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公开(公告)号：US12110545B2
公开(公告)日：2024-10-08
申请号：US16475862
申请日：2018-01-05
IPC分类号： C12Q1/6869 , G16B20/50 , G16B30/10
CPC分类号： C12Q1/6869 , G16B20/50 , G16B30/10
摘要： The present disclosure relates to methods of analyzing activity of nucleases, such as ZFNs, TALENs or CRISPR-associated nucleases, in particular their cutting at “off-target” loci.
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公开(公告)号：US12031132B2
公开(公告)日：2024-07-09
申请号：US17019154
申请日：2020-09-11
发明人： Jennifer Leah Gori , Edouard Aupepin De Lamothe-Dreuzy , Jack Heath , John Anthony Zuris , KaiHsin Chang
IPC分类号： C12N15/113 , A61K9/00
CPC分类号： C12N15/113 , A61K9/0009 , C12N2310/313
摘要： Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.
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公开(公告)号：US20240207448A1
公开(公告)日：2024-06-27
申请号：US18555716
申请日：2022-04-15
CPC分类号： A61K48/005 , A61P27/02 , C12N9/22 , C12N15/11 , C12N15/86 , G01N33/6848 , C12N2310/20 , C12N2750/14143
摘要： CRISPR/RNA-guided nuclease-related compositions and methods for treatment of RHO-associated retinitis pigmentosa, e.g., autosomal-dominant retinitis pigmentosa (adRP).
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4.发明公开公开(公告)号：US20240167059A1
公开(公告)日：2024-05-23
申请号：US18520878
申请日：2023-11-28
IPC分类号： C12N15/90 , C12N5/0735 , C12N5/074 , C12N5/0789 , C12N9/22 , C12N15/10 , C12N15/113 , C12N15/86
CPC分类号： C12N15/902 , C12N5/0606 , C12N5/0647 , C12N5/0696 , C12N9/22 , C12N15/102 , C12N15/113 , C12N15/86 , C12N2310/20
摘要： Disclosed herein are genome editing systems and related methods which allow for the detection and quantitative measurement of all possible on-target gene editing outcomes, including targeted integration. The compositions and methods described herein rely on the use of donor templates comprising a 5′ homology arm, a cargo, a one or more priming sites, a 3′ homology arm, and optionally stuffer sequence.
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公开(公告)号：US20240141386A1
公开(公告)日：2024-05-02
申请号：US18161806
申请日：2023-01-30
IPC分类号： C12N15/90 , A61K9/00 , A61K35/761 , C12N9/22 , C12N15/11 , C12N15/113 , C12N15/86
CPC分类号： C12N15/907 , A61K9/0048 , A61K35/761 , C12N9/22 , C12N15/11 , C12N15/113 , C12N15/86 , C12N2310/20 , C12N2320/32 , C12N2330/50 , C12N2330/51 , C12N2750/14143 , C12N2800/80
摘要： Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease.
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6.发明公开公开(公告)号：US20240067992A1
公开(公告)日：2024-02-29
申请号：US18450549
申请日：2023-08-16
发明人： Alexandra GLUCKSMANN , Deborah PALESTRANT , Louis Anthony TARTAGLIA , Jordi MATA-FINK , Agnieszka Dorota CZECHOWICZ
IPC分类号： C12N15/90 , A61K38/46 , A61K47/54 , A61K48/00 , C12N9/22 , C12N9/96 , C12N15/11 , C12N15/82 , C12N15/85
CPC分类号： C12N15/907 , A61K38/465 , A61K47/549 , A61K48/005 , C12N9/22 , C12N9/96 , C12N15/11 , C12N15/8216 , C12N15/85 , C12N15/902 , C12N2310/3513 , C12N2320/33 , C12Y301/00 , Y02A50/30
摘要： Methods and compositions useful in targeting a payload to or editing target nucleic acid are disclosed herein.
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公开(公告)号：US20230002760A1
公开(公告)日：2023-01-05
申请号：US17572403
申请日：2022-01-10
IPC分类号： C12N15/11 , C12N15/113 , A61P7/00 , A61K35/17 , C12N5/0783 , C12N9/22 , C12N9/96
摘要： CRISPR/Cas-related compositions and methods which provide for efficient gene editing of eukaryotic cells using modified gRNAs.
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公开(公告)号：US20220389460A1
公开(公告)日：2022-12-08
申请号：US17840152
申请日：2022-06-14
申请人： EDITAS MEDICINE, INC. , THE BROAD INSTITUTE, INC. , THE UNIVERSITY OF IOWA RESEARCH FOUNDATION , MASSACHUSETTS INSTITUTE OF TECHNOLOGY
发明人： Feng ZHANG , Deborah PALESTRANT , Beverly DAVIDSON , Jordi MATA-FINK , Edgardo RODRIGUEZ , Alexis BORISY
IPC分类号： C12N15/90 , C12N15/63 , C12N15/11 , C12N15/113 , C12N9/22
摘要： Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.
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公开(公告)号：US11339437B2
公开(公告)日：2022-05-24
申请号：US15667603
申请日：2017-08-02
IPC分类号： C12Q1/6883 , C12N9/22 , C12N15/10
摘要： Compositions and methods for treatment of CEP290 related diseases are disclosed.
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