公开(公告)号：EP3763814A1

公开(公告)日：2021-01-13

申请号：EP20185077.3

申请日：2016-05-06

申请人： The Children's Medical Center Corporation ,  The Broad Institute, Inc. ,  Massachusetts Institute of Technology

发明人： BAUER, Daniel E. ,  ORKIN, Stuart H. ,  SANJANA, Neville ,  SHALEM, Ophir ,  ZHANG, Feng

IPC分类号： C12N15/10 ,  C12N5/078 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

摘要： Provided herein are nucleic acid molecules that target the BCL11A enhancer functional regions, compositions comprising the nucleic acid molecules and methods for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels. In particular, the nucleic acid molecules target the +62, +58, and/or the +55 enhancer functional regions.

公开(公告)号：EP3294873B1

公开(公告)日：2020-08-19

申请号：EP16793259.9

申请日：2016-05-06

申请人： The Children's Medical Center Corporation ,  The Broad Institute, Inc. ,  Massachusetts Institute of Technology

发明人： BAUER, Daniel E. ,  ORKIN, Stuart H. ,  SANJANA, Neville ,  SHALEM, Ophir ,  ZHANG, Feng

IPC分类号： C12N15/10 ,  C12N5/078 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

公开(公告)号：EP3705490A1

公开(公告)日：2020-09-09

申请号：EP20152702.5

申请日：2013-12-12

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

发明人： ZHANG, Feng ,  RAN, Fei ,  SHALEM, Ophir

IPC分类号： C07K14/44

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 compositions and methods related to components of a CRISPR complex particularly comprising a Cas ortholog enzyme.

公开(公告)号：EP2931899A1

公开(公告)日：2015-10-21

申请号：EP13815327.5

申请日：2013-12-12

申请人： The Broad Institute, Inc. ,  Massachusetts Institute of Technology

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 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： 本发明基因的反弹涉及在功能基因组学中使用并集中于基因功能在细胞中的组合物，方法的应用程序和屏幕，也可以使用载体系统和相关的群集定期间净距短回文重复（CRISPR）其它方面-CAS系统及其部件 ， 提供了用于设计和使用搜索矢量的矢量和载体系统，其中的一些编码CRISPR复杂的一个或多个部件，以及方法。 所以提供了在真核细胞和用于利用CRISPR-CAS系统的方法引导CRISPR复合物形成的方法。

公开(公告)号：EP3011033A1

公开(公告)日：2016-04-27

申请号：EP14736205.7

申请日：2014-06-10

申请人： The Broad Institute, Inc. ,  Massachusetts Institute of Technology

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.

摘要翻译： 本发明基因的反弹涉及库，组合物，方法，应用，试剂盒和在功能基因组学中使用并集中于基因功能在细胞中，也可使用载体系统和相关的群集定期间净距短回文重复（CRISPR）等方面的屏幕 - CAS系统及其部件。 提供了用于设计和使用搜索矢量的矢量和载体系统，其中的一些编码CRISPR复杂的一个或多个部件，以及方法。 所以提供了在真核细胞和用于利用CRISPR-CAS系统的方法引导CRISPR复合物形成的方法。

公开(公告)号：EP3011033B1

公开(公告)日：2020-02-19

申请号：EP14736205.7

申请日：2014-06-10

申请人： The Broad Institute, Inc. ,  Massachusetts Institute of Technology

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

公开(公告)号：EP2898075A1

公开(公告)日：2015-07-29

申请号：EP13814364.9

申请日：2013-12-12

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

发明人： ZHANG, Feng ,  RAN, Fei ,  SHALEM, Ophir

IPC分类号： C12N15/63

CPC分类号： C12N9/22 ,  A61K48/00 ,  C12N9/96 ,  C12N15/1082 ,  C12N15/63 ,  C12N15/8213 ,  C12N15/85 ,  C12N15/86 ,  C12N15/907 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2810/10

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 compositions and methods related to components of a CRISPR complex particularly comprising a Cas ortholog enzyme.

公开(公告)号：EP3725885A1

公开(公告)日：2020-10-21

申请号：EP20157288.0

申请日：2014-06-10

申请人： The Broad Institute, Inc. ,  Massachusetts Institute of Technology

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

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

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：EP3064585A1

公开(公告)日：2016-09-07

申请号：EP16151694.3

申请日：2013-12-12

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

发明人： ZHANG, Feng ,  RAN, Fei ,  SHALEM, Ophir

IPC分类号： C12N15/63

CPC分类号： C12N15/113 ,  C12N9/22 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/63 ,  C12N15/79 ,  C12N15/907 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/11 ,  C12N2320/30 ,  C12N2750/14143 ,  G06F19/18 ,  G06F19/22

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 compositions and methods related to components of a CRISPR complex particularly comprising a Cas ortholog enzyme.

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。 提供了涉及特别包含Cas直向同源酶的CRISPR复合物组分的组合物和方法。

公开(公告)号：EP2898075B1

公开(公告)日：2016-03-09

申请号：EP13814364.9

申请日：2013-12-12

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

发明人： ZHANG, Feng ,  RAN, Fei ,  SHALEM, Ophir

IPC分类号： C12N15/63

CPC分类号： C12N9/22 ,  A61K48/00 ,  C12N9/96 ,  C12N15/1082 ,  C12N15/63 ,  C12N15/8213 ,  C12N15/85 ,  C12N15/86 ,  C12N15/907 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2810/10

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 compositions and methods related to components of a CRISPR complex particularly comprising a Cas ortholog enzyme.

摘要翻译： 本发明提供了工程和的系统，方法，和组合物序列和/或靶序列的活动的操作优化。 本发明提供了相关的复杂CRISPR特别是包括在CAS直向同源酶的组分的组合物和方法。