公开(公告)号：US11597919B2

公开(公告)日：2023-03-07

申请号：US16592744

申请日：2019-10-03

申请人： The Broad Institute, Inc. ,  Massachusetts Institute of Technology ,  University of Tokyo ,  PRESIDENT AND FELLOWS OF HARVARD COLLEGE

发明人： Silvana Konermann ,  Alexandro Trevino ,  Mark Brigham ,  Fei Ran ,  Patrick Hsu ,  Chie-yu Lin ,  Osamu Nureki ,  Hiroshi Nishimasu ,  Ryuichiro Ishitani ,  Feng Zhang

IPC分类号： C12N9/22 ,  A61K38/46 ,  A61K48/00 ,  C12N15/113 ,  C12N15/90 ,  C12Q1/6876 ,  A61P43/00

摘要： The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems.

公开(公告)号：US10550372B2

公开(公告)日：2020-02-04

申请号：US15179912

申请日：2016-06-10

申请人： The Broad Institute Inc. ,  Massachusetts Institute of Technology ,  University of Tokyo ,  President and Fellows of Harvard College

发明人： Silvana Konermann ,  Alexandro Trevino ,  Mark Brigham ,  Fei Ran ,  Patrick Hsu ,  Chie-yu Lin ,  Osamu Nureki ,  Hiroshi Nishimasu ,  Ryuichiro Ishitani ,  Feng Zhang

IPC分类号： C12N9/22 ,  C12N15/113 ,  A61K38/46 ,  C12N15/90 ,  C12Q1/6876 ,  A61K48/00

摘要： The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems.

公开(公告)号：US20160355797A1

公开(公告)日：2016-12-08

申请号：US15179912

申请日：2016-06-10

申请人： The Broad Institute Inc. ,  Massachusetts Institute of Technology ,  University of Tokyo ,  President and Fellows of Harvard College

发明人： Silvana Konermann ,  Alexandro Trevino ,  Mark Brigham ,  Fei Ran ,  Patrick Hsu ,  Chie-yu Lin ,  Osamu Nureki ,  Hiroshi Nishimasu ,  Ryuichiro Ishitani ,  Feng Zhang

IPC分类号： C12N9/22 ,  C12Q1/68 ,  A61K38/46 ,  C12N15/90

摘要： The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems.

摘要翻译： 本发明提供用于改变靶基因序列和相关基因产物的表达的系统，方法和组合物。 提供了关于CRISPR-Cas系统的Cas蛋白的结构信息，使用该信息来生成CRISPR复合物的修饰组分，编码CRISPR复合物的一种或多种组分或修饰组分的载体和载体系统，以及方法 用于设计和使用这些矢量和组件。 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。 特别地，本发明包括优化的功能性CRISPR-Cas酶系统。

公开(公告)号：US10711285B2

公开(公告)日：2020-07-14

申请号：US14972523

申请日：2015-12-17

申请人： The Broad Institute, Inc. ,  Massachusetts Institute of Technology ,  President and Fellows of Harvard College

发明人： Feng Zhang ,  Patrick Hsu ,  Chie-yu Lin ,  Fei Ran

IPC分类号： C12N15/90 ,  C12N9/22 ,  C12N15/10 ,  C12N15/63 ,  A61K48/00

摘要： The invention provides for delivery, 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. 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.

公开(公告)号：US11008588B2

公开(公告)日：2021-05-18

申请号：US14972927

申请日：2015-12-17

申请人： The Broad Institute, Inc. ,  Massachusetts Institute of Technology ,  President and Fellows of Harvard College

发明人： Feng Zhang ,  Chie-yu Lin ,  Fei Ran

IPC分类号： C12N15/90 ,  C12N15/10 ,  C12N9/22 ,  C12N15/63

摘要： The invention provides for delivery, 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. 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.

公开(公告)号：US10851357B2

公开(公告)日：2020-12-01

申请号：US15179711

申请日：2016-06-10

申请人： The Broad Institute, Inc. ,  Massachusetts Institute of Technology ,  UNIVERSITY OF IOWA RESEARCH FOUNDATION

发明人： Beverly Davidson ,  Chie-yu Lin ,  Edgardo Rodriguez ,  Feng Zhang

IPC分类号： C12N9/22 ,  C12N15/10 ,  C12N15/63 ,  A01K67/027 ,  C12N15/90 ,  A61K38/46 ,  A61K48/00 ,  C12N15/113 ,  C12N15/86 ,  A61K31/713 ,  C12N7/00 ,  B82Y5/00

摘要： The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences especially for use as to nucleotide repeat disorders. Provided are delivery systems and tissues or organ which are targeted as sites for delivery especially for use as to nucleotide repeat disorders. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex or system especially for use as to nucleotide repeat disorders, as well as methods for the design and of such. Also provided are methods of directing CRISPR complex or system formation in eukaryotic cells especially for use as to nucleotide repeat disorders including with consideration of specificity for target recognition and avoidance of toxicity and editing or modifying a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

公开(公告)号：US20160354487A1

公开(公告)日：2016-12-08

申请号：US15179941

申请日：2016-06-10

申请人： The Broad Institute Inc. ,  Massachusetts Institute of Technology ,  UNIVERSITY OF IOWA RESEARCH FOUNDATION

发明人： Feng Zhang ,  Beverly Davidson ,  Chie-yu Lin ,  Edgardo Rodriguez

IPC分类号： A61K48/00 ,  A61K38/46 ,  C12N15/86 ,  C12N15/113 ,  C12N9/22

CPC分类号： C12N9/22 ,  A01K67/0276 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0318 ,  A61K31/713 ,  A61K38/465 ,  A61K48/005 ,  B82Y5/00 ,  C12N7/00 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/113 ,  C12N15/63 ,  C12N15/86 ,  C12N15/907 ,  C12N2740/15043 ,  C12N2750/14143 ,  C12Y301/00

摘要： The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a SIN CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing SIN CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

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，方法和组合物的递送，工程和优化。 提供作为递送站点的递送系统和组织或器官。 还提供了其中一些编码SIN CRISPR复合物的一种或多种组分的载体和载体系统，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SIN CRISPR复合物形成的方法，以确保提高靶标识别和避免毒性的特异性，并编辑或修饰目标基因组中的靶位点以改变或改善疾病状态 。