JP2022516647A - 非毒性ｃａｓ９酵素およびその用途 - Google Patents

非毒性ｃａｓ９酵素およびその用途 Download PDF

Info

Publication number
JP2022516647A
JP2022516647A JP2021539465A JP2021539465A JP2022516647A JP 2022516647 A JP2022516647 A JP 2022516647A JP 2021539465 A JP2021539465 A JP 2021539465A JP 2021539465 A JP2021539465 A JP 2021539465A JP 2022516647 A JP2022516647 A JP 2022516647A
Authority
JP
Japan
Prior art keywords
cells
cas9
cell
hdr
sequence
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2021539465A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022516647A5 (https=
JPWO2020146290A5 (https=
Inventor
クリストファー ハックリー，
Original Assignee
クリスプ－エイチアール セラピューティクス， インコーポレイテッド
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by クリスプ－エイチアール セラピューティクス， インコーポレイテッド filed Critical クリスプ－エイチアール セラピューティクス， インコーポレイテッド
Publication of JP2022516647A publication Critical patent/JP2022516647A/ja
Publication of JP2022516647A5 publication Critical patent/JP2022516647A5/ja
Publication of JPWO2020146290A5 publication Critical patent/JPWO2020146290A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • 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
    • C12N15/11—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
    • 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
    • C12N15/87—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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • 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)
JP2021539465A 2019-01-07 2020-01-06 非毒性ｃａｓ９酵素およびその用途 Pending JP2022516647A (ja)

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 (3)

Publication Number Publication Date
JP2022516647A true JP2022516647A (ja) 2022-03-01
JP2022516647A5 JP2022516647A5 (https=) 2023-01-19
JPWO2020146290A5 JPWO2020146290A5 (https=) 2023-01-19

Family

ID=71520905

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021539465A Pending JP2022516647A (ja) 2019-01-07 2020-01-06 非毒性ｃａｓ９酵素およびその用途

Country Status (8)

Country Link
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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025013948A1 (ja) * 2023-07-12 2025-01-16 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

Families Citing this family (11)

* 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
WO2025137069A1 (en) * 2023-12-18 2025-06-26 University Of Maryland, Baltimore Compositions and methods for enhanced genome editing using cas9 fusion proteins

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160304855A1 (en) * 2015-04-14 2016-10-20 City Of Hope Coexpression of cas9 and trex2 for targeted mutagenesis
JP2018522544A (ja) * 2015-06-12 2018-08-16 プラク・バイオケム・ビー．ブイ．Ｐｕｒａｃ Ｂｉｏｃｈｅｍ Ｂ．Ｖ． 熱安定性ｃａｓ９ヌクレアーゼ
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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4357457B1 (en) * 2012-10-23 2024-10-16 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
US9388430B2 (en) * 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150165054A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
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
CA2963820A1 (en) * 2014-11-07 2016-05-12 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
US11274288B2 (en) * 2016-02-16 2022-03-15 Emendobio Inc. Compositions and methods for promoting homology directed repair mediated gene editing
US9982267B2 (en) * 2016-10-12 2018-05-29 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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160304855A1 (en) * 2015-04-14 2016-10-20 City Of Hope Coexpression of cas9 and trex2 for targeted mutagenesis
JP2018522544A (ja) * 2015-06-12 2018-08-16 プラク・バイオケム・ビー．ブイ．Ｐｕｒａｃ Ｂｉｏｃｈｅｍ Ｂ．Ｖ． 熱安定性ｃａｓ９ヌクレアーゼ
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 (2)

* Cited by examiner, † Cited by third party
Title
CHARPENTIER, M. ET AL., NATURE COMMUNICATIONS, vol. 9, no. 1133, JPN6023050719, 19 March 2018 (2018-03-19), pages 1 - 11, ISSN: 0005214843 *
CLEMENTS, THOMAS P. ET AL., GENESIS, vol. Vol. 55, Issue 8, JPN6023050718, August 2017 (2017-08-01), pages 1 - 6, ISSN: 0005412242 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025013948A1 (ja) * 2023-07-12 2025-01-16 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

Also Published As

Publication number Publication date
EP3908659A4 (en) 2024-04-03
WO2020146290A1 (en) 2020-07-16
GB202307873D0 (en) 2023-07-12
US20210403922A1 (en) 2021-12-30
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
CN113811611A (zh) 2021-12-17
GB2596660A (en) 2022-01-05
US20240093207A1 (en) 2024-03-21
CA3125009A1 (en) 2020-07-16
GB2616539A (en) 2023-09-13

Similar Documents

Publication Publication Date Title
JP2022516647A (ja) 非毒性ｃａｓ９酵素およびその用途
JP7593920B2 (ja) 核酸構築物及び使用方法
US20210277370A1 (en) Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
EP3080260B1 (en) Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
CN116096885A (zh) 用于靶向C9orf72的组合物和方法
KR102738651B1 (ko) 간에서 목적하는 단백질 발현하기 위한 플랫폼
CN116801913A (zh) 用于靶向bcl11a的组合物和方法
WO2025061173A1 (zh) 一种融合蛋白及其应用
JP2024540390A (ja) 可動因子及びそのキメラ構築物
AU2018286393A2 (en) Genome editing system for repeat expansion mutation
US20240122989A1 (en) Methods and compositions for production of genetically modified primary cells
US20260103689A1 (en) Transposase mutant with enhanced efficiency of gene transfer
WO2023220649A2 (en) Effector protein compositions and methods of use thereof
KR20230016751A (ko) 염기 편집기 및 이의 용도
WO2025132755A1 (en) Compositions and methods for delivering transgenes
WO2024059811A2 (en) Retron directed gene editing
WO2023168235A2 (en) Systems and methods of gene editing
CN120775024A (zh) 一种提高人α-珠蛋白表达的方法及其应用
CN120189530A (zh) 基因编辑系统及其应用
WO2024091958A1 (en) Effector proteins, compositions, systems and methods for the modification of serpina1
HK40051560A (en) Nucleic acid constructs and methods of use

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230105

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230105

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231208

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240219

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240506

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240910