUCHATEAU PHILIPPE , JUILLERAT ALEXANDRE , SILVA GEORGE H , EPINAT JEAN-CHARLES
IPC分类号： C12N9/22 , C12N15/10
CPC分类号： C12N15/01 , C12N9/16 , C12N9/22 , C12N15/102
摘要： The present invention relates to a method for increasing double-strand break-induced mutagenesis at a genomic locus of interest in a cell, thereby giving new tools for genome engineering, including therapeutic applications and cell line engineering. More specifically, the present invention concerns a method for increasing double-strand break-induced mutagenesis at a genomic locus of interest, leading to a loss of genetic information and preventing any scarless re-ligation of said genomic locus of interest by NHEJ. The present invention also relates to engineered endonucleases, chimeric or not, vectors, compositions and kits used to implement this method.
摘要翻译：本发明涉及在细胞中感兴趣的基因座上增加双链断裂诱变的方法，从而为基因组工程提供新的工具，包括治疗应用和细胞系工程。更具体地说，本发明涉及在感兴趣的基因组轨迹上增加双链断裂诱导诱变的方法，导致遗传信息的丧失，并且防止NHEJ对所述感兴趣的所述基因组座位的任何无疤痕重新连接。本发明还涉及用于实施该方法的工程内切核酸酶，嵌合体或非嵌合体载体，组合物和试剂盒。

6. 发明申请

WO2010015899A2 NOVEL METHOD TO GENERATE MEGANUCLEASES WITH ALTERED CHARACTERISTICS 审中-公开
标题翻译：生成具有改变特性的大分子的新方法
公开(公告)号：WO2010015899A2
公开(公告)日：2010-02-11
申请号：PCT/IB2009000486
申请日：2009-02-09
申请人： CELLECTIS , PAQUES FREDERIC , GRIZOT SYLVESTRE , DUCHATEAU PHILIPPE
发明人： PAQUES FREDERIC , GRIZOT SYLVESTRE , DUCHATEAU PHILIPPE
IPC分类号： C07K14/435 , A01K67/00 , A61K38/46 , C07K14/195 , C12N9/22 , C12N15/62 , C12N15/90
CPC分类号： C12N15/1058 , C07K2319/00 , C12N9/22
摘要： Method to generate and select a meganuclease having at least two altered characteristics in comparison to a parent meganuclease, comprising the steps: a. constructing from a parent meganuclease, a first series of variants which differ from said parent meganuclease by at least one acid amino substitution; b. screening the variants from said first series of step a. and selecting those which have a first altered characteristic; c. constructing from the selected variants of step b. a second series of variants having a least one other amino acid substitution; d. screening the variants from said series of step b. and selecting those which have said first altered characteristic and a second altered characteristic. Polypeptide obtained from said method.
摘要翻译：产生和选择与亲本大范围核酸酶相比具有至少两种改变特征的大范围核酸酶的方法，包括以下步骤：a。从亲本大范围核酸酶构建第一系列变体，其与所述亲本大范围核酸酶的差异在于至少一个酸性氨基取代; 湾筛选来自所述第一系列步骤a的变体。并选择具有第一改变特征的那些; C。从步骤b的选定变体构建。具有至少一个其他氨基酸取代的第二系列变体; d。筛选来自所述一系列步骤b的变体。并选择具有所述第一改变特性和第二改变特性的那些。从所述方法获得的多肽。

7. 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。

8. 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。

9. 发明申请

WO2013182910A3 TRANSCRIPTION ACTIVATOR-LIKE EFFECTOR (TALE) FUSION PROTEIN 审中-公开
标题翻译：转录激活子效应（TALE）融合蛋白
公开(公告)号：WO2013182910A3
公开(公告)日：2014-02-13
申请号：PCT/IB2013001741
申请日：2013-06-05
申请人： CELLECTIS , JUILLERAT ALEXANDRE , DUCHATEAU PHILIPPE
发明人： JUILLERAT ALEXANDRE , DUCHATEAU PHILIPPE
IPC分类号： C12N9/22 , C12N15/10
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。

10. 发明申请

WO2012138927A3 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：WO2012138927A3
公开(公告)日：2013-04-25
申请号：PCT/US2012032426
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 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。

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