公开(公告)号：US20230374550A1

公开(公告)日：2023-11-23

申请号：US18106039

申请日：2023-02-06

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

CPC分类号： C12N15/907 ,  C12N9/22 ,  C12N15/1082 ,  C12N15/63 ,  A61K48/005 ,  C12N15/102 ,  C12N2800/80

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：US20190040399A1

公开(公告)日：2019-02-07

申请号：US15967510

申请日：2018-04-30

申请人： THE BROAD INSTITUTE INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  PRESIDENT AND FELLOWS OF HARVARD COLLEGE

发明人： Feng ZHANG ,  Le CONG ,  David Benjamin Turitz COX ,  Patrick HSU ,  Shuailiang LIN ,  Fei RAN ,  Randall Jeffrey PLATT ,  Neville Espi SANJANA

IPC分类号： C12N15/74 ,  C12N9/22 ,  C12N15/113 ,  C12N15/63 ,  C12N15/70 ,  C12N15/85 ,  C12N15/90 ,  C12N15/10

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.

公开(公告)号：US20240035007A1

公开(公告)日：2024-02-01

申请号：US18089023

申请日：2022-12-27

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 ,  A61K48/00 ,  C12N15/90 ,  A61K38/46 ,  C12Q1/6876

CPC分类号： C12N9/22 ,  C12N15/113 ,  A61K48/005 ,  C12N15/907 ,  C12N2310/20 ,  C12N15/902 ,  C12Q1/6876 ,  C12Y301/00 ,  A61K38/465

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：US20190017058A1

公开(公告)日：2019-01-17

申请号：US15967495

申请日：2018-04-30

申请人： THE BROAD INSTITUTE INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  PRESIDENT AND FELLOWS OF HARVARD COLLEGE

发明人： Feng Zhang ,  Le CONG ,  David Benjamin Turitz COX ,  Patrick HSU ,  Shuailiang LIN ,  Fei RAN ,  Randall Jeffrey PLATT ,  Neville Espi SANJANA

IPC分类号： C12N15/74 ,  C12N9/22 ,  C12N15/113 ,  C12N15/63 ,  C12N15/70 ,  C12N15/85 ,  C12N15/90 ,  C12N15/10

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.

公开(公告)号：US20220010339A1

公开(公告)日：2022-01-13

申请号：US17489308

申请日：2021-09-29

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

发明人： Feng ZHANG ,  Omar O. ABUDAYYEH ,  James E. DAHLMAN ,  Patrick HSU ,  David A. SCOTT

IPC分类号： C12N15/90 ,  C12N15/11 ,  A01K67/027 ,  C07K14/47 ,  C12N9/22 ,  C12N9/96

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, wherein the guide sequence is modified by secondary structure to increase the specificity of the CRISPR-Cas system and whereby the secondary structure can protect against exonuclease activity and allow for 5′ additions to the guide sequence.

公开(公告)号：US20200377912A1

公开(公告)日：2020-12-03

申请号：US16844548

申请日：2020-04-09

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：US20160153006A1

公开(公告)日：2016-06-02

申请号：US14972523

申请日：2015-12-17

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

发明人： Feng ZHANG ,  Patrick HSU ,  Chie-yu LIN ,  Fei RAN

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

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.

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。