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公开(公告)号：US11778993B2
公开(公告)日：2023-10-10
申请号：US14384957
申请日：2013-03-15
Applicant: CELLECTIS, S.A.
Inventor： Philippe Duchateau , Alexandre Juillerat , Claudia Bertonati
CPC classification number: A01K67/0275 , C07K5/06 , C07K14/00 , C12N5/10 , C12N9/22 , C12N15/63 , C12N15/10 , C12N15/82
Abstract: The present invention relates to polypeptides and more particularly to Transcription Activator-Like Effector derived proteins that allow to efficiently target and/or process nucleic acids. The present invention also concerns methods to use these proteins. The present invention also relates to vectors, compositions and kits in which RVD domains and Transcription Activator-Like Effector (TALE) proteins of the present invention are used.
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公开(公告)号：US20150218230A1
公开(公告)日：2015-08-06
申请号：US14424757
申请日：2013-08-30
Applicant: CELLECTIS, S.A.
Inventor： Alexandre Juillerat , Philippe Duchateau
IPC: C07K14/195 , C07H21/00
CPC classification number: C07K14/195 , C07H21/00 , C07K2319/80 , C12N15/10 , C12N15/102 , C12N15/63 , C12N15/8216 , C12N2501/60
Abstract: Methods for improving or modulating targeting specificity of TALE proteins by introducing alternative RVDs into their modular nucleic acid binding domains. Polynucleotides encoding TALE proteins having alternative targeting specificity towards a nucleic acid target sequence. TALE proteins having alternative targeting specificity towards a nucleic acid target sequence and methods of making and using them.
Abstract translation: 通过将替代RVD引入其模块化核酸结合结构域来改善或调节TALE蛋白的靶向特异性的方法。 编码具有针对核酸靶序列的替代靶向特异性的TALE蛋白的多核苷酸。 具有针对核酸靶序列的替代靶向特异性的TALE蛋白质及其制备和使用它们的方法。
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Patent Agency Ranking
公开(公告)号：US10508289B2
公开(公告)日：2019-12-17
申请号：US15031996
申请日：2014-10-24
Applicant: CELLECTIS, S.A.
Inventor： Philippe Duchateau , Alexandre Juillerat
IPC: C12N15/90 , C12N9/22 , A61K48/00 , C07K14/195 , A61K38/00
Abstract: The present invention is in the field of genetic editing tools and methods of genetic engineering. It relates to the engineering of rare-cutting endonucleases designed to contract highly repetitive motives in chromosomes, which are at the origin of certain genetic diseases, in particular the so-called “triplet repeat diseases”, such as the Huntington disease. The invention encompasses the method for contracting the repetitive motives, the rare-cutting endonucleases for use to contract repetitive motives in a gene subjected to repeat disorder, the polynucleotides and vectors encoding thereof as well as the resulting pharmaceutical compositions.
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公开(公告)号：US10378007B2
公开(公告)日：2019-08-13
申请号：US14424757
申请日：2013-08-30
Applicant: CELLECTIS, S.A.
Inventor： Alexandre Juillerat , Philippe Duchateau
IPC: C12N15/10 , C07K14/195 , C07H21/00 , C12N15/82 , C12N15/63
Abstract: Methods for improving or modulating targeting specificity of TALE proteins by introducing alternative RVDs into their modular nucleic acid binding domains. Polynucleotides encoding TALE proteins having alternative targeting specificity towards a nucleic acid target sequence. TALE proteins having alternative targeting specificity towards a nucleic acid target sequence and methods of making and using them.
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5.发明申请公开(公告)号：US20160298098A1
公开(公告)日：2016-10-13
申请号：US15069672
申请日：2016-03-14
Applicant: CELLECTIS, S.A.
Inventor： Philippe Duchateau , Julien Valton , Claudia Bertonati , Jean-Charles Epinat , George H. Silva , Alexandre Juillerat , Marine Beurdeley
CPC classification number: C12N9/16 , A61K38/00 , C07K14/4703 , C07K2319/00 , C07K2319/80 , C12N9/22 , C12P19/34
Abstract: 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 singe polypeptide chain for simple and efficient vectorization and does not require dimerization to target a specific single double-stranded DNA tar et 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.
Abstract translation: 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（TALENS）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，使得活性实体由简单且有效的载体化的单个多肽链组成，并且不 需要二聚化来靶向目标特定的单链双链DNA焦油和序列，并在DNA附近处理DNA靶序列。 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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6.发明公开公开(公告)号：US20240299453A1
公开(公告)日：2024-09-12
申请号：US18562536
申请日：2022-05-20
Applicant: CELLECTIS S.A. , ALBERT-LUDWIGS-UNIVERSITÄT FREIBURG
Inventor： Toni Cathomen , Tatjana Cornu , Viviane Dettmer-Monaco , Simone Haas , Julia Rositzka , Philippe Duchateau , Alexandre Juillerat
CPC classification number: A61K35/17 , A61K48/005 , C12N5/0636 , C12N9/22 , C12N15/86 , C12N15/907 , C12N2501/2302 , C12N2501/51 , C12N2501/515 , C12N2501/53 , C12N2510/00 , C12N2750/14143 , C12N2800/80 , C12N2830/42
Abstract: The present invention generally relates to the field of genome engineering (gene editing), and more specifically to gene therapy for the treatment of Hyper-lgE syndrome (HIES). In particular, the present invention provides means and methods for genetically modifying HSCs or T-cells involving gene editing reagents, such as TALE-nucleases, that specifically target an endogenous STATS gene comprising at least one mutation causing Hyper-lgE syndrome (HIES), thereby allowing the restoration of the normal cellular phenotype. The present invention also provides populations of engineered HSCs or T-cells which comprise cells comprising an exogenous polynucleotide sequence comprising at least a partial or complete sequence of a functional STATS gene, said exogenous polynucleotide sequence being integrated in an endogenous STATS gene comprising at least one mutation causing Hyper-lgE syndrome (HIES), resulting in the expression of a functional STATS polypeptide. The present invention further provides pharmaceutical compositions comprising the cell populations of the invention, and their use in gene therapy for the treatment of Hyper-lgE syndrome (HIES).
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公开(公告)号：US20150067900A1
公开(公告)日：2015-03-05
申请号：US14384957
申请日：2013-03-15
Applicant: CELLECTIS, S.A.
Inventor： Philippe Duchateau , Alexandre Juillerat , Claudia Bertonati
IPC: A01K67/027 , C12N15/82 , C12N15/10
CPC classification number: A01K67/0275 , C12N15/10 , C12N15/63 , C12N15/82 , C12N15/8213 , C12Q1/6834 , C12Q2522/101
Abstract: The present invention relates to polypeptides and more particularly to Transcription Activator-Like Effector derived proteins that allow to efficiently target and/or process nucleic acids. The present invention also concerns methods to use these proteins. The present invention also relates to vectors, compositions and kits in which RVD domains and Transcription Activator-Like Effector (TALE) proteins of the present invention are used.
Abstract translation: 本发明涉及多肽，更具体地涉及允许有效靶向和/或加工核酸的转录激活子样效应物衍生蛋白。 本发明还涉及使用这些蛋白质的方法。 本发明还涉及使用本发明的RVD结构域和转录激活子效应（TALE）蛋白质的载体，组合物和试剂盒。
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