JP2024524514A - ポリヌクレオチドを標的とする新規ｒｎａプログラム可能システム - Google Patents

ポリヌクレオチドを標的とする新規ｒｎａプログラム可能システム Download PDF

Info

Publication number
JP2024524514A
JP2024524514A JP2024500032A JP2024500032A JP2024524514A JP 2024524514 A JP2024524514 A JP 2024524514A JP 2024500032 A JP2024500032 A JP 2024500032A JP 2024500032 A JP2024500032 A JP 2024500032A JP 2024524514 A JP2024524514 A JP 2024524514A
Authority
JP
Japan
Prior art keywords
protein
tnpb
rna
sequence
polynucleotide
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
JP2024500032A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2023275601A5 (https=
JP2024524514A5 (https=
Inventor
シクスニス、ヴィルギニジュス
カルヴェリス、タウトヴィダス
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.)
Vilniaus Universitetas
Original Assignee
Vilniaus Universitetas
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 Vilniaus Universitetas filed Critical Vilniaus Universitetas
Publication of JP2024524514A publication Critical patent/JP2024524514A/ja
Publication of JPWO2023275601A5 publication Critical patent/JPWO2023275601A5/ja
Publication of JP2024524514A5 publication Critical patent/JP2024524514A5/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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2800/90—Vectors containing a transposable element

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2024500032A 2021-07-02 2021-07-02 ポリヌクレオチドを標的とする新規ｒｎａプログラム可能システム Pending JP2024524514A (ja)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/IB2021/055958 WO2023275601A1 (en) 2021-07-02 2021-07-02 A novel rna-programmable system for targeting polynucleotides

Publications (3)

Publication Number Publication Date
JP2024524514A true JP2024524514A (ja) 2024-07-05
JPWO2023275601A5 JPWO2023275601A5 (https=) 2024-07-12
JP2024524514A5 JP2024524514A5 (https=) 2024-07-12

Family

ID=76859656

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024500032A Pending JP2024524514A (ja) 2021-07-02 2021-07-02 ポリヌクレオチドを標的とする新規ｒｎａプログラム可能システム

Country Status (5)

Country Link
US (1) US20240368630A1 (https=)
EP (1) EP4363566A1 (https=)
JP (1) JP2024524514A (https=)
KR (1) KR20240027724A (https=)
WO (1) WO2023275601A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116355878B (zh) * 2023-02-28 2024-04-26 华中农业大学 新型TnpB编程性核酸酶及其应用
CN116355938A (zh) * 2023-03-01 2023-06-30 山东大学 基于微型RNA引导核酸酶TnpB的基因编辑载体系统及其构建方法和应用
WO2024192291A1 (en) 2023-03-15 2024-09-19 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
WO2024199134A1 (en) * 2023-03-27 2024-10-03 Beijing Astragenomics Technology Co., Ltd. Isolated nuclease and use thereof
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
CN116970590B (zh) * 2023-09-22 2024-01-30 北京科芙兰德生物科学有限责任公司 小于380个氨基酸的超级迷你型基因编辑器及其应用
WO2025117544A1 (en) * 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
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

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170321198A1 (en) * 2015-06-18 2017-11-09 The Broad Institute Inc. Novel crispr enzymes and systems
US20180320163A1 (en) * 2015-06-18 2018-11-08 The Broad Institute Inc. Novel crispr enzymes and systems
WO2021102042A1 (en) * 2019-11-19 2021-05-27 The Broad Institute, Inc. Retrotransposons and use thereof
US20210166783A1 (en) * 2016-08-17 2021-06-03 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2022159892A1 (en) * 2021-01-25 2022-07-28 The Broad Institute, Inc. Reprogrammable tnpb polypeptides and use thereof

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170321198A1 (en) * 2015-06-18 2017-11-09 The Broad Institute Inc. Novel crispr enzymes and systems
US20180320163A1 (en) * 2015-06-18 2018-11-08 The Broad Institute Inc. Novel crispr enzymes and systems
US20210166783A1 (en) * 2016-08-17 2021-06-03 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2021102042A1 (en) * 2019-11-19 2021-05-27 The Broad Institute, Inc. Retrotransposons and use thereof
WO2022159892A1 (en) * 2021-01-25 2022-07-28 The Broad Institute, Inc. Reprogrammable tnpb polypeptides and use thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NATURE BIOTECHNOLOGY, vol. 38, JPN6025028350, 2020, pages 824 - 844, ISSN: 0005642509 *

Also Published As

Publication number Publication date
EP4363566A1 (en) 2024-05-08
KR20240027724A (ko) 2024-03-04
US20240368630A1 (en) 2024-11-07
WO2023275601A1 (en) 2023-01-05

Similar Documents

Publication Publication Date Title
JP2024524514A (ja) ポリヌクレオチドを標的とする新規ｒｎａプログラム可能システム
JP7605852B2 (ja) クラスｉｉのｖ型ｃｒｉｓｐｒ系
JP7223377B2 (ja) 熱安定性ｃａｓ９ヌクレアーゼ
JP7201153B2 (ja) プログラム可能ｃａｓ９－リコンビナーゼ融合タンパク質およびその使用
CN114410625B (zh) 通过Cas9-crRNA复合物的RNA指导的DNA裂解
CN105408497B (zh) 使用截短的引导RNA(tru-gRNA)提高RNA引导的基因组编辑的特异性
KR102319192B1 (ko) Crispr 하이브리드 dna/rna 폴리뉴클레오티드 및 사용 방법
JP2023519953A (ja) クラス２のｉｉ型ｃｒｉｓｐｒシステム
CA2956224A1 (en) Cas9 proteins including ligand-dependent inteins
JP7328695B2 (ja) 安定で副作用の少ないゲノム編集用複合体及びそれをコードする核酸
CN110799525A (zh) 具有改变的PAM特异性的CPF1(CAS12a)的变体
AU2016274452A1 (en) Thermostable Cas9 nucleases
WO2014204578A1 (en) Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
JP2017500035A (ja) 遺伝子編集用のｃａｓ多様体
US20250154513A1 (en) Applications of Streptococcus-derived Cas9 nucleases on minimal Adenine-rich PAM targets
CA3144530A1 (en) Crispr type v-u1 system from mycobacterium mucogenicum and uses thereof
JP7698579B2 (ja) デフルビーモナス属（Ｄｅｆｌｕｖｉｉｍｏｎａｓ）種由来のＣａｓ９タンパク質に基づくＤＮＡカット手段
Dong et al. A single digestion, single-stranded oligonucleotide mediated PCR-independent site-directed mutagenesis method
Bigelytė Miniature CRISPR-Cas nucleases: from characterization to application
RU2791447C1 (ru) Средство разрезания ДНК на основе ScCas12a белка из бактерии Sedimentisphaera cyanobacteriorum
RU2788197C1 (ru) Средство разрезания ДНК на основе Cas9 белка из бактерии Streptococcus uberis NCTC3858
Karvelis et al. PAM recognition by miniature CRISPR nucleases triggers programmable double-stranded DNA target cleavage
Hille et al. Insights into spacer acquisition of the type VA CRISPR–Cas system of Francisella novicida U112
WO2025074246A2 (en) New crispr-cas effector proteins and the use thereof
CA2957441C (en) Genome editing using campylobacter jejuni crispr/cas system-derived rgen

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240628

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240628

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250715

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251015

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20260113