JP2023520504A - Ｃａｓ１２ｉ２変異体ポリペプチドを含む組成物及びその使用 - Google Patents

Ｃａｓ１２ｉ２変異体ポリペプチドを含む組成物及びその使用 Download PDF

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JP2023520504A
JP2023520504A JP2022560154A JP2022560154A JP2023520504A JP 2023520504 A JP2023520504 A JP 2023520504A JP 2022560154 A JP2022560154 A JP 2022560154A JP 2022560154 A JP2022560154 A JP 2022560154A JP 2023520504 A JP2023520504 A JP 2023520504A
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composition
mutant
polypeptide
cas12i2
seq
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JP2023520504A5 (enExample
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シャオロン ショーン，
ブレンダン， ジェイ ヒルバート，
クイントン， ノーマン ウェッセルズ，
ノア， マイケル ジャカイモ，
ロイ ジブラット，
ジェイソン， マイケル カート，
ティア， マリー ディドマッソ，
ジェフリー， レイモンド ハスウェル，
アンソニー， ジェームス ギャリティ，
コリン， アレクグザンダー マッガァ，
デイヴィッド， エー． スコット，
デレク， マイケル セルキオン，
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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 JP2023520504A publication Critical patent/JP2023520504A/ja
Publication of JP2023520504A5 publication Critical patent/JP2023520504A5/ja
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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
    • C12N15/11—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
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • 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
    • C12N15/63—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
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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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Medicinal Chemistry (AREA)
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  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2022560154A 2020-03-31 2021-03-31 Ｃａｓ１２ｉ２変異体ポリペプチドを含む組成物及びその使用 Pending JP2023520504A (ja)

Applications Claiming Priority (37)

Application Number Priority Date Filing Date Title
US202063003090P 2020-03-31 2020-03-31
US202063002999P 2020-03-31 2020-03-31
US202063003064P 2020-03-31 2020-03-31
US63/003,064 2020-03-31
US63/003,090 2020-03-31
US63/002,999 2020-03-31
US202063012007P 2020-04-17 2020-04-17
US63/012,007 2020-04-17
US202063026562P 2020-05-18 2020-05-18
US202063026277P 2020-05-18 2020-05-18
US202063026523P 2020-05-18 2020-05-18
US202063026606P 2020-05-18 2020-05-18
US63/026,523 2020-05-18
US63/026,606 2020-05-18
US63/026,277 2020-05-18
US63/026,562 2020-05-18
US202063063819P 2020-08-10 2020-08-10
US202063063859P 2020-08-10 2020-08-10
US202063063831P 2020-08-10 2020-08-10
US202063063879P 2020-08-10 2020-08-10
US63/063,819 2020-08-10
US63/063,879 2020-08-10
US63/063,831 2020-08-10
US63/063,859 2020-08-10
US202063085752P 2020-09-30 2020-09-30
US202063085792P 2020-09-30 2020-09-30
US202063085895P 2020-09-30 2020-09-30
US202063085862P 2020-09-30 2020-09-30
US63/085,792 2020-09-30
US63/085,895 2020-09-30
US63/085,752 2020-09-30
US63/085,862 2020-09-30
US202163147850P 2021-02-10 2021-02-10
US202163147968P 2021-02-10 2021-02-10
US63/147,968 2021-02-10
US63/147,850 2021-02-10
PCT/US2021/025257 WO2021202800A1 (en) 2020-03-31 2021-03-31 Compositions comprising a cas12i2 variant polypeptide and uses thereof

Publications (2)

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JP2023520504A true JP2023520504A (ja) 2023-05-17
JP2023520504A5 JP2023520504A5 (enExample) 2024-04-11

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US (1) US20230332119A1 (enExample)
EP (1) EP4127154A1 (enExample)
JP (1) JP2023520504A (enExample)
KR (1) KR20230009379A (enExample)
CN (2) CN121380024A (enExample)
AU (1) AU2021249119A1 (enExample)
BR (1) BR112022019713A2 (enExample)
CA (1) CA3177749A1 (enExample)
CO (1) CO2022015170A2 (enExample)
IL (1) IL296791A (enExample)
MX (1) MX2022012189A (enExample)
WO (1) WO2021202800A1 (enExample)

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AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
US20230193243A1 (en) * 2020-05-29 2023-06-22 Arbor Biotechnologies, Inc. Compositions comprising a cas12i2 polypeptide and uses thereof
EP4237557A1 (en) * 2020-10-30 2023-09-06 Arbor Biotechnologies, Inc. Compositions comprising an rna guide targeting pdcd1 and uses thereof
EP4347818A2 (en) * 2021-06-01 2024-04-10 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof
IL308803A (en) 2021-06-04 2024-01-01 Arbor Biotechnologies Inc Gene editing systems comprising guide RNA targeting hydroxy acid oxidase 1 (HAO1) and uses thereof
US20230034581A1 (en) * 2021-06-04 2023-02-02 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof
WO2022256619A2 (en) 2021-06-04 2022-12-08 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting transthyretin (ttr) and uses thereof
WO2023018856A1 (en) 2021-08-11 2023-02-16 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting polypyrimidine tract binding protein 1 (ptbp1) and uses thereof
US20230203539A1 (en) 2021-08-11 2023-06-29 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof
WO2023034475A1 (en) 2021-09-01 2023-03-09 Arbor Biotechnologies, Inc. Cells modified by a cas12i polypeptide
EP4399293A4 (en) * 2021-09-10 2025-10-15 Arbor Biotechnologies Inc COMPOSITIONS COMPRISING A CAS12I POLYPEPTIDE AND USES THEREOF
CA3237337A1 (en) * 2021-11-02 2023-05-11 Huidagene Therapeutics (Singapore) Pte. Ltd. Novel crispr-cas12i systems and uses thereof
TW202334421A (zh) * 2021-11-05 2023-09-01 美商阿伯生物技術公司 包含靶向ciita之rna引導之組合物及其用途
WO2023122433A1 (en) 2021-12-22 2023-06-29 Arbor Biotechnologies, Inc. Gene editing systems targeting hydroxyacid oxidase 1 (hao1) and lactate dehydrogenase a (ldha)
CN118749029A (zh) * 2022-01-24 2024-10-08 辉大(上海)生物科技有限公司 新型CRISPR-Cas12i系统及其用途
CN118995668A (zh) * 2022-04-25 2024-11-22 辉大基因治疗(新加坡)私人有限公司 新型CRISPR-Cas12i系统及其用途
EP4627083A1 (en) 2022-11-30 2025-10-08 Arbor Biotechnologies, Inc. Reverse transcriptase-mediated genetic editing of transthyretin (ttr) and uses thereof
KR20250151430A (ko) 2023-02-15 2025-10-21 아버 바이오테크놀로지스, 인크. 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법
WO2025096594A2 (en) 2023-10-30 2025-05-08 Allogene Therapeutics, Inc. Cells expressing anti-cd19 chimeric antigen receptors and methods of use thereof
CN117431235A (zh) * 2023-11-06 2024-01-23 微光基因(苏州)有限公司 CRISPR-Cas系统及其应用
WO2025147604A1 (en) 2024-01-05 2025-07-10 Arbor Biotechnologies, Inc. Methods for treating primary hyperoxaluria via genetic editing of hydroxyacid oxidase 1
WO2025171210A1 (en) 2024-02-09 2025-08-14 Arbor Biotechnologies, Inc. Compositions and methods for gene editing via homology-mediated end joining
WO2026015829A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Small reverse transcriptases and gene editing systems comprising such
WO2026015832A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Reverse transcriptases and gene editing systems comprising such
CN120424908B (zh) * 2025-07-10 2025-09-12 南京农业大学 一种基于LAMP与CRISPR/Cas的检测方法和应用

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WO2021202800A1 (en) 2021-10-07
CN121380024A (zh) 2026-01-23
CO2022015170A2 (es) 2022-11-08
IL296791A (en) 2022-11-01
BR112022019713A2 (pt) 2022-12-20
MX2022012189A (es) 2023-01-05
CN115698278A (zh) 2023-02-03
KR20230009379A (ko) 2023-01-17
EP4127154A1 (en) 2023-02-08
CN115698278B (zh) 2025-10-17
CA3177749A1 (en) 2021-10-07
US20230332119A1 (en) 2023-10-19
AU2021249119A1 (en) 2022-10-20

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