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1. 发明授权

US09044492B2 Method for modulating the efficiency of double-strand break-induced mutagenesis 有权
标题翻译：调节双链断裂诱变诱变效率的方法
公开(公告)号：US09044492B2
公开(公告)日：2015-06-02
申请号：US13367098
申请日：2012-02-06
申请人： Fabien Delacote , Philippe Duchateau , Christophe Perez-Michaut
发明人： Fabien Delacote , Philippe Duchateau , Christophe Perez-Michaut
IPC分类号： C12Q1/68 , A61K31/7088 , C12N15/10
CPC分类号： A61K31/7088 , C12N15/102 , C12N15/1086
摘要： A method for modulating 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. A method for modulating double-strand break-induced mutagenesis, concerns the identification of effectors that modulate double-strand break-induced mutagenesis by use of interfering agents; these agents are capable of modulating double-strand break-induced mutagenesis through their respective direct or indirect actions on said effectors. Methods of using these effectors, interfering agents and derivatives, respectively, by introducing them into a cell in order to modulate and more particularly to increase double-strand break-induced mutagenesis. Specific derivatives of identified effectors and interfering agents, vectors encoding them, compositions and kits comprising such derivatives for modulating or increasing double-strand break-induced mutagenesis.
摘要翻译：调节细胞中目的基因座的双链断裂诱变诱变的方法，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。调节双链断裂诱变诱变的方法涉及鉴定通过使用干扰剂调节双链断裂诱导诱变的效应物; 这些试剂能够通过其对所述效应物的各自的直接或间接作用来调节双链断裂诱变的诱变。通过将这些效应物，干扰剂和衍生物分别引入细胞以调节并更具体地增加双链断裂诱导的诱变来分别使用这些效应子，干扰剂和衍生物的方法。鉴定的效应子和干扰剂的特异性衍生物，编码它们的载体，包含用于调节或增加双链断裂诱变诱变的衍生物的组合物和试剂盒。

2. 发明申请

US20140121115A1 CUSTOM-MADE MEGANUCLEASE AND USE THEREOF 审中-公开
标题翻译：自定义MEGANUCLEASE及其使用
公开(公告)号：US20140121115A1
公开(公告)日：2014-05-01
申请号：US13725798
申请日：2012-12-21
申请人： Sylvain Arnould , Sylvia Bruneau , Jean-Pierre Cabaniols , Patrick Chames , Andre Choulika , Phillipe Duchateau , Jean-Charles Epinat , Agnes Gouble , Emmanuel Lacroix , Frederic Paques , Christophe Perez-Michaut , Julianne Smith , Davie Sourdive
发明人： Sylvain Arnould , Sylvia Bruneau , Jean-Pierre Cabaniols , Patrick Chames , Andre Choulika , Phillipe Duchateau , Jean-Charles Epinat , Agnes Gouble , Emmanuel Lacroix , Frederic Paques , Christophe Perez-Michaut , Julianne Smith , Davie Sourdive
IPC分类号： C12N15/10
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：新的稀释内切核酸酶，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，包含所述多核苷酸序列的重组载体细胞，动物或植物，用于产生所述稀少内切核酸酶的方法和任何用途更具体地，涉及基因工程，抗病毒治疗和基因治疗。

3. 发明申请

US20120244131A1 METHOD FOR MODULATING THE EFFICIENCY OF DOUBLE-STRAND BREAK-INDUCED MUTAGENESIS 有权
标题翻译：调节双链断裂诱导突变效率的方法
公开(公告)号：US20120244131A1
公开(公告)日：2012-09-27
申请号：US13367098
申请日：2012-02-06
申请人： Fabien Delacote , Philippe Duchateau , Christophe Perez-Michaut
发明人： Fabien Delacote , Philippe Duchateau , Christophe Perez-Michaut
IPC分类号： A61K31/7088 , G01N21/76 , C12N15/63 , C12N15/79 , A61P35/00 , C12N1/19 , C12N1/15 , C07H21/02 , C12N15/11 , A61K35/00 , C40B30/06 , C12N5/10
CPC分类号： A61K31/7088 , C12N15/102 , C12N15/1086
摘要： A method for modulating 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. A method for modulating double-strand break-induced mutagenesis, concerns the identification of effectors that modulate double-strand break-induced mutagenesis by use of interfering agents; these agents are capable of modulating double-strand break-induced mutagenesis through their respective direct or indirect actions on said effectors. Methods of using these effectors, interfering agents and derivatives, respectively, by introducing them into a cell in order to modulate and more particularly to increase double-strand break-induced mutagenesis. Specific derivatives of identified effectors and interfering agents, vectors encoding them, compositions and kits comprising such derivatives for modulating or increasing double-strand break-induced mutagenesis.
摘要翻译：调节细胞中目的基因座的双链断裂诱变诱变的方法，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。调节双链断裂诱变诱变的方法涉及鉴定通过使用干扰剂调节双链断裂诱导诱变的效应物; 这些试剂能够通过其对所述效应物的各自的直接或间接作用来调节双链断裂诱变的诱变。通过将这些效应物，干扰剂和衍生物分别引入细胞以调节并更具体地增加双链断裂诱导的诱变来分别使用这些效应子，干扰剂和衍生物的方法。鉴定的效应子和干扰剂的特异性衍生物，编码它们的载体，包含用于调节或增加双链断裂诱变诱变的衍生物的组合物和试剂盒。

4. 发明申请

US20130061341A1 MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM A XP GENE AND USES THEREOF 审中-公开
标题翻译：从一个XP基因中清除DNA靶序列的MEGANUCLEASE变体及其用途
公开(公告)号：US20130061341A1
公开(公告)日：2013-03-07
申请号：US13560225
申请日：2012-07-27
申请人： Sylvain Arnould , Christophe Perez-Michaut , Julianne Smith
发明人： Sylvain Arnould , Christophe Perez-Michaut , Julianne Smith
IPC分类号： C12N9/16 , C12N15/63 , C12N1/21 , C12N5/10 , A61P17/00 , C12N15/90 , A01K67/027 , A01H5/00 , A61K38/46 , A61K48/00 , C12N15/55 , C12N1/19
CPC分类号： C12N9/22
摘要： An I-CreI variant which has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG (SEQ ID NO: 229) core domain situated from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from a xeroderma pigmentosum gene. Use of said variant and derived products for the prevention and the treatment of Xeroderma pigmentosum.
摘要翻译：具有至少两个取代的I-CreI变体，位于位于I-CreI的位置26至40和44至77的LAGLIDADG（SEQ ID NO：229）核心结构域的两个功能亚结构域中的每一个中，所述变体为能够从干性皮肤色素变性基因切割DNA靶序列。所述变体和衍生产品用于预防和治疗色素性皮炎。

5. 发明授权

US07842489B2 Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof 失效
标题翻译：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中诱导同源重组及其应用
公开(公告)号：US07842489B2
公开(公告)日：2010-11-30
申请号：US12710969
申请日：2010-02-23
申请人： Sylvain Arnould , Sylvia Bruneau , Jean-Pierre Cabaniols , Patrick Chames , Andre Choulika , Philippe Duchateau , Jean-Charles Epinat , Agnes Gouble , Emmanuel Lacroix , Frederic Paques , Christophe Perez-Michaut , Julianne Smith , David Sourdive
发明人： Sylvain Arnould , Sylvia Bruneau , Jean-Pierre Cabaniols , Patrick Chames , Andre Choulika , Philippe Duchateau , Jean-Charles Epinat , Agnes Gouble , Emmanuel Lacroix , Frederic Paques , Christophe Perez-Michaut , Julianne Smith , David Sourdive
IPC分类号： C12N9/22 , C12N15/55
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： A single chain homing endonuclease, comprising a first variant of I-CreI having the amino acid sequence of accession number pdb 1g9y (SEQ ID NO: 23 is residues 1-153 of pdb Ig9y) and a second variant of I-CreI variant having the amino acid sequence of accession number pdb 1g9y (SEQ ID NO: 23 is residues 1-153 of pdb Ig9y) in a single polypeptide.
摘要翻译：包含具有登录号pdb 1g9y（SEQ ID NO：23的氨基酸序列为pdb Ig9y的残基1-153）的I-CreI的第一变体的单链归巢内切核酸酶以及I-CreI变体的第二变体，其具有在单个多肽中，登录号pdb 1g9y（SEQ ID NO：23是pdb Ig9y的残基1-153）的氨基酸序列。

6. 发明申请

US20100203031A1 METHOD FOR ENHANCING THE CLEAVAGE ACTIVITY OF I-CREI DERIVED MEGANUCLEASES 审中-公开
标题翻译：提高I-CREI衍生化合物的清除活性的方法
公开(公告)号：US20100203031A1
公开(公告)日：2010-08-12
申请号：US12663445
申请日：2008-06-06
申请人： Sylvestre Grizot , Agnes Gouble , Christophe Perez-Michaut
发明人： Sylvestre Grizot , Agnes Gouble , Christophe Perez-Michaut
IPC分类号： A61K38/46 , C12N9/16 , C07H21/04 , C12N15/63 , C12N5/10 , A01K67/027 , A01H5/00 , A61K31/7088 , A61P31/12 , A01N63/00 , A01P1/00
CPC分类号： C12N9/22
摘要： A method for enhancing the cleavage activity of an I-CreI derived meganuclease, comprising the site-specific mutation of at least one amino acid residue which is selected in the group consisting of: the glycine at position 19, the phenylalanine at position 54, the phenylalanine at position 87, the serine at position 79, the valine at position 105 and the isoleucine at position 132 of I-CreI, and its application for the manufacturing of meganuclease cleaving a DNA target of interest, for use in genome therapy (treatment of genetic diseases) and genome engineering (making of transgenic animals, transgenic plants and recombinant cell lines).
摘要翻译：一种用于增强I-CreI衍生的大范围核酸酶的切割活性的方法，其包含至少一个氨基酸残基的位点特异性突变，其选自：位置19处的甘氨酸，第54位的苯丙氨酸，第87位的苯丙氨酸，79位的丝氨酸，105位的缬氨酸和I-CreI的132位的异亮氨酸，以及其用于制造用于基因组治疗的感兴趣的DNA靶的大范围核酸酶的应用（治疗遗传疾病）和基因组工程（转基因动物，转基因植物和重组细胞系的制备）。

7. 发明申请

US20100144012A1 USE OF MEGANUCLEASES FOR INDUCING HOMOLOGOUS RECOMBINATION EX VIVO AND IN TOTO IN VERTEBRATE SOMATIC TISSUES AND APPLICATION THEREOF 失效
标题翻译：用于诱导异体重组和血栓形成的皮质组织中的巨细胞病毒的应用及其应用
公开(公告)号：US20100144012A1
公开(公告)日：2010-06-10
申请号：US12710969
申请日：2010-02-23
申请人： Sylvain ARNOULD , Sylvia Bruneau , Jean-Pierre Cabaniols , Patrick Chames , André Choulika , Philippe Duchateau , Jean-Charles Epinat , Agnes Gouble , Emmanuel Lacroix , Frédéric Paques , Christophe Perez-Michaut , Julianne Smith , David Sourdive
发明人： Sylvain ARNOULD , Sylvia Bruneau , Jean-Pierre Cabaniols , Patrick Chames , André Choulika , Philippe Duchateau , Jean-Charles Epinat , Agnes Gouble , Emmanuel Lacroix , Frédéric Paques , Christophe Perez-Michaut , Julianne Smith , David Sourdive
IPC分类号： C12N9/16
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： A single chain homing endonuclease, comprising a first variant of I-CreI having the amino acid sequence of accession number pdb 1g9y and a second variant of I-CreI variant having the amino acid sequence of accession number pdb 1g9y in a single polypeptide.
摘要翻译：单链归巢内切核酸酶，其包含具有登录号pdb 1g9y的氨基酸序列的I-CreI的第一变体和在单个多肽中具有登录号pdb 1g9y的氨基酸序列的I-CreI变体的第二变体。

8. 发明申请

US20120322689A1 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：用于诱导异体重组和血栓形成的皮质组织中的巨细胞病毒的应用及其应用
公开(公告)号：US20120322689A1
公开(公告)日：2012-12-20
申请号：US13524995
申请日：2012-06-15
申请人： Jean-Charles Epinat , Christophe Perez-Michaut , Frederic Paques
发明人： Jean-Charles Epinat , Christophe Perez-Michaut , Frederic Paques
IPC分类号： C12Q1/68 , C40B30/08 , G01N21/64
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： The present invention provides a method for detecting nucleic acid cleavage induced by a modified rare-cutting endonuclease derived from an initial rare-cutting endonuclease, said modified rare-cutting endonuclease being able to cleave a DNA target sequence, which may be different from the recognition and cleavage site of the initial rare-cutting endonuclease. In one embodiment of the invention expression of a marker gene is used as an indicator of target DNA cleavage.
摘要翻译：本发明提供一种用于检测由初始稀释内切核酸酶衍生的改性稀释内切核酸酶诱导的核酸切割的方法，所述修饰的稀有切割内切核酸酶能够切割DNA靶序列，其可能不同于识别和初始稀释内切核酸酶的切割位点。在本发明的一个实施方案中，标记基因的表达用作靶DNA切割的指示剂。

9. 发明申请

US20120159659A1 CUSTOM-MADE MEGANUCLEASE AND USE THEREOF 审中-公开
标题翻译：自定义MEGANUCLEASE及其使用
公开(公告)号：US20120159659A1
公开(公告)日：2012-06-21
申请号：US12892635
申请日：2010-09-28
申请人： Sylvain Arnould , Sylvia Bruneau , Jean-Pierre Cabaniols , Patrick Chames , André Choulika , Philippe Duchateau , Jean-Pierre Epinat , Agnès Gouble , Emmanuel Lacroix , Frédéric Paques , Christophe Perez-Michaut , Julianne Smith , David Sourdive
发明人： Sylvain Arnould , Sylvia Bruneau , Jean-Pierre Cabaniols , Patrick Chames , André Choulika , Philippe Duchateau , Jean-Pierre Epinat , Agnès Gouble , Emmanuel Lacroix , Frédéric Paques , Christophe Perez-Michaut , Julianne Smith , David Sourdive
IPC分类号： A01K67/027 , C12N15/55 , C12N1/19 , C12N1/21 , A01H5/00 , C12N9/22 , C12N15/63
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：新的稀释内切核酸酶，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，包含所述多核苷酸序列的重组载体细胞，动物或植物，用于产生所述稀少内切核酸酶的方法和任何用途更具体地，涉及基因工程，抗病毒治疗和基因治疗。

10. 发明申请

US20110287513A1 USE OF MEGANUCLEASES FOR INDUCING HOMOLOGOUS RECOMBINATION EX VIVO AND IN TOTO IN VERTEBRATE SOMATIC TISSUES AND APPLICATION THEREOF 有权
标题翻译：用于诱导异体重组和血栓形成的皮质组织中的巨细胞病毒的应用及其应用
公开(公告)号：US20110287513A1
公开(公告)日：2011-11-24
申请号：US13152961
申请日：2011-06-03
申请人： Sylvain Arnould , Sylvia Bruneau , Jean-Pierre Cabaniols , Patrick Chames , Andre Choulika , Philippe Duchateau , Jean-Charles Epinat , Agnes Gouble , Emmanuel Lacroix , Frederic Paques , Christophe Perez-Michaut , Julianne Smith , David Sourdive
发明人： Sylvain Arnould , Sylvia Bruneau , Jean-Pierre Cabaniols , Patrick Chames , Andre Choulika , Philippe Duchateau , Jean-Charles Epinat , Agnes Gouble , Emmanuel Lacroix , Frederic Paques , Christophe Perez-Michaut , Julianne Smith , David Sourdive
IPC分类号： C12N9/16
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： A single chain homing endonuclease, comprising a first variant of I-CreI having the amino acid sequence of accession number pdb 1g9y and a second variant of I-CreI variant having the amino acid sequence of accession number pdb 1g9y in a single polypeptide.
摘要翻译：单链归巢内切核酸酶，其包含具有登录号pdb 1g9y的氨基酸序列的I-CreI的第一变体和在单个多肽中具有登录号pdb 1g9y的氨基酸序列的I-CreI变体的第二变体。

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