CN113811611A - 无毒cas9酶及其应用 - Google Patents

无毒cas9酶及其应用 Download PDF

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CN113811611A
CN113811611A CN202080019483.3A CN202080019483A CN113811611A CN 113811611 A CN113811611 A CN 113811611A CN 202080019483 A CN202080019483 A CN 202080019483A CN 113811611 A CN113811611 A CN 113811611A
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leu
glu
cells
ile
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克里斯多佛·哈克利
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Christopher Hull Therapy
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Christopher Hull Therapy
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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
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • 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
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Genetics & Genomics (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN202080019483.3A 2019-01-07 2020-01-06 无毒cas9酶及其应用 Pending CN113811611A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962789347P 2019-01-07 2019-01-07
US62/789,347 2019-01-07
US201962823477P 2019-03-25 2019-03-25
US62/823,477 2019-03-25
US201962824164P 2019-03-26 2019-03-26
US62/824,164 2019-03-26
US201962855612P 2019-05-31 2019-05-31
US62/855,612 2019-05-31
PCT/US2020/012438 WO2020146290A1 (en) 2019-01-07 2020-01-06 A non-toxic cas9 enzyme and application thereof

Publications (1)

Publication Number Publication Date
CN113811611A true CN113811611A (zh) 2021-12-17

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ID=71520905

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080019483.3A Pending CN113811611A (zh) 2019-01-07 2020-01-06 无毒cas9酶及其应用

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US (3) US20210403922A1 (https=)
EP (1) EP3908659A4 (https=)
JP (1) JP2022516647A (https=)
CN (1) CN113811611A (https=)
AU (1) AU2020206997A1 (https=)
CA (1) CA3125009A1 (https=)
GB (2) GB2596660B (https=)
WO (1) WO2020146290A1 (https=)

Families Citing this family (12)

* 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
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
EP4243608A1 (en) * 2020-11-11 2023-09-20 Leibniz-Institut für Pflanzenbiochemie (IPB) Fusion protein for editing endogenous dna of a eukaryotic cell
AU2021409732A1 (en) * 2020-12-23 2023-07-20 Intellia Therapeutics, Inc. Compositions and methods for reducing hla-a in a cell
EP4317549A4 (en) * 2021-03-31 2025-03-26 Ddbio. Co, Ltd., (Shang Hai) Vector and method for screening functional antigen-binding protein
CN113481184A (zh) * 2021-08-06 2021-10-08 北京大学 融合蛋白以及其使用方法
EP4396340A4 (en) * 2021-09-01 2025-09-24 Univ Leland Stanford Junior RNA-GUIDED GENOME RECOMBINATION AT THE KILOBASE SCALE
WO2023168235A2 (en) * 2022-03-01 2023-09-07 Crisp-Hr Therapeutics, Inc. Systems and methods of gene editing
CN116286741A (zh) * 2022-03-03 2023-06-23 清华大学 5’→3’核酸外切酶在基因编辑系统中的用途和基因编辑系统及其编辑方法
EP4286401A1 (en) * 2022-06-03 2023-12-06 Technische Universität Dresden Use of an artificial protein or of a nucleic acid encoding the artificial protein for providing a functional ribonucleoprotein complex
JP2025012122A (ja) * 2023-07-12 2025-01-24 国立研究開発法人農業・食品産業技術総合研究機構 遺伝子組換えチョウ目昆虫
WO2025137069A1 (en) * 2023-12-18 2025-06-26 University Of Maryland, Baltimore Compositions and methods for enhanced genome editing using cas9 fusion proteins

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150071898A1 (en) * 2013-09-06 2015-03-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150344912A1 (en) * 2012-10-23 2015-12-03 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
CN105934516A (zh) * 2013-12-12 2016-09-07 哈佛大学的校长及成员们 用于基因编辑的cas变体
US20170175140A1 (en) * 2015-12-16 2017-06-22 Regents Of The University Of Minnesota Methods for using a 5'-exonuclease to increase homologous recombination in eukaryotic cells
WO2017142923A1 (en) * 2016-02-16 2017-08-24 Emendobio Inc. Compositions and methods for promoting homology directed repair mediated gene editing
US20180100158A1 (en) * 2016-10-12 2018-04-12 Feldan Bio Inc. Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same
WO2018162702A1 (en) * 2017-03-10 2018-09-13 Institut National De La Sante Et De La Recherche Medicale (Inserm) Nuclease fusions for enhancing genome editing by homology-directed transgene integration
US20180298392A1 (en) * 2014-11-07 2018-10-18 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing

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WO2016007604A1 (en) * 2014-07-09 2016-01-14 Gen9, Inc. Compositions and methods for site-directed dna nicking and cleaving
EP3204496A1 (en) * 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
US10155938B2 (en) * 2015-04-14 2018-12-18 City Of Hope Coexpression of CAS9 and TREX2 for targeted mutagenesis
GB201510296D0 (en) * 2015-06-12 2015-07-29 Univ Wageningen Thermostable CAS9 nucleases

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150344912A1 (en) * 2012-10-23 2015-12-03 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
US20150071898A1 (en) * 2013-09-06 2015-03-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
CN105934516A (zh) * 2013-12-12 2016-09-07 哈佛大学的校长及成员们 用于基因编辑的cas变体
US20180298392A1 (en) * 2014-11-07 2018-10-18 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
US20170175140A1 (en) * 2015-12-16 2017-06-22 Regents Of The University Of Minnesota Methods for using a 5'-exonuclease to increase homologous recombination in eukaryotic cells
WO2017142923A1 (en) * 2016-02-16 2017-08-24 Emendobio Inc. Compositions and methods for promoting homology directed repair mediated gene editing
US20180100158A1 (en) * 2016-10-12 2018-04-12 Feldan Bio Inc. Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same
WO2018162702A1 (en) * 2017-03-10 2018-09-13 Institut National De La Sante Et De La Recherche Medicale (Inserm) Nuclease fusions for enhancing genome editing by homology-directed transgene integration

Non-Patent Citations (3)

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Title
M. CHARPENTIER ET AL.: ""CtIP fusion to Cas9 enhances transgene integration by homology-dependent repair"", 《NATURE COMMUNICATIONS 》, vol. 9, no. 1133, 19 March 2018 (2018-03-19), pages 1 - 11 *
THOMAS P. CLEMENTS ET AL.: ""RICE CRISPR: Rapidly increased cut ends by an exonuclease Cas9 fusion in zebrafish"", 《GENESIS》, vol. 55, 31 December 2017 (2017-12-31), pages 1 - 6 *
翁震等: ""CRISPR-Cas9 技术在非肿瘤性血液病中的应用"", 《上海交通大学学报（医学版）》, vol. 38, no. 11, 30 November 2018 (2018-11-30), pages 1396 - 1400 *

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EP3908659A4 (en) 2024-04-03
WO2020146290A1 (en) 2020-07-16
GB202307873D0 (en) 2023-07-12
US20210403922A1 (en) 2021-12-30
JP2022516647A (ja) 2022-03-01
US20240043851A1 (en) 2024-02-08
EP3908659A1 (en) 2021-11-17
GB2616539B (en) 2023-12-20
AU2020206997A1 (en) 2021-08-26
GB2596660B (en) 2023-09-13
GB202110538D0 (en) 2021-09-08
GB2596660A (en) 2022-01-05
US20240093207A1 (en) 2024-03-21
CA3125009A1 (en) 2020-07-16
GB2616539A (en) 2023-09-13

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