JP2023540493A - Ｒｎａ誘導型ｒｎａターゲティングｃｒｉｓｐｒエフェクターのための系、方法及び組成物 - Google Patents

Ｒｎａ誘導型ｒｎａターゲティングｃｒｉｓｐｒエフェクターのための系、方法及び組成物 Download PDF

Info

Publication number
JP2023540493A
JP2023540493A JP2023514409A JP2023514409A JP2023540493A JP 2023540493 A JP2023540493 A JP 2023540493A JP 2023514409 A JP2023514409 A JP 2023514409A JP 2023514409 A JP2023514409 A JP 2023514409A JP 2023540493 A JP2023540493 A JP 2023540493A
Authority
JP
Japan
Prior art keywords
amino acids
rna
cell
discas7
cas7
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
JP2023514409A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023540493A5 (https=
Inventor
アブディエ，オマー
グーテンバーグ，ジョナサン
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.)
Massachusetts Institute of Technology
Original Assignee
Massachusetts Institute of Technology
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 Massachusetts Institute of Technology filed Critical Massachusetts Institute of Technology
Publication of JP2023540493A publication Critical patent/JP2023540493A/ja
Publication of JP2023540493A5 publication Critical patent/JP2023540493A5/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
    • C12N15/10—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
    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • 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
    • C12N2320/10—Applications; Uses in screening processes
    • 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
    • C12N2320/33—Alteration of splicing
    • 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
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6816—Hybridisation assays characterised by the detection means
    • C12Q1/6825—Nucleic acid detection involving sensors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023514409A 2020-09-02 2021-07-01 Ｒｎａ誘導型ｒｎａターゲティングｃｒｉｓｐｒエフェクターのための系、方法及び組成物 Pending JP2023540493A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063073898P 2020-09-02 2020-09-02
US63/073,898 2020-09-02
US202163208606P 2021-06-09 2021-06-09
US63/208,606 2021-06-09
PCT/US2021/040160 WO2022051020A2 (en) 2020-09-02 2021-07-01 Systems, methods, and compositions for rna-guided rna-targeting crispr effectors

Publications (2)

Publication Number Publication Date
JP2023540493A true JP2023540493A (ja) 2023-09-25
JP2023540493A5 JP2023540493A5 (https=) 2024-07-09

Family

ID=77104154

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023514409A Pending JP2023540493A (ja) 2020-09-02 2021-07-01 Ｒｎａ誘導型ｒｎａターゲティングｃｒｉｓｐｒエフェクターのための系、方法及び組成物

Country Status (6)

Country Link
US (2) US12012621B2 (https=)
EP (1) EP4208544A2 (https=)
JP (1) JP2023540493A (https=)
AU (1) AU2021336681A1 (https=)
CA (1) CA3190991A1 (https=)
WO (1) WO2022051020A2 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023215751A1 (en) * 2022-05-02 2023-11-09 The Broad Institute, Inc. Programmable nuclease-peptidase compositions
US20250283112A1 (en) * 2022-05-13 2025-09-11 The Regents Of The University Of California Compositions and Methods for RNA Editing
EP4532525A1 (en) * 2022-05-25 2025-04-09 Massachusetts Institute Of Technology Systems, methods, and compositions for rna-guided rna-targeting crispr effectors with cas7-11 variants
EP4536851A1 (en) * 2022-06-13 2025-04-16 Board of Regents, The University of Texas System Type iii-d crispr-cas system and uses thereof
US20240229023A9 (en) * 2022-08-16 2024-07-11 Massachusetts Institute Of Technology Rna-triggered protein cleavage and applications by the crispr cas7-11-csx29 complex
CN116411048B (zh) * 2023-02-14 2024-12-20 重庆大学 一种基于CRISPR/Cas12a的多核苷酸激酶活性检测方法
WO2024227050A2 (en) * 2023-04-26 2024-10-31 The Regents Of The University Of California Engineered type iii rna-targeting crispr effectors
WO2025006808A1 (en) * 2023-06-27 2025-01-02 Montana State University Rna editing in living cells
CN116854787B (zh) * 2023-09-05 2023-11-07 四川大学 一种Cas7-11蛋白的抑制剂及其应用
CN118086259B (zh) * 2024-04-26 2024-07-16 四川大学 抑制Cas7-11酶活的蛋白及其应用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11898141B2 (en) 2014-05-27 2024-02-13 The Broad Institute, Inc. High-throughput assembly of genetic elements
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2018338318B2 (en) * 2017-09-21 2022-12-22 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
WO2019222555A1 (en) 2018-05-16 2019-11-21 Arbor Biotechnologies, Inc. Novel crispr-associated systems and components

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NATURE REVIEWS MICROBIOLOGY, JPN6024031827, ISSN: 0005525984 *

Also Published As

Publication number Publication date
US20220073891A1 (en) 2022-03-10
WO2022051020A9 (en) 2022-06-02
WO2022051020A2 (en) 2022-03-10
US12012621B2 (en) 2024-06-18
EP4208544A2 (en) 2023-07-12
CA3190991A1 (en) 2022-03-10
AU2021336681A1 (en) 2023-03-30
WO2022051020A3 (en) 2022-04-14
US20240294890A1 (en) 2024-09-05

Similar Documents

Publication Publication Date Title
US12012621B2 (en) Systems, methods, and compositions for RNA-guided RNA-targeting CRISPR effectors
JP7585373B2 (ja) 変更ＰＡＭ特異性を有する遺伝子操作ＣＲＩＳＰＲ－Ｃａｓ９ヌクレアーゼ
US20250154483A1 (en) Novel crispr dna and rna targeting enzymes and systems
JP6896786B2 (ja) 配列操作のためのＣＲＩＳＰＲ−Ｃａｓ成分系、方法および組成物
US11788088B2 (en) CRISPR/Cas system and method for genome editing and modulating transcription
CN110892069B (zh) 基于基因组编辑的外显子跳跃诱导方法
JP7223377B2 (ja) 熱安定性ｃａｓ９ヌクレアーゼ
Byrne et al. Genome editing in human stem cells
Ran et al. Genome engineering using the CRISPR-Cas9 system
Lei et al. APOBEC3 induces mutations during repair of CRISPR–Cas9-generated DNA breaks
Trevino et al. Genome editing using Cas9 nickases
Ranganathan et al. Expansion of the CRISPR–Cas9 genome targeting space through the use of H1 promoter-expressed guide RNAs
Fernandez et al. Optimized CRISPR-Cpf1 system for genome editing in zebrafish
WO2017181107A2 (en) Modified cpf1 mrna, modified guide rna, and uses thereof
CN105408497A (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
EP3940078A1 (en) Off-target single nucleotide variants caused by single-base editing and high-specificity off-target-free single-base gene editing tool
CA3093580A1 (en) Novel crispr dna and rna targeting enzymes and systems
Brok-Volchanskaya et al. Phage T4 SegB protein is a homing endonuclease required for the preferred inheritance of T4 tRNA gene region occurring in co-infection with a related phage
WO2023230498A1 (en) Systems, methods, and compositions for rna-guided rna-targeting crispr effectors with cas7-11 variants
Ahmad et al. Functional analysis of the NucS/EndoMS of the hyperthermophilic archaeon Sulfolobus islandicus REY15A
Wörtz et al. Cas1 and Fen1 display equivalent functions during archaeal DNA repair
Gentry et al. An 11bp region with stem formation potential is essential for de novo DNA methylation of the RPS element
Anandavijayan Molecular biology of human DDX49-novel biochemical activities
Singh Reprogramming the genome: CRISPR-cas-based human disease therapy
Hu Genome engineering using the crisper-cas9 system

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240701

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240701

A871 Explanation of circumstances concerning accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A871

Effective date: 20240701

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240813

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241113

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250212

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250312

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250808

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20251111