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公开(公告)号：US20240117383A1
公开(公告)日：2024-04-11
申请号：US18537754
申请日：2023-12-12
CPC分类号： C12N15/907 , C12N5/0636 , C12N5/0646 , C12N5/0696 , C12N9/22 , C12N15/1082 , C12N15/11 , C12N2310/20 , C12N2800/80
摘要： Strategies, systems, compositions, and methods for efficient production of knock-in cellular clones without reporter genes. An essential gene is targeted using a knock-in cassette that comprises an exogenous coding sequence for a gene product of interest (or “cargo sequence”) in frame with and downstream (3′) of an exogenous coding sequence or partial coding sequence of the essential gene. Undesired targeting events create a non-functional version of the essential gene, in essence a knock-out, which is “rescued” by correct integration of the knock-in cassette, which restores the essential gene coding region so that a functional gene product is produced and positions the cargo sequence in frame with and downstream of the essential gene coding sequence.
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公开(公告)号：US11866726B2
公开(公告)日：2024-01-09
申请号：US16630510
申请日：2018-07-13
IPC分类号： C12N15/90 , C12N9/22 , C12N15/10 , C12N5/0735 , C12N5/0789 , C12N5/074 , 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：US11851690B2
公开(公告)日：2023-12-26
申请号：US16569336
申请日：2019-09-12
IPC分类号： C07H21/02 , C12N9/22 , C12N15/11 , A61K31/7105 , A61K38/46
CPC分类号： C12N9/22 , A61K31/7105 , A61K38/465 , C12N15/111 , C12N2310/20 , C12N2320/30
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：US11806408B2
公开(公告)日：2023-11-07
申请号：US16642582
申请日：2018-08-28
发明人： Shannon E. Boye , Sanford L. Boye , Morgan Maeder
CPC分类号： A61K48/005 , A61K35/761 , A61K48/0075 , C12N15/86 , C07H21/02 , C07H21/04 , C12N2310/20 , C12N2750/14143 , C12N2800/80
摘要： Provided herein are methods and compositions for treating an eye disorder, for example cone-rod dystrophy type 6 (CORD6). In certain aspects, a therapeutically effective amount of a composition comprising nucleic acids is administered to a subject to treat an autosomal dominant disorder or condition, such as a condition associated with a dominant mutation in a guanylate cyclase 2D (GUCY2D) gene, such as knocking out a dominant mutant form of the gene in the subject. Further provided herein are recombinant AAV particles that comprise one or more recombinant AAV genomes comprising nucleic acids that encode a guide RNA that targets a GUCY2D gene and/or an RNA-guided endonuclease.
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公开(公告)号：US20230340456A1
公开(公告)日：2023-10-26
申请号：US18138915
申请日：2023-04-25
CPC分类号： C12N15/102 , A61K9/5123 , A61K31/7105 , A61K38/465 , C12N9/22 , C12N15/11 , C12Y301/11002 , C12N2310/20 , C12N2800/80
摘要： The present disclosure is directed to methods of producing a modified nucleic acid comprising a precise deletion in a target nucleic acid in a cell comprising generating, within the cell, a first single strand break on a first strand of the target nucleic acid and a second single strand break on a second strand of the target nucleic acid, thereby forming a double strand break in the target nucleic acid having a first 3′ overhang and a second 3′ overhang; processing the first 3′ overhang and the second 3′ overhang with an exonuclease molecule, thereby deleting the segment of the target nucleic acid that was located between the first single strand break and the second single strand break, and forming a processed double strand break; and allowing the processed double strand break to be repaired by at least one DNA repair pathway, thereby producing the modified nucleic acid comprising the precise deletion in the target nucleic acid in the cell. Gene editing systems, vectors, polynucleotides, and methods of treatment are also disclosed herein.
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公开(公告)号：US20230313174A1
公开(公告)日：2023-10-05
申请号：US17959787
申请日：2022-10-04
IPC分类号： C12N15/10
CPC分类号： C12N15/1058
摘要： The present disclosure relates to compositions, systems and methods for analyzing activity of nucleases.
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97.发明授权公开(公告)号：US11692205B2
公开(公告)日：2023-07-04
申请号：US17332764
申请日：2021-05-27
CPC分类号： C12N15/907 , C12N9/22 , C12N9/96 , C12N15/102 , C12N15/11 , C12N15/113 , C12N15/63 , C12N2310/20 , C12N2810/10
摘要： Engineered nucleic acids encoding genome editing system components are provided, as are engineered RNA-guided nucleases that include inserts encoded in part by cellular genomic or other sequences recognized by guide RNAs.
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公开(公告)号：US11667911B2
公开(公告)日：2023-06-06
申请号：US15761968
申请日：2016-09-23
CPC分类号： C12N15/102 , A61K9/5123 , A61K31/7105 , A61K38/465 , C12N9/22 , C12N15/11 , C12Y301/11002 , C12N2310/20 , C12N2800/80
摘要： The present disclosure is directed to methods of producing a modified nucleic acid comprising a precise deletion in a target nucleic acid in a cell comprising generating, within the cell, a first single strand break on a first strand of the target nucleic acid and a second single strand break on a second strand of the target nucleic acid, thereby forming a double strand break in the target nucleic acid having a first 3′ overhang and a second 3′ overhang; processing the first 3′ overhang and the second 3′ overhang with an exonuclease molecule, thereby deleting the segment of the target nucleic acid that was located between the first single strand break and the second single strand break, and forming a processed double strand break; and allowing the processed double strand break to be repaired by at least one DNA repair pathway, thereby producing the modified nucleic acid comprising the precise deletion in the target nucleic acid in the cell. Gene editing systems, vectors, polynucleotides, and methods of treatment are also disclosed herein.
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公开(公告)号：US20230126434A1
公开(公告)日：2023-04-27
申请号：US17836165
申请日：2022-06-09
发明人： Jennifer Leah Gori
IPC分类号： C12N15/87 , A61K35/28 , C07K14/315 , C12N5/0789 , C12N9/22 , A01K67/027 , C12N15/10 , A61K35/17 , A61K35/545
摘要： The methods and compositions described herein surprisingly increase CRISPR/Cas-mediated gene editing in stem cells by transiently treating the cells with a stem cell viability enhancer prior to and/or after contacting the cells with one or more CRISPR/Cas9 components. Further, this treatment also surprisingly results in increased engraftment of the stem cells into the target tissue of a subject. The present disclosure also provides one or more modified CRISPR/Cas9 components which, when used in combination with the stem cell viability enhancer, further increases the frequency of gene editing in stem cells, increases stem cell viability, and increases stem cell engraftment.
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公开(公告)号：US20230111575A1
公开(公告)日：2023-04-13
申请号：US16474198
申请日：2017-12-29
发明人： Jim Heil , Stephanie King , Sam Saccomano , Stacy Capehart , Cecilia Fernandez , Hariharan Jayaram , Bruce Eaton , Karin Zemski Berry
IPC分类号： C12N15/113 , C07H21/04 , C07H21/02 , A61K31/712
摘要： Chemical syntheses of guide molecules are disclosed, along with compositions and methods relating thereto.
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