JP2024531607A - Ｃｒｉｓｐｒヌクレアーゼを含む組成物及びその使用 - Google Patents

Ｃｒｉｓｐｒヌクレアーゼを含む組成物及びその使用 Download PDF

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Publication number
JP2024531607A
JP2024531607A JP2024515064A JP2024515064A JP2024531607A JP 2024531607 A JP2024531607 A JP 2024531607A JP 2024515064 A JP2024515064 A JP 2024515064A JP 2024515064 A JP2024515064 A JP 2024515064A JP 2024531607 A JP2024531607 A JP 2024531607A
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Prior art keywords
modification comprises
mutant
nucleic acid
polypeptide
modification
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Japanese (ja)
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JP2024531607A5 (enExample
Inventor
シャオロン チョン，
キントン ノーマン ウェッセルズ，
ロイ ジブラット，
ノア マイケル ジャキモ，
アンソニー ジェイムズ ギャリティ，
ローレン イー． アルフォンス，
Original Assignee
アーバー バイオテクノロジーズ， インコーポレイテッド
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Publication of JP2024531607A publication Critical patent/JP2024531607A/ja
Publication of JP2024531607A5 publication Critical patent/JP2024531607A5/ja
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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/25—Site-specific endodeoxyribonucleases specific for altered bases (3.1.25)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024515064A 2021-09-08 2022-09-08 Ｃｒｉｓｐｒヌクレアーゼを含む組成物及びその使用 Pending JP2024531607A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163241821P 2021-09-08 2021-09-08
US63/241,821 2021-09-08
US202263296741P 2022-01-05 2022-01-05
US63/296,741 2022-01-05
PCT/US2022/076112 WO2023039472A2 (en) 2021-09-08 2022-09-08 Compositions comprising a crispr nuclease and uses thereof

Publications (2)

Publication Number Publication Date
JP2024531607A true JP2024531607A (ja) 2024-08-29
JP2024531607A5 JP2024531607A5 (enExample) 2025-09-16

Family

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JP2024515064A Pending JP2024531607A (ja) 2021-09-08 2022-09-08 Ｃｒｉｓｐｒヌクレアーゼを含む組成物及びその使用

Country Status (7)

Country Link
US (2) US12344870B2 (enExample)
EP (1) EP4399310A4 (enExample)
JP (1) JP2024531607A (enExample)
AU (1) AU2022344256A1 (enExample)
CA (1) CA3230609A1 (enExample)
TW (1) TW202321447A (enExample)
WO (1) WO2023039472A2 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2022344256A1 (en) 2021-09-08 2024-02-29 Arbor Biotechnologies, Inc. Compositions comprising a crispr nuclease and uses thereof
KR20250151430A (ko) 2023-02-15 2025-10-21 아버 바이오테크놀로지스, 인크. 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법
WO2025171210A1 (en) 2024-02-09 2025-08-14 Arbor Biotechnologies, Inc. Compositions and methods for gene editing via homology-mediated end joining

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP3500967A1 (en) 2016-08-17 2019-06-26 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2019178427A1 (en) * 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
WO2020018142A1 (en) * 2018-07-16 2020-01-23 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
US20220049273A1 (en) 2019-03-01 2022-02-17 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
MX2022004549A (es) * 2019-10-17 2022-07-21 Pairwise Plants Services Inc Variantes de nucleasas cas12a, metodos de fabricacion y uso de las mismas.
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
AU2022344256A1 (en) 2021-09-08 2024-02-29 Arbor Biotechnologies, Inc. Compositions comprising a crispr nuclease and uses thereof

Also Published As

Publication number Publication date
US12344870B2 (en) 2025-07-01
WO2023039472A2 (en) 2023-03-16
US20250290053A1 (en) 2025-09-18
CA3230609A1 (en) 2023-03-16
US20230235304A1 (en) 2023-07-27
TW202321447A (zh) 2023-06-01
WO2023039472A3 (en) 2023-08-24
EP4399310A4 (en) 2025-07-16
EP4399310A2 (en) 2024-07-17
AU2022344256A1 (en) 2024-02-29

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