CN116209755A - 可编程核酸酶和使用方法 - Google Patents
可编程核酸酶和使用方法 Download PDFInfo
- Publication number
- CN116209755A CN116209755A CN202180056722.7A CN202180056722A CN116209755A CN 116209755 A CN116209755 A CN 116209755A CN 202180056722 A CN202180056722 A CN 202180056722A CN 116209755 A CN116209755 A CN 116209755A
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- Prior art keywords
- programmable
- nuclease
- nucleic acid
- casφ
- sequence
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- 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
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- 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
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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]
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- 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
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- 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]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
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- 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
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- 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
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- 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)
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 (1)
| Publication Number | Publication Date |
|---|---|
| CN116209755A true CN116209755A (zh) | 2023-06-02 |
Family
ID=78829953
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202180056722.7A Pending CN116209755A (zh) | 2020-06-03 | 2021-06-03 | 可编程核酸酶和使用方法 |
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=) |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN116844639A (zh) * | 2023-06-14 | 2023-10-03 | 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 |
| CN119351374A (zh) * | 2024-12-23 | 2025-01-24 | 内蒙古大学 | 工程化改造超紧凑型CRISPR/Cas12j-8及其应用 |
| CN120330238A (zh) * | 2025-04-10 | 2025-07-18 | 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 |
Families Citing this family (19)
| 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 |
| 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 |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20170175128A1 (en) * | 2014-04-18 | 2017-06-22 | Editas Medicine, Inc. | Crispr-cas-related methods, compositions and components for cancer immunotherapy |
| US20190093090A1 (en) * | 2015-12-29 | 2019-03-28 | Monsanto Technology Llc | Novel CRISPR-Associated Transposases and Uses Thereof |
| WO2020028729A1 (en) * | 2018-08-01 | 2020-02-06 | Mammoth Biosciences, Inc. | Programmable nuclease compositions and methods of use thereof |
Family Cites Families (6)
| 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 |
| US9790490B2 (en) * | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| EP3445853A1 (en) * | 2016-04-19 | 2019-02-27 | The Broad Institute, Inc. | Cpf1 complexes with reduced indel activity |
| MX2021010559A (es) * | 2019-03-07 | 2021-12-15 | Univ California | Polipéptidos efectores de crispr-cas y métodos de uso de estos. |
-
2021
- 2021-06-03 EP EP21819013.0A patent/EP4162052A4/en active Pending
- 2021-06-03 AU AU2021282578A patent/AU2021282578A1/en active Pending
- 2021-06-03 JP JP2022574652A patent/JP2023529151A/ja not_active Withdrawn
- 2021-06-03 WO PCT/US2021/035781 patent/WO2021247924A1/en not_active Ceased
- 2021-06-03 CA CA3178670A patent/CA3178670A1/en active Pending
- 2021-06-03 US US18/000,640 patent/US20230357735A1/en active Pending
- 2021-06-03 CN CN202180056722.7A patent/CN116209755A/zh active Pending
-
2022
- 2022-08-11 US US17/819,137 patent/US20230167454A1/en not_active Abandoned
-
2024
- 2024-05-23 US US18/673,055 patent/US20250066797A1/en active Pending
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20170175128A1 (en) * | 2014-04-18 | 2017-06-22 | Editas Medicine, Inc. | Crispr-cas-related methods, compositions and components for cancer immunotherapy |
| US20190093090A1 (en) * | 2015-12-29 | 2019-03-28 | Monsanto Technology Llc | Novel CRISPR-Associated Transposases and Uses Thereof |
| WO2020028729A1 (en) * | 2018-08-01 | 2020-02-06 | Mammoth Biosciences, Inc. | Programmable nuclease compositions and methods of use thereof |
Non-Patent Citations (1)
| Title |
|---|
| BASEM AL-SHAYEB: "Clades of huge phages from across Earth\'s ecosystems", NATURE., vol. 578, no. 7795, 12 February 2020 (2020-02-12), pages 425 - 431 * |
Cited By (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN116844639A (zh) * | 2023-06-14 | 2023-10-03 | 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 |
| CN119351374A (zh) * | 2024-12-23 | 2025-01-24 | 内蒙古大学 | 工程化改造超紧凑型CRISPR/Cas12j-8及其应用 |
| 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 |
| CN120330238A (zh) * | 2025-04-10 | 2025-07-18 | 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 |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2021247924A1 (en) | 2021-12-09 |
| AU2021282578A1 (en) | 2023-02-02 |
| EP4162052A4 (en) | 2024-07-10 |
| US20230167454A1 (en) | 2023-06-01 |
| EP4162052A1 (en) | 2023-04-12 |
| US20230357735A1 (en) | 2023-11-09 |
| JP2023529151A (ja) | 2023-07-07 |
| US20250066797A1 (en) | 2025-02-27 |
| CA3178670A1 (en) | 2021-12-09 |
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