JP7581046B2 - 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ - Google Patents
変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ Download PDFInfo
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- JP7581046B2 JP7581046B2 JP2020511253A JP2020511253A JP7581046B2 JP 7581046 B2 JP7581046 B2 JP 7581046B2 JP 2020511253 A JP2020511253 A JP 2020511253A JP 2020511253 A JP2020511253 A JP 2020511253A JP 7581046 B2 JP7581046 B2 JP 7581046B2
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- C—CHEMISTRY; METALLURGY
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
- C12N9/1007—Methyltransferases (general) (2.1.1.)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1025—Acyltransferases (2.3)
- C12N9/1029—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
- C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
- C12P19/00—Preparation of compounds containing saccharide radicals
- C12P19/26—Preparation of nitrogen-containing carbohydrates
- C12P19/28—N-glycosides
- C12P19/30—Nucleotides
- C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
- C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
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- Wood Science & Technology (AREA)
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- Bioinformatics & Cheminformatics (AREA)
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- Biotechnology (AREA)
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- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024080681A JP2024112895A (ja) | 2017-08-23 | 2024-05-17 | 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762549303P | 2017-08-23 | 2017-08-23 | |
| US62/549,303 | 2017-08-23 | ||
| US201862641687P | 2018-03-12 | 2018-03-12 | |
| US62/641,687 | 2018-03-12 | ||
| PCT/US2018/047577 WO2019040650A1 (en) | 2017-08-23 | 2018-08-22 | GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024080681A Division JP2024112895A (ja) | 2017-08-23 | 2024-05-17 | 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020532967A JP2020532967A (ja) | 2020-11-19 |
| JP2020532967A5 JP2020532967A5 (enExample) | 2021-09-30 |
| JP7581046B2 true JP7581046B2 (ja) | 2024-11-12 |
Family
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Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020511253A Active JP7581046B2 (ja) | 2017-08-23 | 2018-08-22 | 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ |
| JP2024080681A Pending JP2024112895A (ja) | 2017-08-23 | 2024-05-17 | 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024080681A Pending JP2024112895A (ja) | 2017-08-23 | 2024-05-17 | 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US11286468B2 (enExample) |
| EP (1) | EP3672612A4 (enExample) |
| JP (2) | JP7581046B2 (enExample) |
| CN (2) | CN111278450B (enExample) |
| AU (4) | AU2018320865B2 (enExample) |
| BR (1) | BR112020003596A2 (enExample) |
| CA (1) | CA3073448A1 (enExample) |
| WO (1) | WO2019040650A1 (enExample) |
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| US9890364B2 (en) | 2012-05-29 | 2018-02-13 | The General Hospital Corporation | TAL-Tet1 fusion proteins and methods of use thereof |
| AU2015280069B2 (en) | 2014-06-23 | 2021-08-12 | The General Hospital Corporation | Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq) |
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| AU2016226077B2 (en) | 2015-03-03 | 2021-12-23 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| CA3000816A1 (en) | 2015-09-11 | 2017-03-16 | The General Hospital Corporation | Full interrogation of nuclease dsbs and sequencing (find-seq) |
| AU2017302551B2 (en) | 2016-07-26 | 2023-04-27 | The General Hospital Corporation | Variants of CRISPR from Prevotella and Francisella 1 (Cpf1) |
| EP3555297A1 (en) | 2016-12-19 | 2019-10-23 | Editas Medicine, Inc. | Assessing nuclease cleavage |
| WO2018129368A2 (en) | 2017-01-06 | 2018-07-12 | Editas Medicine, Inc. | Methods of assessing nuclease cleavage |
| WO2018195545A2 (en) | 2017-04-21 | 2018-10-25 | The General Hospital Corporation | Variants of cpf1 (cas12a) with altered pam specificity |
| US11499151B2 (en) | 2017-04-28 | 2022-11-15 | Editas Medicine, Inc. | Methods and systems for analyzing guide RNA molecules |
| CA3063733A1 (en) | 2017-05-25 | 2018-11-29 | The General Hospital Corporation | Base editors with improved precision and specificity |
| CN110997908A (zh) | 2017-06-09 | 2020-04-10 | 爱迪塔斯医药公司 | 工程化的cas9核酸酶 |
| EP3652312A1 (en) | 2017-07-14 | 2020-05-20 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| WO2019040650A1 (en) | 2017-08-23 | 2019-02-28 | The General Hospital Corporation | GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY |
| AU2019256287B2 (en) | 2018-04-17 | 2026-02-05 | The General Hospital Corporation | Sensitive in vitro assays for substrate preferences and sites of nucleic acid binding, modifying, and cleaving agents |
| KR102853088B1 (ko) * | 2018-05-11 | 2025-09-02 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법 |
| WO2019217942A1 (en) | 2018-05-11 | 2019-11-14 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| AU2019291918B2 (en) | 2018-06-29 | 2025-06-12 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| EP3850088A4 (en) | 2018-09-07 | 2023-07-19 | Beam Therapeutics, Inc. | Compositions and methods for improving base editing |
| KR20210055733A (ko) | 2018-09-07 | 2021-05-17 | 빔 테라퓨틱스, 인크. | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
| JP7646552B2 (ja) | 2019-01-31 | 2025-03-17 | ビーム セラピューティクス インク. | 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ |
| WO2020163396A1 (en) | 2019-02-04 | 2020-08-13 | The General Hospital Corporation | Adenine dna base editor variants with reduced off-target rna editing |
| US12600971B2 (en) | 2019-02-13 | 2026-04-14 | Beam Therapeutics Inc. | Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence |
| WO2020168135A1 (en) | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| CN114026226B (zh) * | 2019-04-26 | 2024-10-18 | 株式会社图尔金 | 靶特异性crispr突变体 |
| CN112266418A (zh) * | 2019-07-08 | 2021-01-26 | 中国科学院微生物研究所 | 改进的基因组编辑系统及其应用 |
| CN117264998A (zh) * | 2019-07-10 | 2023-12-22 | 苏州齐禾生科生物科技有限公司 | 双功能基因组编辑系统及其用途 |
| CN119913134A (zh) * | 2019-08-29 | 2025-05-02 | 比姆医疗股份有限公司 | 用于编辑突变以允许转录或表达的组合物和方法 |
| US20220290121A1 (en) * | 2019-08-30 | 2022-09-15 | The General Hospital Corporation | Combinatorial Adenine and Cytosine DNA Base Editors |
| CA3153563A1 (en) * | 2019-09-09 | 2021-03-18 | Beam Therapeutics Inc. | Novel crispr enzymes, methods, systems and uses thereof |
| US12594301B2 (en) | 2019-09-27 | 2026-04-07 | Beam Therapeutics Inc. | Compositions and methods for treatment of liquid cancers |
| KR102421129B1 (ko) * | 2019-10-14 | 2022-07-15 | 연세대학교 산학협력단 | 신규 프로토스페이서 인접 모티프 서열 및 이를 이용한 세포의 유전체에서 표적 핵산을 변형시키는 방법 |
| EP4093864A4 (en) * | 2020-01-24 | 2024-04-10 | The General Hospital Corporation | Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants |
| WO2021151085A2 (en) | 2020-01-24 | 2021-07-29 | The General Hospital Corporation | Crispr-cas enzymes with enhanced on-target activity |
| BR112022017715A2 (pt) | 2020-03-04 | 2022-11-16 | Flagship Pioneering Innovations Vi Llc | Métodos e composições para modular um genoma |
| AU2021347359A1 (en) | 2020-09-25 | 2023-05-18 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| CN112430613A (zh) * | 2020-12-08 | 2021-03-02 | 安徽省农业科学院水稻研究所 | 一种宽编辑范围的SpG基因及其应用 |
| WO2022152746A1 (en) | 2021-01-13 | 2022-07-21 | Alia Therapeutics Srl | K526d cas9 variants and applications thereof |
| CA3231712A1 (en) | 2021-09-08 | 2023-03-16 | Flagship Pioneering Innovations Vi, Llc | Pah-modulating compositions and methods |
| KR20260037664A (ko) | 2021-09-08 | 2026-03-18 | 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 | 게놈을 조절하기 위한 방법 및 조성물 |
| CN116286736B (zh) * | 2022-01-17 | 2026-05-01 | 四川大学 | 一种具有扩大的pam识别范围的融合蛋白及其应用 |
| KR20260094824A (ko) * | 2023-10-25 | 2026-06-23 | 씨에스피씨 종콰이 팔마씨우티컬 테크놀로지 (스자좡) 컴퍼니 리미티드 | 고효율, 저오프타겟 유전자 편집 도구 |
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