KR20230009379A - Cas12i2 변이체 폴리펩타이드를 포함하는 조성물 및 이의 용도 - Google Patents

Cas12i2 변이체 폴리펩타이드를 포함하는 조성물 및 이의 용도 Download PDF

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KR20230009379A
KR20230009379A KR1020227037945A KR20227037945A KR20230009379A KR 20230009379 A KR20230009379 A KR 20230009379A KR 1020227037945 A KR1020227037945 A KR 1020227037945A KR 20227037945 A KR20227037945 A KR 20227037945A KR 20230009379 A KR20230009379 A KR 20230009379A
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composition
variant
cas12i2
polypeptide
seq
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Korean (ko)
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샤오롱 총
브렌던 제이 힐버트
퀸턴 노먼 웨셀스
노아 마이클 자키모
로이 지블랫
제이슨 마이클 카르테
티아 마리 디토마소
제프리 레이먼드 하스웰
앤서니 제임스 개리티
콜린 알렉산더 맥고
데이비드 에이. 스콧
데렉 마이클 세르치오네
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아버 바이오테크놀로지스, 인크.
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Publication of KR20230009379A publication Critical patent/KR20230009379A/ko
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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
    • C12N15/11—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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    • 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]
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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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    • 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
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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
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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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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KR1020227037945A 2020-03-31 2021-03-31 Cas12i2 변이체 폴리펩타이드를 포함하는 조성물 및 이의 용도 Pending KR20230009379A (ko)

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

Publications (1)

Publication Number Publication Date
KR20230009379A true KR20230009379A (ko) 2023-01-17

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

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
WO2021243267A2 (en) * 2020-05-29 2021-12-02 Arbor Biotechnologies, Inc. Compositions comprising a cas12i2 polypeptide and uses thereof
US20230407343A1 (en) * 2020-10-30 2023-12-21 Arbor Biotechnologies, Inc. Compositions comprising an rna guide targeting pdcd1 and uses thereof
IL308806A (en) * 2021-06-01 2024-01-01 Arbor Biotechnologies Inc Gene editing systems including nuclease crisper and their uses
CA3222159A1 (en) 2021-06-04 2022-12-08 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof
US20240261435A1 (en) 2021-06-04 2024-08-08 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting transthyretin (ttr) and uses thereof
WO2022256655A2 (en) 2021-06-04 2022-12-08 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof
KR20240052763A (ko) 2021-08-11 2024-04-23 아버 바이오테크놀로지스, 인크. 스타스민 2 (stmn2)를 표적화하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도
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
WO2023034475A1 (en) 2021-09-01 2023-03-09 Arbor Biotechnologies, Inc. Cells modified by a cas12i polypeptide
US20230287456A1 (en) * 2021-09-10 2023-09-14 Arbor Biotechnologies, Inc. Compositions comprising a cas12i polypeptide and uses thereof
KR20240111314A (ko) * 2021-11-02 2024-07-16 후이다진 테라퓨틱스 (싱가포르) 피티이. 엘티디. 신규 crispr-cas12i 시스템 및 그의 용도
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)
US20250101394A1 (en) * 2022-01-24 2025-03-27 Huidagene Therapeutics (Singapore) Pte. Ltd. Novel crispr-cas12i systems and uses thereof
EP4514954A4 (en) * 2022-04-25 2026-03-25 Huidagene Therapeutics Singapore Pte Ltd NEW CRISPR-CAS12I SYSTEMS AND THEIR USES
EP4627083A1 (en) 2022-11-30 2025-10-08 Arbor Biotechnologies, Inc. Reverse transcriptase-mediated genetic editing of transthyretin (ttr) 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
AU2024369584A1 (en) 2023-10-30 2026-04-09 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

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2931897T3 (en) 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS
EP3011030B1 (en) * 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
AU2014346559B2 (en) 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
PL3765615T3 (pl) * 2018-03-14 2023-11-13 Arbor Biotechnologies, Inc. Nowe enzymy i układy crispr ukierunkowane na dna
KR20210139265A (ko) * 2019-02-13 2021-11-22 빔 테라퓨틱스, 인크. 표적 서열에서 핵염기를 변형하기 위한 아데노신 데아미나제 염기 편집기 및 이의 사용 방법
WO2024178144A1 (en) * 2023-02-22 2024-08-29 Prime Medicine, Inc. Methods and compositions for editing nucleotide sequences

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CN121380024A (zh) 2026-01-23
CO2022015170A2 (es) 2022-11-08
CN115698278B (zh) 2025-10-17
MX2022012189A (es) 2023-01-05
IL296791A (en) 2022-11-01
WO2021202800A1 (en) 2021-10-07
CN115698278A (zh) 2023-02-03
EP4127154A1 (en) 2023-02-08
CA3177749A1 (en) 2021-10-07
JP2023520504A (ja) 2023-05-17
AU2021249119A1 (en) 2022-10-20
BR112022019713A2 (pt) 2022-12-20
US20230332119A1 (en) 2023-10-19

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