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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Japan
Prior art keywords
modification comprises
mutant
nucleic acid
polypeptide
modification
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Pending
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JP2024515064A
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Japanese (ja)
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JP2024531607A5 (https=
JPWO2023039472A5 (https=
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
Publication of JPWO2023039472A5 publication Critical patent/JPWO2023039472A5/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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (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 (3)

Publication Number Publication Date
JP2024531607A true JP2024531607A (ja) 2024-08-29
JP2024531607A5 JP2024531607A5 (https=) 2025-09-16
JPWO2023039472A5 JPWO2023039472A5 (https=) 2025-09-16

Family

ID=85506919

Family Applications (1)

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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 (https=)
EP (1) EP4399310A4 (https=)
JP (1) JP2024531607A (https=)
AU (1) AU2022344256A1 (https=)
CA (1) CA3230609A1 (https=)
TW (1) TW202321447A (https=)
WO (1) WO2023039472A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3230609A1 (en) 2021-09-08 2023-03-16 Shaorong Chong Compositions comprising a crispr nuclease and uses thereof
WO2024173645A1 (en) 2023-02-15 2024-08-22 Arbor Biotechnologies, Inc. Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript
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
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US12431216B2 (en) 2016-08-17 2025-09-30 Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
PL3765615T3 (pl) * 2018-03-14 2023-11-13 Arbor Biotechnologies, Inc. Nowe enzymy i układy crispr ukierunkowane na dna
US12553045B2 (en) 2018-07-16 2026-02-17 Arbor Biotechnologies, Inc. Crispr DNA targeting enzymes and systems
US20220049273A1 (en) 2019-03-01 2022-02-17 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
BR112022007125A2 (pt) * 2019-10-17 2022-07-05 Pairwise Plants Services Inc Variantes de nucleases cas12a e métodos de produção e uso das mesmas
CN115052986A (zh) * 2019-12-04 2022-09-13 阿伯生物技术公司 包含核酸酶的组合物及其用途
CA3230609A1 (en) 2021-09-08 2023-03-16 Shaorong Chong Compositions comprising a crispr nuclease and uses thereof

Also Published As

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

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