KR20240055073A - 클래스 ii, v형 crispr 시스템 - Google Patents

클래스 ii, v형 crispr 시스템 Download PDF

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Publication number
KR20240055073A
KR20240055073A KR1020247011373A KR20247011373A KR20240055073A KR 20240055073 A KR20240055073 A KR 20240055073A KR 1020247011373 A KR1020247011373 A KR 1020247011373A KR 20247011373 A KR20247011373 A KR 20247011373A KR 20240055073 A KR20240055073 A KR 20240055073A
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KR
South Korea
Prior art keywords
sequence
endonuclease
engineered
nucleic acid
effector
Prior art date
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Pending
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KR1020247011373A
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English (en)
Korean (ko)
Inventor
브라이언 씨 토마스
크리스토퍼 브라운
신디 카스텔
리사 알렉산더
릴리아나 곤잘레스-오소리오
카르네발리 폴라 마테우스
돔 카스탄조
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메타지노미, 인크.
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Publication of KR20240055073A publication Critical patent/KR20240055073A/ko
Pending legal-status Critical Current

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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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • 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
    • 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
    • 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
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • 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
    • 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]
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
KR1020247011373A 2021-09-08 2022-09-06 클래스 ii, v형 crispr 시스템 Pending KR20240055073A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163241928P 2021-09-08 2021-09-08
US63/241,928 2021-09-08
PCT/US2022/075992 WO2023039378A1 (en) 2021-09-08 2022-09-06 Class ii, type v crispr systems

Publications (1)

Publication Number Publication Date
KR20240055073A true KR20240055073A (ko) 2024-04-26

Family

ID=85506852

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247011373A Pending KR20240055073A (ko) 2021-09-08 2022-09-06 클래스 ii, v형 crispr 시스템

Country Status (9)

Country Link
US (1) US20240336905A1 (https=)
EP (1) EP4399305A4 (https=)
JP (1) JP2024535672A (https=)
KR (1) KR20240055073A (https=)
CN (1) CN117999351A (https=)
AU (1) AU2022342157A1 (https=)
CA (1) CA3228222A1 (https=)
MX (1) MX2024003007A (https=)
WO (1) WO2023039378A1 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021178933A2 (en) 2020-03-06 2021-09-10 Metagenomi Ip Technologies, Llc Class ii, type v crispr systems
WO2022240858A1 (en) 2021-05-10 2022-11-17 Mammoth Biosciences, Inc. Effector proteins and methods of use
GB2625500B (en) 2021-09-21 2026-04-01 Scribe Therapeutics Inc Engineered CasX repressor systems
US20230257739A1 (en) * 2022-01-21 2023-08-17 Mammoth Biosciences, Inc. Effector proteins and methods of use
AU2023276763A1 (en) 2022-05-25 2024-12-12 Metagenomi Therapeutics, Inc. Supplementation of liver enzyme expression
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
CN119403922A (zh) * 2022-06-10 2025-02-07 拜耳公司 新的小型v型rna可编程内切核酸酶系统
CN120712347A (zh) * 2023-02-21 2025-09-26 中国农业大学 CRISPR/Cas效应蛋白及系统
WO2024187140A2 (en) * 2023-03-08 2024-09-12 Metagenomi, Inc. Class 2, type v crispr systems
WO2024196741A2 (en) * 2023-03-17 2024-09-26 Mammoth Biosciences, Inc. Crispr systems targeting the braf gene relating to the treatment of melanoma
WO2024196911A1 (en) * 2023-03-20 2024-09-26 Mammoth Biosciences, Inc. Ultracompact precision editing systems and uses thereof
WO2024254519A2 (en) * 2023-06-08 2024-12-12 Mammoth Biosciences, Inc. Compositions and methods for the modification of human genes expressed by liver cells
WO2025076084A1 (en) * 2023-10-02 2025-04-10 Acrigen Biosciences Engineered nucleases and compositions and methods thereof

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020123887A2 (en) * 2018-12-14 2020-06-18 Pioneer Hi-Bred International, Inc. Novel crispr-cas systems for genome editing
JP2024509353A (ja) * 2021-01-25 2024-03-01 ザ・ブロード・インスティテュート・インコーポレイテッド 再プログラム可能なＴｎｐＢポリペプチド及びその使用
WO2023141590A2 (en) * 2022-01-21 2023-07-27 Mammoth Biosciences, Inc. Effector proteins and methods of use
WO2023232109A1 (zh) * 2022-06-01 2023-12-07 中国科学院遗传与发育生物学研究所 新的crispr基因编辑系统
JP2025519628A (ja) * 2022-06-10 2025-06-26 アクリジェン バイオサイエンシーズ 核酸改変のための組成物及び方法

Also Published As

Publication number Publication date
MX2024003007A (es) 2024-03-25
EP4399305A1 (en) 2024-07-17
AU2022342157A1 (en) 2024-03-28
WO2023039378A1 (en) 2023-03-16
US20240336905A1 (en) 2024-10-10
CN117999351A (zh) 2024-05-07
JP2024535672A (ja) 2024-10-02
EP4399305A4 (en) 2025-10-01
CA3228222A1 (en) 2023-03-16

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