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公开(公告)号：US20240167018A1
公开(公告)日：2024-05-23
申请号：US18317248
申请日：2023-05-15
申请人： The Broad Institute, Inc. , Massachusetts Institute of Technology , Children's Medical Center Corporation
发明人： Daniel E. Bauer , Stuart H. Orkin , Neville Espi Sanjana , Ophir Shalem , Jason Wright , Feng Zhang
IPC分类号： C12N15/10
CPC分类号： C12N15/1079 , C12N15/102 , C12N15/1075 , C12N15/1082
摘要： The application relates to a deep scanning mutagenesis library to interrogate phenotypic changes in a population of cells comprising a plurality of CRISPR-Cas system guide RNAs targeting genomic sequences within at least one continuous genomic region, wherein the guide RNAs target at least 100 genomic sequences upstream of a PAM sequence for every 1000 base pairs within the continuous genomic region and methods for their use.
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公开(公告)号：US20240102004A1
公开(公告)日：2024-03-28
申请号：US18345958
申请日：2023-06-30
申请人： THE BROAD INSTITUTE, INC. , MASSACHUSETTS INSTITUTE OF TECHNOLOGY , CHILDREN'S MEDICAL CENTER CORPORATION
发明人： Daniel E. Bauer , Stuart H. Orkin , Neville Espi Sanjana , Ophir Shalem , Jason Wright , Feng Zhang
IPC分类号： C12N15/10
CPC分类号： C12N15/1079 , C12N15/102 , C12N15/1075 , C12N15/1082
摘要： The application relates to a deep scanning mutagenesis library to interrogate phenotypic changes in a population of cells comprising a plurality of CRISPR-Cas system guide RNAs targeting genomic sequences within at least one continuous genomic region, wherein the guide RNAs target at least 100 genomic sequences upstream of a PAM sequence for every 1000 base pairs within the continuous genomic region and methods for their use.
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公开(公告)号：US20230235285A1
公开(公告)日：2023-07-27
申请号：US18148886
申请日：2022-12-30
申请人： The Children's Medical Center Corporation , The Broad Institute, Inc. , Massachusetts Institute of Technology
发明人： Daniel E. Bauer , Stuat H. Orkin , Neville Sanjana , Ophir Shalem , Feng Zhang
CPC分类号： C12N5/0641 , C12N15/102 , C12N9/22 , C12N15/11 , C12N15/86 , C12N2510/00 , C12N2310/20 , C12N2740/15041
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.
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公开(公告)号：US11685917B2
公开(公告)日：2023-06-27
申请号：US15807007
申请日：2017-11-08
申请人： THE BROAD INSTITUTE, INC. , MASSACHUSETTS INSTITUTE OF TECHNOLOGY , CHILDREN'S MEDICAL CENTER CORPORATION
发明人： Daniel E. Bauer , Stuart H. Orkin , Neville Espi Sanjana , Ophir Shalem , Jason Wright , Feng Zhang
IPC分类号： C12N15/10
CPC分类号： C12N15/1079 , C12N15/102 , C12N15/1075 , C12N15/1082
摘要： The application relates to a deep scanning mutagenesis library to interrogate phenotypic changes in a population of cells comprising a plurality of CRISPR-Cas system guide RNAs targeting genomic sequences within at least one continuous genomic region, wherein the guide RNAs target at least 100 genomic sequences upstream of a PAM sequence for every 1000 base pairs within the continuous genomic region and methods for their use.
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公开(公告)号：US11572543B2
公开(公告)日：2023-02-07
申请号：US15572523
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： Daniel E. Bauer , Stuart H. Orkin , Neville Sanjana , Ophir Shalem , Feng Zhang
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.
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公开(公告)号：US20180171297A1
公开(公告)日：2018-06-21
申请号：US15572523
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： Daniel E. Bauer , Stuart H. Orkin , Neville Sanjana , Ophir Shalem , Feng Zhang
CPC分类号： C12N5/0641 , C12N9/22 , C12N15/102 , C12N15/11 , C12N15/86 , C12N2310/20 , C12N2510/00 , C12N2740/15041
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.
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公开(公告)号：US20180119138A1
公开(公告)日：2018-05-03
申请号：US15807007
申请日：2017-11-08
申请人： THE BROAD INSTITUTE INC. , MASSACHUSETTS INSTITUTE OF TECHNOLOGY , CHILDREN'S MEDICAL CENTER CORPORATION
发明人： Daniel E. Bauer , Stuart H. Orkin , Neville Espi Sanjana , Ophir Shalem , Jason Wright , Feng Zhang
IPC分类号： C12N15/10
CPC分类号： C12N15/1079 , C12N15/102 , C12N15/1075 , C12N15/1082
摘要： The application relates to a deep scanning mutagenesis library to interrogate phenotypic changes in a population of cells comprising a plurality of CRISPR-Cas system guide RNAs targeting genomic sequences within at least one continuous genomic region, wherein the guide RNAs target at least 100 genomic sequences upstream of a PAM sequence for every 1000 base pairs within the continuous genomic region and methods for their use.
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公开(公告)号：US20240191223A1
公开(公告)日：2024-06-13
申请号：US18334074
申请日：2023-06-13
申请人： The Broad Institute, Inc. , Massachusetts Institute of Technology , President and Fellows of Harvard College
CPC分类号： C12N15/102 , C12N9/22 , C12N15/111 , C12N15/113 , C12N15/8213 , C12N15/8509 , C12N2310/20 , C12N2800/22
摘要： The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a novel RNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.
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公开(公告)号：US20240182928A1
公开(公告)日：2024-06-06
申请号：US18235760
申请日：2023-08-18
申请人： The Broad Institute, Inc. , Massachusetts Institute of Technology , President and Fellows of Harvard College , Rutgers, The State University of New Jersey , Skolkovo Institute of Science and Technology , The United States of America, as represented by the Secretary, Dept. of Health and Human Services
发明人： Konstantin Severinov , Feng Zhang , Yuri I. Wolf , Sergey Shmakov , Ekaterina Semenova , Leonid Minakhin , Kira S. Makarova , Eugene Koonin , Silvana Konermann , Julia Joung , Jonathan S. Gootenberg , Omar O. Abudayyeh , Eric S. Lander
IPC分类号： C12N15/90 , C12N9/22 , C12N15/10 , C12N15/11 , C12N15/113 , C12N15/63 , C12N15/82 , C12N15/85
CPC分类号： C12N15/907 , C12N9/22 , C12N15/102 , C12N15/111 , C12N15/113 , C12N15/63 , C12N15/8201 , C12N15/85 , C12N2310/111 , C12N2310/20 , C12N2800/22
摘要： The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a novel RNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.
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公开(公告)号：US20240132916A1
公开(公告)日：2024-04-25
申请号：US18276471
申请日：2022-02-09
发明人： Feng Zhang , Michael Segel , Alim Ladha , Michelle Walsh , Christopher Frangieh
CPC分类号： C12N15/907 , C12N9/22 , C12N15/11 , C12N2310/20 , C12N2800/90
摘要： Systems and methods for targeted gene modification, targeted insertion, perturbation of gene transcripts, and nucleic acid editing. Novel nucleic acid targeting systems comprise components of CRISPR systems and non-LTR retrotransposon elements.
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