公开(公告)号：WO2017184786A8

公开(公告)日：2017-10-26

申请号：PCT/US2017/028461

申请日：2017-04-19

申请人： THE BROAD INSTITUTE INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  ZHANG, Feng ,  ZETSCHE, Bernd ,  YAN, Winston ,  SANJANA, Neville, Espi ,  JONES, Sara

发明人： ZHANG, Feng ,  ZETSCHE, Bernd ,  YAN, Winston ,  SANJANA, Neville, Espi ,  JONES, Sara

IPC分类号： C12N15/11 ,  C12N9/22 ,  C12N15/113 ,  C12N15/63

摘要： The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA-targeting systems comprising a novel DNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.

公开(公告)号：WO2014093701A9

公开(公告)日：2014-11-06

申请号：PCT/US2013074800

申请日：2013-12-12

申请人： BROAD INST INC ,  MASSACHUSETTS INST TECHNOLOGY ,  ZHANG FENG ,  SANJANA NEVILLE ESPI ,  SHALEM OPHIR

发明人： ZHANG FENG ,  SANJANA NEVILLE ESPI ,  SHALEM OPHIR

IPC分类号： C12N15/63 ,  C12N15/10 ,  C40B40/08

CPC分类号： C12N15/1034 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/1093 ,  C12N15/63 ,  C12N15/635 ,  C12N15/907 ,  C12N2750/14143

摘要： The present invention generally relates to compositions, methods applications and screens used in functional genomics that focus on gene function in a ceil and that may use vector systems and other aspects related to Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)- Cas systems and components thereof. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRJSPR-Cas system.

摘要翻译： 本发明一般涉及专注于细胞基因功能的功能基因组学中使用的组合物，方法应用和筛选，并且可以使用载体系统和与聚簇定期间隔短回归重复（CRISPR）-Cas系统及其组分相关的其它方面 。 提供了载体和载体系统，其中一些编码CRISPR复合物的一个或多个组分，以及用于设计和使用这种载体的方法。 还提供了在真核细胞中引导CRISPR复合物形成的方法以及利用CRJSPR-Cas系统的方法。

公开(公告)号：WO2014093655A9

公开(公告)日：2014-12-04

申请号：PCT/US2013074736

申请日：2013-12-12

申请人： BROAD INST INC ,  MASSACHUSETTS INST TECHNOLOGY ,  HARVARD COLLEGE ,  ZHANG FENG ,  CONG LE ,  PLATT RANDALL JEFFREY ,  RAN FEI ,  SANJANA NEVILLE ESPI

发明人： ZHANG FENG ,  CONG LE ,  PLATT RANDALL JEFFREY ,  RAN FEI ,  SANJANA NEVILLE ESPI

IPC分类号： C12N15/63

CPC分类号： C12N15/63 ,  C12N7/00 ,  C12N9/22 ,  C12N15/01 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/85 ,  C12N15/86 ,  C12N2320/30 ,  C12N2740/15043

摘要： The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors with additional functional domains. Also provided are methods of directing CRISPR complex formation in prokaryotic and eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity.

摘要翻译： 本发明提供了用于操纵靶序列的序列和/或活性的系统，方法和组合物的工程和优化。 提供了载体和载体系统，其中一些编码CRISPR复合物的一种或多种组分，以及用于设计和使用具有附加功能域的这种载体的方法。 还提供了指导原核和真核细胞中CRISPR复合物形成的方法，以确保增强靶标识别的特异性和避免毒性。

公开(公告)号：WO2014093655A3

公开(公告)日：2014-07-31

申请号：PCT/US2013074736

申请日：2013-12-12

申请人： BROAD INST INC ,  MASSACHUSETTS INST TECHNOLOGY ,  HARVARD COLLEGE ,  ZHANG FENG ,  CONG LE ,  PLATT RANDALL JEFFREY ,  RAN FEI ,  SANJANA NEVILLE ESPI

发明人： ZHANG FENG ,  CONG LE ,  PLATT RANDALL JEFFREY ,  RAN FEI ,  SANJANA NEVILLE ESPI

IPC分类号： C12N15/63

CPC分类号： C12N15/63 ,  C12N7/00 ,  C12N9/22 ,  C12N15/01 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/85 ,  C12N15/86 ,  C12N2320/30 ,  C12N2740/15043

摘要： The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors with additional functional domains. Also provided are methods of directing CRISPR complex formation in prokaryotic and eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity.

摘要翻译： 本发明提供了用于操纵靶序列的序列和/或活性的系统，方法和组合物的工程和优化。 提供了载体和载体系统，其中一些编码CRISPR复合物的一个或多个组分，以及用于设计和使用具有附加功能结构域的载体的方法。 还提供了在原核和真核细胞中引导CRISPR复合物形成的方法，以确保增强靶标识别和避免毒性的特异性。

公开(公告)号：WO2014204727A1

公开(公告)日：2014-12-24

申请号：PCT/US2014041806

申请日：2014-06-10

申请人： BROAD INST INC ,  MASSACHUSETTS INST TECHNOLOGY ,  ZHANG FENG ,  SHALEM OPHIR ,  SANJANA NEVILLE ESPI ,  DOENCH JOHN ,  ROOT DAVID ,  BIEWENER HARTENIAN ELLA NICOLE

发明人： ZHANG FENG ,  SHALEM OPHIR ,  SANJANA NEVILLE ESPI ,  DOENCH JOHN ,  ROOT DAVID ,  BIEWENER HARTENIAN ELLA NICOLE

IPC分类号： C12N15/63 ,  C12N9/22 ,  C12N15/10

CPC分类号： C12N15/1093 ,  C12N9/22 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/63 ,  C12N15/907

摘要： The present invention generally relates to libraries, compositions, methods, applications, kits and screens used in functional genomics that focus on gene function in a cell and that may use vector systems and other aspects related to Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas systems and components thereof. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system.

摘要翻译： 本发明一般涉及专注于细胞中的基因功能的功能基因组学中使用的文库，组合物，方法，应用，试剂盒和筛选，并且可以使用与聚簇定期间隔短回归度重复（CRISPR）相关的载体系统和其它方面 - Cas系统及其组件。 提供了载体和载体系统，其中一些编码CRISPR复合物的一个或多个组分，以及用于设计和使用这种载体的方法。 还提供了在真核细胞中引导CRISPR复合物形成的方法以及利用CRISPR-Cas系统的方法。

公开(公告)号：WO2014093595A1

公开(公告)日：2014-06-19

申请号：PCT/US2013/074611

申请日：2013-12-12

申请人： THE BROAD INSTITUTE, INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  PRESIDENT AND FELLOWS OF HARVARD COLLEGE ,  THE ROCKEFELLER UNIVERSITY ,  ZHANG, Feng ,  BIKARD, David, Olivier ,  CONG, Le ,  COX, David Benjamin, Turitz ,  HSU, Patrick ,  JIANG, Wenyan ,  LIN, Shauiliang ,  MARRAFFINI, Luciano ,  PLATT, Randall, Jeffrey ,  RAN, Fei ,  SANJANA, Neville, Espi

发明人： ZHANG, Feng ,  BIKARD, David, Olivier ,  CONG, Le ,  COX, David Benjamin, Turitz ,  HSU, Patrick ,  JIANG, Wenyan ,  LIN, Shauiliang ,  MARRAFFINI, Luciano ,  PLATT, Randall, Jeffrey ,  RAN, Fei ,  SANJANA, Neville, Espi

IPC分类号： C12N15/63

CPC分类号： C12N15/746 ,  C12N9/22 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/113 ,  C12N15/63 ,  C12N15/70 ,  C12N15/74 ,  C12N15/85 ,  C12N15/8509 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/3519 ,  C12N2310/531 ,  C12N2800/101

摘要： The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR/Cas system.

摘要翻译： 本发明提供了用于操纵靶序列的序列和/或活性的系统，方法和组合物。 提供了载体和载体系统，其中一些编码CRISPR复合物的一个或多个组分，以及用于设计和使用这种载体的方法。 还提供了在真核细胞中引导CRISPR复合物形成的方法以及通过引入使用CRISPR / Cas系统的精确突变来选择特异性细胞的方法。

公开(公告)号：WO2014018423A8

公开(公告)日：2014-01-30

申请号：PCT/US2013/051418

申请日：2013-07-21

申请人： THE BROAD INSTITUTE, INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  PRESIDENT AND FELLOWS OF HARVARD COLLEGE ,  ZHANG, Feng ,  BRIGHAM, Mark ,  CONG, Le ,  KONERMANN, Silvana ,  SANJANA, Neville, Espi

发明人： ZHANG, Feng ,  BRIGHAM, Mark ,  CONG, Le ,  KONERMANN, Silvana ,  SANJANA, Neville, Espi

IPC分类号： A61K48/00 ,  C12N15/62 ,  C12N15/63 ,  C12N15/90

摘要： The present invention generally relates to methods and compositions used for the spatial and temporal control of gene expression that may use inducible transcriptional effectors. The invention particularly relates to inducible methods of altering or perturbing expression of a genomic locus of interest in a cell wherein the genomic locus may be contacted with a non- naturally occurring or engineered composition comprising a deoxyribonucleic acid (DNA) binding polypeptide.

摘要翻译： 本发明一般涉及用于基因表达的空间和时间控制的方法和组合物，其可以使用可诱导的转录效应子。 本发明特别涉及改变或干扰细胞中目的基因组基因座的表达的可诱导方法，其中所述基因组座位可以与包含脱氧核糖核酸（DNA）结合多肽的非天然存在的或工程化的组合物接触。

公开(公告)号：WO2017184786A1

公开(公告)日：2017-10-26

申请号：PCT/US2017/028461

申请日：2017-04-19

申请人： THE BROAD INSTITUTE INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  ZHANG, Feng ,  ZETSCHE, Bemd ,  YAN, Winston ,  SANJANA, Neville, Espi ,  JONES, Sara

发明人： ZHANG, Feng ,  ZETSCHE, Bemd ,  YAN, Winston ,  SANJANA, Neville, Espi ,  JONES, Sara

IPC分类号： C12N15/11 ,  C12N9/22 ,  C12N15/113 ,  C12N15/63

摘要： The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA-targeting systems comprising a novel DNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.

摘要翻译： 本发明提供了用于靶向核酸的系统，方法和组合物。 具体而言，本发明提供了非天然存在的或工程化的靶向DNA的系统，其包含新的靶向DNA的CRISPR效应子蛋白和至少一种靶向核酸成分如引导RNA。

公开(公告)号：WO2014204727A9

公开(公告)日：2014-12-24

申请号：PCT/US2014/041806

申请日：2014-06-10

申请人： THE BROAD INSTITUTE INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  ZHANG, Feng ,  SHALEM, Ophir ,  SANJANA, Neville, Espi ,  DOENCH, John ,  ROOT, David ,  BIEWENER HARTENIAN, Ella Nicole

发明人： ZHANG, Feng ,  SHALEM, Ophir ,  SANJANA, Neville, Espi ,  DOENCH, John ,  ROOT, David ,  BIEWENER HARTENIAN, Ella Nicole

IPC分类号： C12N15/63 ,  C12N9/22 ,  C12N15/10

摘要： The present invention generally relates to libraries, compositions, methods, applications, kits and screens used in functional genomics that focus on gene function in a cell and that may use vector systems and other aspects related to Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas systems and components thereof. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system.

公开(公告)号：WO2014093701A1

公开(公告)日：2014-06-19

申请号：PCT/US2013/074800

申请日：2013-12-12

申请人： THE BROAD INSTITUTE, INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  ZHANG, Feng ,  SANJANA, Neville, Espi ,  SHALEM, Ophir

发明人： ZHANG, Feng ,  SANJANA, Neville, Espi ,  SHALEM, Ophir

IPC分类号： C12N15/63 ,  C12N15/10 ,  C40B40/08

CPC分类号： C12N15/1034 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/1093 ,  C12N15/63 ,  C12N15/635 ,  C12N15/907 ,  C12N2750/14143

摘要： The present invention generally relates to compositions, methods applications and screens used in functional genomics that focus on gene function in a ceil and that may use vector systems and other aspects related to Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)- Cas systems and components thereof. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRJSPR-Cas system.

摘要翻译： 本发明一般涉及用于功能基因组学中的组合物，方法应用和筛选，其集中于细胞中的基因功能，并且可以使用载体系统和与成簇的规则间隔短回文重复相关的其他方面 CRISPR） - Cas系统及其组件。 提供了载体和载体系统，其中一些编码CRISPR复合物的一个或多个组分，以及设计和使用这些载体的方法。 还提供了指导真核细胞中CRISPR复合物形成的方法和利用CRJSPR-Cas系统的方法。