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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- 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
- 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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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)
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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)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (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 (3) | AU2018320865B2 (enExample) |
| BR (1) | BR112020003596A2 (enExample) |
| CA (1) | CA3073448A1 (enExample) |
| WO (1) | WO2019040650A1 (enExample) |
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| EP3919621A1 (en) | 2014-06-23 | 2021-12-08 | The General Hospital Corporation | Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq) |
| AU2015330699B2 (en) | 2014-10-10 | 2021-12-02 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| JP6817215B2 (ja) | 2015-03-03 | 2021-01-20 | ザ ジェネラル ホスピタル コーポレイション | 変更PAM特異性を有する遺伝子操作CRISPR−Cas9ヌクレアーゼ |
| CN108350453A (zh) | 2015-09-11 | 2018-07-31 | 通用医疗公司 | 核酸酶dsb的完全查询和测序(find-seq) |
| US11168313B2 (en) | 2016-07-26 | 2021-11-09 | The General Hospital Corporation | Variants of CRISPR from Prevotella and Francisella 1 (Cpf1) |
| WO2018119010A1 (en) | 2016-12-19 | 2018-06-28 | Editas Medicine, Inc. | Assessing nuclease cleavage |
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| JP7518620B2 (ja) | 2017-06-09 | 2024-07-18 | エディタス・メディシン,インコーポレイテッド | 操作されたcas9ヌクレアーゼ |
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| CA3073448A1 (en) | 2017-08-23 | 2019-02-28 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
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| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| KR20250133482A (ko) * | 2018-05-11 | 2025-09-05 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법 |
| AU2019291918B2 (en) | 2018-06-29 | 2025-06-12 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| AU2019336245B2 (en) | 2018-09-07 | 2026-02-26 | Beam Therapeutics Inc. | Compositions and methods for delivering a nucleobase editing system |
| EP3850088A4 (en) | 2018-09-07 | 2023-07-19 | Beam Therapeutics, Inc. | Compositions and methods for improving base editing |
| CN114040977B (zh) | 2019-01-31 | 2024-09-03 | 比姆医疗股份有限公司 | 非靶脱氨反应减低的核碱基编辑器和用于定性核碱基编辑器的测定 |
| EP3921417A4 (en) | 2019-02-04 | 2022-11-09 | The General Hospital Corporation | VARIANTS OF ADENINE DNA BASES WITH REDUCED OFF-TARGET RNA EDIT |
| AU2020223314A1 (en) | 2019-02-13 | 2021-08-12 | Beam Therapeutics Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| SG11202108346WA (en) | 2019-02-13 | 2021-08-30 | Beam Therapeutics Inc | Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence |
| 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 |
| CN112280771A (zh) * | 2019-07-10 | 2021-01-29 | 中国科学院遗传与发育生物学研究所 | 双功能基因组编辑系统及其用途 |
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| CN115279398B (zh) | 2019-09-27 | 2025-07-04 | 比姆医疗股份有限公司 | 治疗液态癌症的组合物和方法 |
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| EP4114937A4 (en) | 2020-03-04 | 2024-09-04 | Flagship Pioneering Innovations VI, LLC | METHODS AND COMPOSITIONS FOR MODULATING A GENOME |
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| 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 |
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