CA3128283A1 - Inhibition of unintended mutations in gene editing - Google Patents

Inhibition of unintended mutations in gene editing Download PDF

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Publication number
CA3128283A1
CA3128283A1 CA3128283A CA3128283A CA3128283A1 CA 3128283 A1 CA3128283 A1 CA 3128283A1 CA 3128283 A CA3128283 A CA 3128283A CA 3128283 A CA3128283 A CA 3128283A CA 3128283 A1 CA3128283 A1 CA 3128283A1
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Prior art keywords
deaminase
fragment
seq
fusion protein
guide rna
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Pending
Application number
CA3128283A
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English (en)
French (fr)
Inventor
Jia Chen
Bei YANG
Li Yang
Xingxu HUANG
Lijie Wang
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ShanghaiTech University
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ShanghaiTech University
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Publication of CA3128283A1 publication Critical patent/CA3128283A1/en
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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
    • 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
    • C12N15/11—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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/503—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from viruses
    • C12N9/506—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from viruses derived from RNA viruses
    • 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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21111—Aqualysin 1 (3.4.21.111)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/22—Cysteine endopeptidases (3.4.22)
    • C12Y304/22044—Nuclear-inclusion-a endopeptidase (3.4.22.44)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3128283A 2019-02-02 2020-02-03 Inhibition of unintended mutations in gene editing Pending CA3128283A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2019074577 2019-02-02
CNPCT/CN2019/074577 2019-02-02
PCT/CN2020/074218 WO2020156575A1 (en) 2019-02-02 2020-02-03 Inhibition of unintended mutations in gene editing

Publications (1)

Publication Number Publication Date
CA3128283A1 true CA3128283A1 (en) 2020-08-06

Family

ID=71841894

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3128283A Pending CA3128283A1 (en) 2019-02-02 2020-02-03 Inhibition of unintended mutations in gene editing

Country Status (12)

Country Link
US (3) US11384353B2 (https=)
EP (1) EP3918083A4 (https=)
JP (3) JP7518549B2 (https=)
KR (1) KR20210126042A (https=)
CN (4) CN116656676A (https=)
AU (3) AU2020214090B2 (https=)
BR (1) BR112021015092A2 (https=)
CA (1) CA3128283A1 (https=)
IL (1) IL285194A (https=)
SG (1) SG11202108303SA (https=)
WO (1) WO2020156575A1 (https=)
ZA (2) ZA202105895B (https=)

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KR20250126155A (ko) 2022-11-04 2025-08-22 라이프 에디트 테라퓨틱스, 인크. 내부 삽입 부위를 갖는 진화된 아데닌 데아미나제 및 rna-가이드 뉴클레아제 융합 단백질 및 사용 방법
WO2024109745A1 (en) * 2022-11-21 2024-05-30 CorrectSequence Therapeutics Co., Ltd Gene editing systems and methods for treating hbv infection
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CN116286740A (zh) * 2023-02-24 2023-06-23 四川大学华西医院 碱基编辑器和引导编辑器及构建与应用
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WO2025061147A1 (en) * 2023-09-21 2025-03-27 Shanghaitech University Rna editing systems of high efficiency and specificity
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WO2025083619A1 (en) 2023-10-18 2025-04-24 Life Edit Therapeutics, Inc. Rna-guided nucleases and acive fragments and variants thereof and methods of use
CN117092084B (zh) * 2023-10-20 2024-01-12 浙江迪福润丝生物科技有限公司 Wnv蛋白酶抑制剂的筛选方法和抑制效果评价方法
WO2025140399A1 (en) * 2023-12-26 2025-07-03 Correctsequence Therapeutics (Shanghai) Co., Ltd Gene editing systems and methods for treating cardiovascular disease
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CN116656676A (zh) 2023-08-29
AU2020214090B2 (en) 2022-09-15
CN113661248A (zh) 2021-11-16
WO2020156575A1 (en) 2020-08-06
JP2024109660A (ja) 2024-08-14
SG11202108303SA (en) 2021-08-30
AU2022287550B2 (en) 2025-07-24
AU2022287550A1 (en) 2023-02-02
ZA202212490B (en) 2024-09-25
US11384353B2 (en) 2022-07-12
JP7518549B2 (ja) 2024-07-18
AU2025256226A1 (en) 2025-11-13
JP7761294B2 (ja) 2025-10-28
AU2020214090A1 (en) 2021-09-16
US20230094769A1 (en) 2023-03-30
CN116355966A (zh) 2023-06-30
JP2026012727A (ja) 2026-01-27
KR20210126042A (ko) 2021-10-19
CN116286802A (zh) 2023-06-23
CN113661248B (zh) 2022-09-16
JP2022526695A (ja) 2022-05-26
ZA202105895B (en) 2024-07-31
BR112021015092A2 (pt) 2022-10-11
IL285194A (en) 2021-09-30
US20220064626A1 (en) 2022-03-03
US20240026342A1 (en) 2024-01-25
EP3918083A1 (en) 2021-12-08
EP3918083A4 (en) 2023-03-29
US11840685B2 (en) 2023-12-12

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