JP2023546671A - 再プログラム可能なｉｓｃｂヌクレアーゼ及びその使用 - Google Patents

再プログラム可能なｉｓｃｂヌクレアーゼ及びその使用 Download PDF

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JP2023546671A
JP2023546671A JP2023524521A JP2023524521A JP2023546671A JP 2023546671 A JP2023546671 A JP 2023546671A JP 2023524521 A JP2023524521 A JP 2023524521A JP 2023524521 A JP2023524521 A JP 2023524521A JP 2023546671 A JP2023546671 A JP 2023546671A
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JP2023546671A5 (enExample
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トラン ハン アルタエ
ソーミャ カナン
ファトマ エスラ デミルジオール
フェン チャン
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Massachusetts Institute of Technology
Broad Institute
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Massachusetts Institute of Technology
Broad Institute
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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
    • C12N15/87—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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
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  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Chemical Or Physical Treatment Of Fibers (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Detergent Compositions (AREA)
JP2023524521A 2020-10-23 2021-10-22 再プログラム可能なｉｓｃｂヌクレアーゼ及びその使用 Pending JP2023546671A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063105191P 2020-10-23 2020-10-23
US202063105177P 2020-10-23 2020-10-23
US63/105,191 2020-10-23
US63/105,177 2020-10-23
US202163156857P 2021-03-04 2021-03-04
US63/156,857 2021-03-04
US202163195659P 2021-06-01 2021-06-01
US63/195,659 2021-06-01
US202163235583P 2021-08-20 2021-08-20
US63/235,583 2021-08-20
PCT/US2021/056361 WO2022087494A1 (en) 2020-10-23 2021-10-22 Reprogrammable iscb nucleases and uses thereof

Publications (2)

Publication Number Publication Date
JP2023546671A true JP2023546671A (ja) 2023-11-07
JP2023546671A5 JP2023546671A5 (enExample) 2024-10-25

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JP2023524521A Pending JP2023546671A (ja) 2020-10-23 2021-10-22 再プログラム可能なｉｓｃｂヌクレアーゼ及びその使用

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Country Link
US (1) US20230392131A1 (enExample)
EP (1) EP4203993A4 (enExample)
JP (1) JP2023546671A (enExample)
KR (1) KR20230111189A (enExample)
AU (1) AU2021364399A1 (enExample)
CA (1) CA3193961A1 (enExample)
IL (1) IL302101A (enExample)
WO (1) WO2022087494A1 (enExample)

Families Citing this family (35)

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US10913941B2 (en) 2019-02-14 2021-02-09 Metagenomi Ip Technologies, Llc Enzymes with RuvC domains
US20230040216A1 (en) * 2019-11-19 2023-02-09 The Broad Institute, Inc. Retrotransposons and use thereof
CA3177051A1 (en) 2020-03-31 2021-10-07 Metagenomi, Inc. Class ii, type ii crispr systems
EP4225928A4 (en) * 2020-10-09 2025-04-30 The Broad Institute, Inc. GENETIC MODIFICATION USING A HELITRON
CN119452085A (zh) 2021-08-27 2025-02-14 宏基因组学公司 具有ruvc结构域的酶
WO2023097228A1 (en) * 2021-11-23 2023-06-01 The Broad Institute, Inc. Reprogrammable iscb nucleases and uses thereof
KR20240110603A (ko) 2021-11-24 2024-07-15 메타지노미, 인크. 엔도뉴클레아제 시스템
US20250283116A1 (en) * 2022-05-06 2025-09-11 Cornell University Use of iscb in genome editing
WO2023230483A2 (en) * 2022-05-23 2023-11-30 The Broad Institute, Inc. Engineered chimeric iscb polypeptides and uses thereof
JP2025525583A (ja) 2022-07-19 2025-08-05 ランパート バイオサイエンス インコーポレイテッド 非免疫原性環状非ウイルス性ｄｎａベクター
WO2024038168A1 (en) * 2022-08-19 2024-02-22 UCB Biopharma SRL Novel rna-guided nucleases and nucleic acid targeting systems comprising such
WO2024083135A1 (en) * 2022-10-17 2024-04-25 Huidagene Therapeutics (Singapore) Pte. Ltd. Iscb polypeptides and uses thereof
WO2024094084A1 (en) * 2022-11-01 2024-05-10 Huidagene Therapeutics Co., Ltd. Iscb polypeptides and uses thereof
CN118620868A (zh) * 2023-03-07 2024-09-10 上海科技大学 IscBn-ωRNA编辑系统及其应用
WO2024192141A1 (en) 2023-03-13 2024-09-19 Dana-Farber Cancer Institute, Inc. Treatment of cancers having a drug-resistant mesenchymal cell state
WO2024192291A1 (en) 2023-03-15 2024-09-19 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
WO2024197185A1 (en) 2023-03-21 2024-09-26 The Broad Institute, Inc. Methods and compositions for dissecting organelle physiology
WO2024206825A2 (en) * 2023-03-31 2024-10-03 Aera Therapeutics, Inc. Modified iscb nucleases, modified guide rnas, and uses thereof
WO2024220715A2 (en) * 2023-04-19 2024-10-24 Mammoth Biosciences, Inc. Effector proteins and uses thereof
WO2024226838A2 (en) 2023-04-25 2024-10-31 The Brigham And Women's Hospital, Inc. Treatment of autoimmune diseases having a pathogenic t cell state
WO2025007044A1 (en) * 2023-06-28 2025-01-02 The University Of Chicago Methods and compositions comprising iscb variants
WO2025049788A1 (en) 2023-08-29 2025-03-06 The Broad Institute, Inc. Optical genetic screens of intracellular and intercellular transcriptional circuits with perturb-fish
EP4771154A2 (en) 2023-09-01 2026-07-08 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025096916A1 (en) 2023-11-03 2025-05-08 The Broad Institute, Inc. Multi-site editing in living cells
WO2025100980A1 (ko) * 2023-11-08 2025-05-15 한국과학기술원 Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도
WO2025101679A1 (en) * 2023-11-10 2025-05-15 Mammoth Biosciences, Inc. Effector proteins and uses thereof
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025149083A1 (en) * 2024-01-11 2025-07-17 Huidagene Therapeutics Co., Ltd. Iscb polypeptides and uses thereof
WO2025155923A1 (en) 2024-01-17 2025-07-24 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor
WO2025160155A1 (en) 2024-01-22 2025-07-31 The Broad Institute, Inc. Epigenetic targeting of prion diseases
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2026107393A2 (en) 2024-11-14 2026-05-21 The Broad Institute, Inc. Aav capsid modifications that reduce immunogenicity

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WO2018035250A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2020191102A1 (en) * 2019-03-18 2020-09-24 The Broad Institute, Inc. Type vii crispr proteins and systems
WO2020191249A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018035250A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2020191102A1 (en) * 2019-03-18 2020-09-24 The Broad Institute, Inc. Type vii crispr proteins and systems
WO2020191249A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences

Non-Patent Citations (2)

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Title
CURRENT OPINION IN MICROBIOLOGY, vol. 37, no. 1, JPN6025041951, 2017, pages 67 - 78, ISSN: 0005707601 *
JOURNAL OF BACTERIOLOGY, vol. 198, no. 5, JPN6025041949, 2016, pages 797 - 807, ISSN: 0005707600 *

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AU2021364399A1 (en) 2023-05-11
IL302101A (en) 2023-06-01
KR20230111189A (ko) 2023-07-25
EP4203993A1 (en) 2023-07-05
CA3193961A1 (en) 2022-04-28
AU2021364399A9 (en) 2023-07-06
WO2022087494A1 (en) 2022-04-28
US20230392131A1 (en) 2023-12-07
EP4203993A4 (en) 2024-10-23

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