JP2024520528A5 - - Google Patents

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Publication number
JP2024520528A5
JP2024520528A5 JP2023573350A JP2023573350A JP2024520528A5 JP 2024520528 A5 JP2024520528 A5 JP 2024520528A5 JP 2023573350 A JP2023573350 A JP 2023573350A JP 2023573350 A JP2023573350 A JP 2023573350A JP 2024520528 A5 JP2024520528 A5 JP 2024520528A5
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JP
Japan
Prior art keywords
gene editing
editing system
polypeptide
sequence
pbs
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023573350A
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English (en)
Japanese (ja)
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JP2024520528A (ja
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Priority claimed from PCT/US2022/031821 external-priority patent/WO2022256440A2/en
Publication of JP2024520528A publication Critical patent/JP2024520528A/ja
Publication of JP2024520528A5 publication Critical patent/JP2024520528A5/ja
Pending legal-status Critical Current

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JP2023573350A 2021-06-01 2022-06-01 Crisprヌクレアーゼを含む遺伝子編集システム及びそれらの使用 Pending JP2024520528A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202163195621P 2021-06-01 2021-06-01
US63/195,621 2021-06-01
US202163236047P 2021-08-23 2021-08-23
US63/236,047 2021-08-23
US202163272937P 2021-10-28 2021-10-28
US63/272,937 2021-10-28
US202263299695P 2022-01-14 2022-01-14
US63/299,695 2022-01-14
PCT/US2022/031821 WO2022256440A2 (en) 2021-06-01 2022-06-01 Gene editing systems comprising a crispr nuclease and uses thereof

Publications (2)

Publication Number Publication Date
JP2024520528A JP2024520528A (ja) 2024-05-24
JP2024520528A5 true JP2024520528A5 (https=) 2025-06-10

Family

ID=82492907

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023573350A Pending JP2024520528A (ja) 2021-06-01 2022-06-01 Crisprヌクレアーゼを含む遺伝子編集システム及びそれらの使用

Country Status (10)

Country Link
US (2) US20230023791A1 (https=)
EP (1) EP4347818A2 (https=)
JP (1) JP2024520528A (https=)
KR (1) KR20240031238A (https=)
AU (1) AU2022284804A1 (https=)
BR (1) BR112023024985A2 (https=)
CA (1) CA3222023A1 (https=)
IL (1) IL308806A (https=)
TW (1) TW202313971A (https=)
WO (1) WO2022256440A2 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220145334A1 (en) * 2020-11-06 2022-05-12 Pairwise Plants Services, Inc. Compositions and methods for rna-encoded dna-replacement of alleles
WO2024163585A1 (en) * 2023-02-01 2024-08-08 Arbor Biotechnologies, Inc. Gene editing systems comprising type v crispr nuclease and engineered guide rna
KR20250151430A (ko) 2023-02-15 2025-10-21 아버 바이오테크놀로지스, 인크. 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법
EP4703472A1 (en) * 2023-04-26 2026-03-04 Eurus Therapeutics Inc. Non-natural polynucleotides for modification of target nucleotide sequence
WO2024235293A1 (zh) * 2023-05-17 2024-11-21 中国科学院遗传与发育生物学研究所 基于环状rna的引导编辑系统
CN116622678B (zh) * 2023-05-24 2026-03-06 尧唐(上海)生物科技有限公司 一种基因编辑蛋白、其相应的基因编辑系统及应用
WO2025130907A1 (zh) * 2023-12-22 2025-06-26 山东舜丰生物科技有限公司 一种用于引导编辑技术的组合物和方法
WO2025171210A1 (en) 2024-02-09 2025-08-14 Arbor Biotechnologies, Inc. Compositions and methods for gene editing via homology-mediated end joining
WO2025207710A1 (en) * 2024-03-26 2025-10-02 Arbor Biotechnologies, Inc. Rna-guided nuclease polypeptides and gene editing systems comprising such
WO2025207709A1 (en) * 2024-03-26 2025-10-02 Arbor Biotechnologies, Inc. Reverse transcription-mediated gene editing systems and uses thereof
WO2025207713A1 (en) * 2024-03-26 2025-10-02 Arbor Biotechnologies, Inc. Reverse transcription-mediated gene editing systems and uses thereof
WO2026015832A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Reverse transcriptases and gene editing systems comprising such
WO2026015829A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Small reverse transcriptases and gene editing systems comprising such
KR102900467B1 (ko) * 2024-11-08 2025-12-19 광주과학기술원 토홀드 매개 가닥 치환 기반의 rpa-crispr 핵산 검출용 조성물 및 검출 방법

Family Cites Families (96)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
WO2015089427A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
EP3835419A1 (en) 2013-12-12 2021-06-16 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
EP3089989B1 (en) 2013-12-31 2020-06-24 The Regents of The University of California Cas9 crystals and methods of use thereof
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
EP3188763B1 (en) 2014-09-02 2020-05-13 The Regents of The University of California Methods and compositions for rna-directed target dna modification
WO2016106239A1 (en) 2014-12-23 2016-06-30 The Regents Of The University Of California Methods and compositions for nucleic acid integration
AU2016263026A1 (en) 2015-05-15 2017-11-09 Pioneer Hi-Bred International, Inc. Guide RNA/Cas endonuclease systems
WO2016196655A1 (en) 2015-06-03 2016-12-08 The Regents Of The University Of California Cas9 variants and methods of use thereof
US20160362667A1 (en) 2015-06-10 2016-12-15 Caribou Biosciences, Inc. CRISPR-Cas Compositions and Methods
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016279077A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US9580727B1 (en) 2015-08-07 2017-02-28 Caribou Biosciences, Inc. Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides
US11008555B2 (en) 2015-09-17 2021-05-18 The Regents Of The University Of California Variant Cas9 polypeptides comprising internal insertions
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
WO2017070598A1 (en) 2015-10-23 2017-04-27 Caribou Biosciences, Inc. Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids
CN108473981B (zh) 2015-12-04 2022-04-12 卡里布生物科学公司 工程化靶向核酸的核酸
EP3390624A4 (en) 2015-12-18 2019-07-10 The Regents of The University of California Modified site-directed modifying polypeptides and methods of use thereof
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
AU2017257274B2 (en) 2016-04-19 2023-07-13 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
CN109642231A (zh) 2016-06-17 2019-04-16 博德研究所 Vi型crispr直向同源物和系统
EP4485466A3 (en) 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
BR112019006388A2 (pt) 2016-09-30 2019-06-25 Univ California enzimas modificadoras de ácido nucleico guiadas por rna e métodos de uso das mesmas
MX2019003674A (es) 2016-09-30 2021-01-08 Univ California Enzimas modificadoras de ácido nucleico guiadas por arn y métodos de uso de estas.
WO2018071672A1 (en) 2016-10-12 2018-04-19 The Regents Of The University Of Colorado Novel engineered and chimeric nucleases
EP3538561A4 (en) 2016-11-11 2020-10-21 The Regents of The University of California RNA-GUIDED VARIANT POLYPEPTIDES AND THEIR METHODS OF USE
US9816093B1 (en) 2016-12-06 2017-11-14 Caribou Biosciences, Inc. Engineered nucleic acid-targeting nucleic acids
JP2020511141A (ja) 2017-03-15 2020-04-16 ザ・ブロード・インスティテュート・インコーポレイテッド 新規Cas13bオルソログCRISPR酵素及び系
US10876101B2 (en) 2017-03-28 2020-12-29 Locanabio, Inc. CRISPR-associated (Cas) protein
AU2018251801B2 (en) 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
US11692184B2 (en) 2017-05-16 2023-07-04 The Regents Of The University Of California Thermostable RNA-guided endonucleases and methods of use thereof
US20200140835A1 (en) 2017-06-06 2020-05-07 The General Hospital Corporation Engineered CRISPR-Cas9 Nucleases
EP3916086A1 (en) 2017-06-23 2021-12-01 Inscripta, Inc. Nucleic acid-guided nucleases
WO2019005866A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. NEW TYPE VI CRISPR ORTHOLOGISTS AND ASSOCIATED SYSTEMS
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
EP3655530A4 (en) 2017-07-17 2021-07-28 The Broad Institute, Inc. NEW TYPE VI CRISPR ORTHOLOGIST AND SYSTEMS
WO2019036185A1 (en) 2017-08-15 2019-02-21 The Regents Of The University Of California COMPOSITIONS AND METHODS FOR IMPROVING GENOMIC SEQUENCE CORRECTION
WO2019071048A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION
WO2019089796A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Cas12c compositions and methods of use
US11970719B2 (en) 2017-11-01 2024-04-30 The Regents Of The University Of California Class 2 CRISPR/Cas compositions and methods of use
WO2019089804A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Casy compositions and methods of use
AU2018358051B2 (en) 2017-11-01 2025-01-09 The Regents Of The University Of California CasZ compositions and methods of use
US10253365B1 (en) 2017-11-22 2019-04-09 The Regents Of The University Of California Type V CRISPR/Cas effector proteins for cleaving ssDNAs and detecting target DNAs
EP3727469A4 (en) 2017-12-22 2021-12-01 The Broad Institute, Inc. NEW CRISPR ENZYMES AND SYSTEMS
US20210079366A1 (en) 2017-12-22 2021-03-18 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
WO2019173248A1 (en) 2018-03-07 2019-09-12 Caribou Biosciences, Inc. Engineered nucleic acid-targeting nucleic acids
WO2019178428A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna and rna targeting enzymes and systems
WO2019178427A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
WO2019222555A1 (en) 2018-05-16 2019-11-21 Arbor Biotechnologies, Inc. Novel crispr-associated systems and components
CN119490976B (zh) 2018-06-13 2026-03-31 卡里布生物科学公司 改造的cascade组分和cascade复合体
US20210269782A1 (en) 2018-06-26 2021-09-02 The Regents Of The University Of California Rna-guided effector proteins and methods of use thereof
WO2020018142A1 (en) 2018-07-16 2020-01-23 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
US20210284981A1 (en) 2018-07-24 2021-09-16 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
SG11202102068TA (en) 2018-07-31 2021-03-30 Broad Inst Inc Novel crispr enzymes and systems
EP3830301B1 (en) 2018-08-01 2024-05-22 Mammoth Biosciences, Inc. Programmable nuclease compositions and methods of use thereof
JP2021532815A (ja) 2018-08-07 2021-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 新規Cas12b酵素およびシステム
US12275964B2 (en) 2018-08-22 2025-04-15 The Regents Of The University Of California Variant type V CRISPR/Cas effector polypeptides and methods of use thereof
WO2020086475A1 (en) 2018-10-22 2020-04-30 Inscripta, Inc. Engineered enzymes
EP3931313A2 (en) 2019-01-04 2022-01-05 Mammoth Biosciences, Inc. Programmable nuclease improvements and compositions and methods for nucleic acid amplification and detection
US20220049273A1 (en) 2019-03-01 2022-02-17 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
JP7649748B2 (ja) 2019-03-07 2025-03-21 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Crispr-casエフェクターポリペプチド及びその使用方法
MX2021010559A (es) 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.
WO2020186213A1 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Novel nucleic acid modifiers
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
WO2020191233A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020197934A1 (en) 2019-03-22 2020-10-01 The Regents Of The University Of California Compositions and methods for modification of target molecules
WO2020206036A1 (en) 2019-04-01 2020-10-08 The Broad Institute, Inc. Novel nucleic acid modifier
WO2020223634A1 (en) 2019-05-01 2020-11-05 Mammoth Biosciences, Inc. Programmable nucleases and methods of use
EP3980533A1 (en) 2019-06-07 2022-04-13 Scribe Therapeutics Inc. Engineered casx systems
WO2020247883A2 (en) 2019-06-07 2020-12-10 Scribe Therapeutics Inc. Deep mutational evolution of biomolecules
CN114269912A (zh) 2019-06-14 2022-04-01 阿伯生物技术公司 新型crispr dna靶向酶及系统
US12454685B2 (en) 2019-07-08 2025-10-28 The Regents Of The University Of California Variant type V CRISPR/Cas effector polypeptides and methods of use thereof
EP3996738A4 (en) 2019-07-11 2023-07-26 Arbor Biotechnologies, Inc. NOVEL ENZYMES AND SYSTEMS TARGETING CRISPR DNA
JP2022546701A (ja) 2019-08-27 2022-11-07 アーバー バイオテクノロジーズ, インコーポレイテッド 新規crispr dnaターゲティング酵素及びシステム
EP4025588A4 (en) 2019-09-05 2023-09-06 Arbor Biotechnologies, Inc. NOVEL CRISPR DNA TARGETING ENZYMES AND SYSTEMS
EP4028047A4 (en) 2019-09-09 2023-07-26 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
CN115175996A (zh) 2019-09-20 2022-10-11 博德研究所 新颖vi型crispr酶和系统
CA3160186A1 (en) * 2019-11-05 2021-05-14 Pairwise Plants Services, Inc. Compositions and methods for rna-encoded dna-replacement of alleles
JP2023505234A (ja) 2019-12-04 2023-02-08 アーバー バイオテクノロジーズ, インコーポレイテッド ヌクレアーゼを含む組成物及びその使用
CA3157131A1 (en) 2019-12-10 2021-06-17 Inscripta, Inc. Novel mad nucleases
US10704033B1 (en) 2019-12-13 2020-07-07 Inscripta, Inc. Nucleic acid-guided nucleases
EP4081533A4 (en) 2019-12-23 2024-03-20 The Regents of The University of California CRISPR-CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE
CN121380024A (zh) * 2020-03-31 2026-01-23 阿伯生物技术公司 包含Cas12i2变体多肽的组合物及其用途

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