公开(公告)号：US20180327761A1

公开(公告)日：2018-11-15

申请号：US16045602

申请日：2018-07-25

Applicant: Cellectis

Inventor： Philippe DUCHATEAU ,  Claudia BERTONATI

IPC: C12N15/82 ,  C12N15/90 ,  C12N15/10 ,  C12Q1/683

CPC classification number: C12N15/8213 ,  C12N15/10 ,  C12N15/907 ,  C12Q1/683 ,  C12Q2521/307

Abstract: The present invention is in the field of a method for genome engineering based on the type II CRISPR system, particularly a method for improving specificity and reducing potential off-site. The method is based on the use of nickase architectures of Cas9 and single or multiple crRNA(s) harboring two different targets lowering the risk of producing off-site cleavage. The present invention also relates to polypeptides, polynucleotides, vectors, compositions, therapeutic applications related to the method described here.

公开(公告)号：US20160102324A1

公开(公告)日：2016-04-14

申请号：US14892707

申请日：2014-05-28

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  Claudia BERTONATI

IPC: C12N15/90 ,  C12N9/22

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N15/1082 ,  C12N15/63 ,  C12N15/902 ,  C12Y301/00 ,  C12Y301/26004

Abstract: The present invention is in the field of CRISPR-Cas system for genome targeting. The present invention relates to new engineered Cas9 scaffolds and uses thereof. More particularly, the present invention relates to methods for genome targeting, cell engineering and therapeutic application. The present invention also relates to vectors, compositions and kits in which the new Cas9 scaffolds of the present invention are used.

Abstract translation: 本发明涉及用于基因组靶向的CRISPR-Cas系统。 本发明涉及新的工程化Cas9支架及其用途。 更具体地，本发明涉及用于基因组靶向，细胞工程和治疗应用的方法。 本发明还涉及其中使用本发明的新的Cas9支架的载体，组合物和试剂盒。

公开(公告)号：US20150225465A1

公开(公告)日：2015-08-13

申请号：US14696920

申请日：2015-04-27

Applicant: Cellectis

Inventor： Claudia BERTONATI ,  Philippe Duchateau ,  Alexandre Juillerat ,  George Silva ,  Julien Valton

IPC: C07K14/195 ,  C12N15/90 ,  C12N9/22

CPC classification number: C07K14/195 ,  A01K67/0275 ,  A01K2217/07 ,  C07K2319/80 ,  C12N9/22 ,  C12N15/62 ,  C12N15/87 ,  C12N15/902 ,  C12N15/907 ,  C12Y301/21

Abstract: The present invention concerns new modular base-per-base specific nucleic acid binding domains (MBBBD) derived from newly identified proteins from the bacterial endosymbiont Burkholderia Rhizoxinica and their use for engineering nucleic acid processing enzymes, such as specific endonucleases or transcription activators.

Abstract translation: 本发明涉及从新鉴定的来自细菌内分泌杆菌伯克霍尔德杆菌（Burkholderia Rhizoxinica）的新鉴定的蛋白质中获得的新的基于碱基的碱基特异性核酸结合结构域（MBBBD）及其用于工程化核酸加工酶如特异性内切核酸酶或转录激活子的用途。

公开(公告)号：US20170073423A1

公开(公告)日：2017-03-16

申请号：US15106783

申请日：2014-12-19

Applicant: CELLECTIS

Inventor： Alexandre JUILLERAT ,  Claudia BERTONATI ,  Julien VALTON ,  Philippe DUCHATEAU ,  Laurent POIROT

IPC: C07K16/30 ,  C07K16/28 ,  C07K14/47

CPC classification number: C07K16/30 ,  C07K14/47 ,  C07K16/2863 ,  C07K16/468 ,  C07K2317/622 ,  C07K2319/03 ,  C07K2319/30 ,  C12N2510/02

Abstract: The present invention relates to a method to engineer immune cell for immunotherapy. In particular said immune cells are engineered with chimeric antigen receptors, which be activated by the combination of hypoxia and ligand extracellular binding as input signals. The invention also relates to new designed chimeric antigen receptors which are able to redirect immune cell specificity and reactivity toward a selected target exploiting the ligand-binding domain properties and the hypoxia condition. The present invention also relates to cells obtained by the present method, in particular T-cells, comprising said chimeric antigen receptors for use in cancer treatments.

Abstract translation: 本发明涉及免疫治疗免疫细胞的设计方法。 特别地，所述免疫细胞用嵌合抗原受体工程化，其通过缺氧和配体细胞外结合的组合被激活作为输入信号。 本发明还涉及新设计的嵌合抗原受体，其能够将免疫细胞特异性和反应性转向利用配体结合结构域性质和缺氧条件的选定靶标。 本发明还涉及通过本方法获得的细胞，特别是包含用于癌症治疗的所述嵌合抗原受体的T细胞。

公开(公告)号：US20160355875A1

公开(公告)日：2016-12-08

申请号：US15028644

申请日：2014-10-10

Applicant: CELLECTIS

Inventor： Julien VALTON ,  Alexandre JUILLERAT ,  Fayza DABOUSSI ,  Marine BEURDELEY ,  Claudia BERTONATI ,  Philippe DUCHATEAU

IPC: C12Q1/68 ,  G01N33/53

CPC classification number: C12Q1/6816 ,  C12Q1/6806 ,  C12Q1/6827 ,  C12Q1/6841 ,  G01N33/5308 ,  C12Q2522/101 ,  C12Q2565/531

Abstract: The present invention relates to a method for the detection of a specific nucleic acid. Specifically, the invention provides a method and kits for detecting the presence of a specific nucleic acid using engineered DNA-binding domains such as Transcription Activator Like-Effector (TALE) domain or modular base-per-base binding domains (MBBBD). The method of the invention is particularly useful for in vitro diagnostic application.

Abstract translation: 本发明涉及用于检测特定核酸的方法。 具体地，本发明提供了使用工程化的DNA结合结构域如转录激活子效应（TALE）结构域或模块化碱基结合结构域（MBBBD）来检测特异性核酸的存在的方法和试剂盒。 本发明的方法对于体外诊断应用特别有用。

公开(公告)号：US20160122774A1

公开(公告)日：2016-05-05

申请号：US14892743

申请日：2014-05-28

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  Claudia BERTONATI

IPC: C12N15/82 ,  C12N15/90

CPC classification number: C12N15/8213 ,  C12N15/10 ,  C12N15/907 ,  C12Q1/683 ,  C12Q2521/307

Abstract: The present invention is in the field of a method for genome engineering based on the type II CRISPR system, particularly a method for improving specificity and reducing potential off-site. The method is based on the use of nickase architectures of Cas9 and single or multiple crRNA(s) harboring two different targets lowering the risk of producing off-site cleavage. The present invention also relates to polypeptides, polynucleotides, vectors, compositions, therapeutic applications related to the method described here.

Abstract translation: 本发明在基于II型CRISPR系统的基因组工程方法的领域中，特别是一种提高特异性并降低非局部潜在能力的方法。 该方法基于使用Cas9的切口结构和含有两个不同靶标的单个或多个crRNA，其降低产生场外切割的风险。 本发明还涉及与这里描述的方法相关的多肽，多核苷酸，载体，组合物，治疗应用。