JP2023543351A - 効率的かつ特異的なゲノム編集のためのコンストラクト及びその使用 - Google Patents

効率的かつ特異的なゲノム編集のためのコンストラクト及びその使用 Download PDF

Info

Publication number
JP2023543351A
JP2023543351A JP2023542827A JP2023542827A JP2023543351A JP 2023543351 A JP2023543351 A JP 2023543351A JP 2023542827 A JP2023542827 A JP 2023542827A JP 2023542827 A JP2023542827 A JP 2023542827A JP 2023543351 A JP2023543351 A JP 2023543351A
Authority
JP
Japan
Prior art keywords
sequence
seq
nuclease
nucleic acid
amino acid
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
JP2023542827A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023543351A5 (https=
Inventor
ジェイミー カーシュナー
ロンミン リウ
リーヤー リャン
ローランド バウムガートナー
ターニャ ワルネッケ
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Artisan Development Labs Inc
Original Assignee
Artisan Development Labs Inc
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 Artisan Development Labs Inc filed Critical Artisan Development Labs Inc
Publication of JP2023543351A publication Critical patent/JP2023543351A/ja
Publication of JP2023543351A5 publication Critical patent/JP2023543351A5/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
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/70—Vectors or expression systems specially adapted for E. coli
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • 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)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
JP2023542827A 2020-09-18 2021-09-20 効率的かつ特異的なゲノム編集のためのコンストラクト及びその使用 Pending JP2023543351A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063080552P 2020-09-18 2020-09-18
US63/080,552 2020-09-18
US202163185315P 2021-05-06 2021-05-06
US63/185,315 2021-05-06
PCT/US2021/051142 WO2022061247A2 (en) 2020-09-18 2021-09-20 Constructs and uses thereof for efficient and specific genome editing

Publications (2)

Publication Number Publication Date
JP2023543351A true JP2023543351A (ja) 2023-10-13
JP2023543351A5 JP2023543351A5 (https=) 2024-09-17

Family

ID=80777856

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023542827A Pending JP2023543351A (ja) 2020-09-18 2021-09-20 効率的かつ特異的なゲノム編集のためのコンストラクト及びその使用

Country Status (6)

Country Link
US (2) US12571006B2 (https=)
EP (1) EP4214311A2 (https=)
JP (1) JP2023543351A (https=)
AU (1) AU2021343526A1 (https=)
CA (1) CA3193089A1 (https=)
WO (1) WO2022061247A2 (https=)

Families Citing this family (5)

* 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
WO2022147157A1 (en) * 2020-12-31 2022-07-07 Gigamune, Inc. Novel nucleic acid-guided nucleases
AU2022227650A1 (en) * 2021-02-25 2023-10-12 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
WO2024042168A1 (en) * 2022-08-26 2024-02-29 UCB Biopharma SRL Novel rna-guided nucleases and nucleic acid targeting systems comprising such rna-guided nucleases
IL321270A (en) * 2022-12-13 2025-08-01 Bayer Ag Engineered RNA-type programmable endonucleases and their uses

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CN108690845B (zh) * 2017-04-10 2021-04-27 中国科学院动物研究所 基因组编辑系统和方法
US20210139889A1 (en) * 2017-06-19 2021-05-13 Yale University Compositions and Methods for Multiplexed Genome Editing and Screening
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
JP7689949B2 (ja) 2019-10-03 2025-06-09 アーティサン ディベロップメント ラブズ インコーポレイテッド 操作されたデュアルガイド核酸を有するｃｒｉｓｐｒシステム
WO2021074191A1 (en) 2019-10-14 2021-04-22 KWS SAAT SE & Co. KGaA Mad7 nuclease in plants and expanding its pam recognition capability
US11453907B2 (en) * 2020-03-23 2022-09-27 The Broad Institute, Inc. Crispr effector system based coronavirus diagnostics

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
"type V CRISPR-associated protein Cas12a/Cpf1 [Phocaeicola plebeius]", DATABASE GENBANK [ONLINE], vol. WP_118425113, [retrieved on 2027-10-29], JPN6025045120, 2019, ISSN: 0005723093 *
THE CRISPR JOURNAL, vol. 2, no. 6, JPN6025045121, 2019, pages 417 - 433, ISSN: 0005723094 *

Also Published As

Publication number Publication date
AU2021343526A1 (en) 2023-05-25
US12571006B2 (en) 2026-03-10
AU2021343526A9 (en) 2024-10-03
EP4214311A2 (en) 2023-07-26
US20240011054A1 (en) 2024-01-11
CA3193089A1 (en) 2022-03-24
WO2022061247A2 (en) 2022-03-24
US20230323405A1 (en) 2023-10-12

Similar Documents

Publication Publication Date Title
US12065678B2 (en) Constructs, compositions and methods thereof having improved genome editing efficiency and specificity
AU2016244241B2 (en) Engineering of systems, methods and optimized guide compositions for sequence manipulation
ES2576126T3 (es) Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
EP2940140B1 (en) Engineering of systems, methods and optimized guide compositions for sequence manipulation
US12571006B2 (en) Constructs and uses thereof for efficient and specific genome editing
US11708588B2 (en) Gene editing
WO2020097360A1 (en) Methods and compositions for genome-wide analysis and use of genome cutting and repair
US20210309980A1 (en) Engineered chimeric nucleic acid guided nuclease constructs and uses thereof
CN116457462A (zh) 用于高效和特异性基因组编辑的构建体和其用途
US20210292747A1 (en) Engineered chimeric nucleic acid guided nucleases compositions, methods for making, and systems for gene editing
RU2796017C2 (ru) Конструирование систем, способы и оптимизированные направляющие композиции для манипуляции с последовательностями
HK1202586B (en) Engineering of systems, methods and optimized guide compositions for sequence manipulation
HK1209153B (en) Engineering of systems, methods and optimized guide compositions for sequence manipulation
HK1209154B (en) Engineering of systems, methods and optimized guide compositions for sequence manipulation

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240906

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240906

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241010

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251104

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20260428