JP2023529151A - プログラム可能なヌクレアーゼ及び使用方法 - Google Patents

プログラム可能なヌクレアーゼ及び使用方法 Download PDF

Info

Publication number
JP2023529151A
JP2023529151A JP2022574652A JP2022574652A JP2023529151A JP 2023529151 A JP2023529151 A JP 2023529151A JP 2022574652 A JP2022574652 A JP 2022574652A JP 2022574652 A JP2022574652 A JP 2022574652A JP 2023529151 A JP2023529151 A JP 2023529151A
Authority
JP
Japan
Prior art keywords
nuclease
programmable
casφ
nucleic acid
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.)
Withdrawn
Application number
JP2022574652A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023529151A5 (https=
Inventor
ルーカス ベンジャミン ハリントン
ウィリアム ダグラス ライト
ペイ－チー リュウ
ベンジャミン ユリウス ローチ
ウィプトラ ジャヤ ハルトノ
ブリジット アン ペイン マッケイ
ダヌータ サストレ フィップス
ユシュアン チェン
ネレア サンヴィセンス
ショーン チェン
デイビッド パエス－エスピノ
Original Assignee
マンモス バイオサイエンシズ 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 マンモス バイオサイエンシズ インコーポレイテッド filed Critical マンモス バイオサイエンシズ インコーポレイテッド
Publication of JP2023529151A publication Critical patent/JP2023529151A/ja
Publication of JP2023529151A5 publication Critical patent/JP2023529151A5/ja
Withdrawn legal-status Critical Current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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • C12N15/10—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
    • 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
    • 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
    • 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
    • C12N15/86—Viral vectors
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6897—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022574652A 2020-06-03 2021-06-03 プログラム可能なヌクレアーゼ及び使用方法 Withdrawn JP2023529151A (ja)

Applications Claiming Priority (17)

Application Number Priority Date Filing Date Title
US202063034346P 2020-06-03 2020-06-03
US63/034,346 2020-06-03
US202063037535P 2020-06-10 2020-06-10
US63/037,535 2020-06-10
US202063040998P 2020-06-18 2020-06-18
US63/040,998 2020-06-18
US202063092481P 2020-10-15 2020-10-15
US63/092,481 2020-10-15
US202063116083P 2020-11-19 2020-11-19
US63/116,083 2020-11-19
US202063124676P 2020-12-11 2020-12-11
US63/124,676 2020-12-11
US202163156883P 2021-03-04 2021-03-04
US63/156,883 2021-03-04
US202163178472P 2021-04-22 2021-04-22
US63/178,472 2021-04-22
PCT/US2021/035781 WO2021247924A1 (en) 2020-06-03 2021-06-03 Programmable nucleases and methods of use

Publications (2)

Publication Number Publication Date
JP2023529151A true JP2023529151A (ja) 2023-07-07
JP2023529151A5 JP2023529151A5 (https=) 2024-06-11

Family

ID=78829953

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022574652A Withdrawn JP2023529151A (ja) 2020-06-03 2021-06-03 プログラム可能なヌクレアーゼ及び使用方法

Country Status (7)

Country Link
US (3) US20230357735A1 (https=)
EP (1) EP4162052A4 (https=)
JP (1) JP2023529151A (https=)
CN (1) CN116209755A (https=)
AU (1) AU2021282578A1 (https=)
CA (1) CA3178670A1 (https=)
WO (1) WO2021247924A1 (https=)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP3830301B1 (en) 2018-08-01 2024-05-22 Mammoth Biosciences, Inc. Programmable nuclease compositions and methods of use thereof
US20240026351A1 (en) * 2020-10-30 2024-01-25 Arbor Biotechnologies, Inc. Compositions comprising an rna guide targeting trac and uses thereof
WO2023173110A1 (en) * 2022-03-11 2023-09-14 Epicrispr Biotechnologies, Inc. Compositions, systems, and methods for treating familial hypercholesterolemia by targeting pcsk9
WO2023220654A2 (en) * 2022-05-10 2023-11-16 Mammoth Biosciences, Inc. Effector protein compositions and methods of use thereof
WO2023220649A2 (en) * 2022-05-10 2023-11-16 Mammoth Biosciences, Inc. Effector protein compositions and methods of use thereof
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
WO2024047587A1 (en) * 2022-08-31 2024-03-07 Regel Therapeutics, Inc. Cas-phi compositions and methods of use
WO2024073114A1 (en) * 2022-09-30 2024-04-04 Vedabio, Inc. Delivery of therapeutics in vivo via a crispr-based cascade system
WO2024091958A1 (en) * 2022-10-25 2024-05-02 Mammoth Biosciences, Inc. Effector proteins, compositions, systems and methods for the modification of serpina1
WO2024107665A1 (en) * 2022-11-14 2024-05-23 Mammoth Biosciences, Inc. Effector proteins, compositions, systems and methods of use thereof
WO2024127370A1 (en) * 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target trac gene and methods of use
EP4637841A1 (en) * 2022-12-22 2025-10-29 Mammoth Biosciences, Inc. Compositions and methods for modifying dux4
EP4673550A2 (en) * 2023-02-27 2026-01-07 Mammoth Biosciences, Inc. Compositions and methods for the modification and regulation of liver gene expression
AU2024236558A1 (en) 2023-03-15 2025-10-09 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
WO2024238321A1 (en) * 2023-05-12 2024-11-21 Mammoth Biosciences, Inc. Compositions and methods for targeted epigenetic modification
CN116844639B (zh) * 2023-06-14 2025-09-12 上海诚明融鑫科技有限公司 一种具有自适应学习控制能力的人类mtDNA快速比对方法及装置
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025050015A1 (en) * 2023-09-01 2025-03-06 Script Biosciences, Inc. Compositions and methods for targeted modification of msh3
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
CN119351374B (zh) * 2024-12-23 2025-03-25 内蒙古大学 工程化改造超紧凑型CRISPR/Cas12j-8及其应用
CN120330238B (zh) * 2025-04-10 2025-12-23 北京林业大学 一种ect2基因在调控杨树次生木质部发育中的应用

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050108791A1 (en) * 2001-12-04 2005-05-19 Edgerton Michael D. Transgenic plants with improved phenotypes
US20110275135A1 (en) * 2010-05-05 2011-11-10 Battelle Energy Alliance, Llc Genetic elements, proteins, and associated methods including application of additional genetic information to gram (+) thermoacidophiles
WO2012164565A1 (en) * 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
WO2015161276A2 (en) * 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016380351B2 (en) * 2015-12-29 2023-04-06 Monsanto Technology Llc Novel CRISPR-associated transposases and uses thereof
EP3445853A1 (en) * 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
EP3830301B1 (en) * 2018-08-01 2024-05-22 Mammoth Biosciences, Inc. Programmable nuclease compositions and methods of use thereof
MX2021010559A (es) * 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.

Also Published As

Publication number Publication date
WO2021247924A1 (en) 2021-12-09
AU2021282578A1 (en) 2023-02-02
CN116209755A (zh) 2023-06-02
EP4162052A4 (en) 2024-07-10
US20230167454A1 (en) 2023-06-01
EP4162052A1 (en) 2023-04-12
US20230357735A1 (en) 2023-11-09
US20250066797A1 (en) 2025-02-27
CA3178670A1 (en) 2021-12-09

Similar Documents

Publication Publication Date Title
US20250066797A1 (en) Programmable nucleases and methods of use
US11814620B2 (en) Effector proteins and methods of use
US20240271113A1 (en) Effector proteins and methods of use
US20240191224A1 (en) Programmable nucleases and methods of use
US20220403357A1 (en) Small type ii cas proteins and methods of use thereof
US20240327810A1 (en) Effector proteins and methods of use
US20240191281A1 (en) Programmable nucleases and methods of use
US20240384247A1 (en) Effector proteins and uses thereof
US20230332218A1 (en) Casy programmable nucleases and rna component systems
US20240318165A1 (en) Type i-b crispr-associated transposase systems
WO2023056451A1 (en) Compositions and methods for assaying for and genotyping genetic variations
US12077775B2 (en) Effector proteins and methods of use
WO2022221581A1 (en) Programmable nucleases and methods of use
US20240191280A1 (en) Enhanced guide nucleic acids and methods of use
US20230257739A1 (en) Effector proteins and methods of use
US20250101498A1 (en) Effector proteins, compositions, systems, devices, kits and methods of use thereof
WO2024220715A2 (en) Effector proteins and uses thereof
WO2024192211A2 (en) Effector proteins and uses thereof
WO2023092136A1 (en) Effector proteins and uses thereof
WO2023220570A2 (en) Engineered cas-phi proteins and uses thereof
WO2025101679A1 (en) Effector proteins and uses thereof

Legal Events

Date Code Title Description
RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20230725

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240603

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240603

A761 Written withdrawal of application

Free format text: JAPANESE INTERMEDIATE CODE: A761

Effective date: 20240814

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20240814