KR20230062873A - 염기 편집 효소 - Google Patents

염기 편집 효소 Download PDF

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Publication number
KR20230062873A
KR20230062873A KR1020237012244A KR20237012244A KR20230062873A KR 20230062873 A KR20230062873 A KR 20230062873A KR 1020237012244 A KR1020237012244 A KR 1020237012244A KR 20237012244 A KR20237012244 A KR 20237012244A KR 20230062873 A KR20230062873 A KR 20230062873A
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KR
South Korea
Prior art keywords
endonuclease
nucleic acid
seq
glu
ala
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Pending
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KR1020237012244A
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English (en)
Korean (ko)
Inventor
쥰 리앙 린
앨런 브룩스
크리스티나 버터필드
크리스토퍼 브라운
신디 캐스텔
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메타지노미, 인크.
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Application filed by 메타지노미, 인크. filed Critical 메타지노미, 인크.
Publication of KR20230062873A publication Critical patent/KR20230062873A/ko
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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]
    • C—CHEMISTRY; METALLURGY
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    • C12N15/09—Recombinant DNA-technology
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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
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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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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
    • C12N15/86—Viral vectors
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    • 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
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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
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2497—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing N- glycosyl compounds (3.2.2)
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    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/02—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2) hydrolysing N-glycosyl compounds (3.2.2)
    • C12Y302/02027—Uracil-DNA glycosylase (3.2.2.27)
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    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Graft Or Block Polymers (AREA)
  • Dental Preparations (AREA)
  • Paints Or Removers (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
KR1020237012244A 2020-09-11 2021-09-10 염기 편집 효소 Pending KR20230062873A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063077057P 2020-09-11 2020-09-11
US63/077,057 2020-09-11
US202163222351P 2021-07-15 2021-07-15
US63/222,351 2021-07-15
PCT/US2021/049962 WO2022056324A1 (en) 2020-09-11 2021-09-10 Base editing enzymes

Publications (1)

Publication Number Publication Date
KR20230062873A true KR20230062873A (ko) 2023-05-09

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Application Number Title Priority Date Filing Date
KR1020237012244A Pending KR20230062873A (ko) 2020-09-11 2021-09-10 염기 편집 효소

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US (3) US12286654B2 (https=)
EP (1) EP4211234A4 (https=)
JP (1) JP2023540797A (https=)
KR (1) KR20230062873A (https=)
AU (1) AU2021339819A1 (https=)
CA (1) CA3192224A1 (https=)
GB (1) GB2605340B (https=)
MX (1) MX2023002913A (https=)
WO (1) WO2022056324A1 (https=)

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EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
GB2617658B (en) 2020-03-06 2024-04-17 Metagenomi Inc Class II, type V CRISPR systems
GB2608292B (en) * 2020-03-31 2024-02-14 Metagenomi Inc Class II, type II CRISPR systems
CA3192224A1 (en) 2020-09-11 2022-03-17 Jyun-Liang LIN Base editing enzymes
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
BR112023014719A2 (pt) * 2021-01-22 2023-12-05 Metagenomi Inc Novas nucleases quiméricas e manipuladas
CA3225082A1 (en) 2021-08-27 2023-03-02 Brian C. Thomas Enzymes with ruvc domains
AU2022380842A1 (en) * 2021-11-05 2024-05-23 Metagenomi Therapeutics, Inc. Base editing enzymes
MX2024006257A (es) 2021-11-24 2024-07-29 Metagenomi Inc Sistemas de endonucleasas.
CN121241133A (zh) * 2023-03-31 2025-12-30 阿伯生物技术公司 Crispr核酸酶多肽和包含此类crispr核酸酶多肽的基因编辑系统
WO2025003344A1 (en) * 2023-06-28 2025-01-02 Alia Therapeutics Srl Type ii cas proteins and applications thereof
WO2025106894A1 (en) * 2023-11-15 2025-05-22 Profluent Bio Inc. Adenine deaminases and compositions, systems, and methods thereof
WO2025240372A1 (en) * 2024-05-13 2025-11-20 Metagenomi, Inc. Compositions and methods for editing t cells

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US11192929B2 (en) 2016-12-08 2021-12-07 Regents Of The University Of Minnesota Site-specific DNA base editing using modified APOBEC enzymes
EP3572525A4 (en) 2017-01-17 2020-09-30 Institute for Basic Science PROCEDURE FOR IDENTIFYING AN OFF-TARGET BASIC EDITING SITE BY BREAKING A SINGLE DNA STRAND
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KR102758434B1 (ko) * 2017-03-30 2025-01-21 고쿠리츠 다이가쿠 호진 교토 다이가쿠 게놈 편집에 의한 엑손 스키핑 유도 방법
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AU2018290843B2 (en) * 2017-06-26 2025-04-24 Massachusetts Institute Of Technology CRISPR/Cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
JP2021506251A (ja) * 2017-12-14 2021-02-22 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系、ならびにゲノム編集および他の適用におけるその使用
US11946040B2 (en) * 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
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EP4211234A1 (en) 2023-07-19
GB2605340A8 (en) 2024-02-21
US20230374482A1 (en) 2023-11-23
GB2605340A (en) 2022-09-28
AU2021339819A1 (en) 2023-05-25
CA3192224A1 (en) 2022-03-17
WO2022056324A1 (en) 2022-03-17
US20220364067A1 (en) 2022-11-17
GB2605340B (en) 2024-12-18
JP2023540797A (ja) 2023-09-26
MX2023002913A (es) 2023-06-01
EP4211234A4 (en) 2025-04-30
US20230348876A1 (en) 2023-11-02
GB202209534D0 (en) 2022-08-10
US12286654B2 (en) 2025-04-29

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