CN115956120A - 用于crispr基因组编辑的修饰的指导rna - Google Patents
用于crispr基因组编辑的修饰的指导rna Download PDFInfo
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- CN115956120A CN115956120A CN202180047016.6A CN202180047016A CN115956120A CN 115956120 A CN115956120 A CN 115956120A CN 202180047016 A CN202180047016 A CN 202180047016A CN 115956120 A CN115956120 A CN 115956120A
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- Prior art keywords
- crrna
- chemically modified
- guide rna
- tracrrna
- rna
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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
- 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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- 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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- 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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- 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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- 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
- C12N2310/10—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
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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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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- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Medicinal Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063023313P | 2020-05-12 | 2020-05-12 | |
| US63/023,313 | 2020-05-12 | ||
| PCT/US2021/032038 WO2021231606A2 (en) | 2020-05-12 | 2021-05-12 | Modified guide rnas for crispr genome editing |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN115956120A true CN115956120A (zh) | 2023-04-11 |
Family
ID=78524997
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202180047016.6A Pending CN115956120A (zh) | 2020-05-12 | 2021-05-12 | 用于crispr基因组编辑的修饰的指导rna |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210388348A1 (https=) |
| EP (1) | EP4150091A4 (https=) |
| JP (1) | JP2023526057A (https=) |
| CN (1) | CN115956120A (https=) |
| AU (1) | AU2021271004A1 (https=) |
| CA (1) | CA3177463A1 (https=) |
| WO (1) | WO2021231606A2 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024053964A1 (ko) * | 2022-09-06 | 2024-03-14 | 주식회사 툴젠 | 캄필로박터 제주니 유래 cas9의 가이드 rna 구조변화를 통한 유전자교정 향상 시스템 |
| IL324634A (en) | 2023-05-15 | 2026-01-01 | Nchroma Bio Inc | Compositions and methods for epigenetic regulation of hbv gene expression |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015138510A1 (en) * | 2014-03-10 | 2015-09-17 | Editas Medicine., Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| CN112877327B (zh) * | 2014-12-18 | 2024-11-22 | 综合基因技术公司 | 基于crispr的组合物和使用方法 |
| EP3250691B9 (en) * | 2015-01-28 | 2023-08-02 | Caribou Biosciences, Inc. | Crispr hybrid dna/rna polynucleotides and methods of use |
| CN107709555A (zh) * | 2015-05-15 | 2018-02-16 | 达尔马科恩有限公司 | 用于Cas9介导的基因编辑的合成的单向导RNA |
| EP4545544A3 (en) * | 2015-06-29 | 2025-10-08 | Ionis Pharmaceuticals, Inc. | Modified crispr rna and modified single crispr rna and uses thereof |
| WO2017058751A1 (en) * | 2015-09-28 | 2017-04-06 | North Carolina State University | Methods and compositions for sequence specific antimicrobials |
| CA2947904A1 (en) * | 2015-11-12 | 2017-05-12 | Pfizer Inc. | Tissue-specific genome engineering using crispr-cas9 |
| CA3034931A1 (en) * | 2016-09-23 | 2018-03-29 | Board Of Trustees Of Southern Illinois University | Tuning crispr/cas9 activity with chemically modified nucleotide substitutions |
| AU2017374044B2 (en) * | 2016-12-08 | 2023-11-30 | Intellia Therapeutics, Inc. | Modified guide RNAs |
| WO2018111946A1 (en) * | 2016-12-12 | 2018-06-21 | Integrated Dna Technologies, Inc. | Genome editing detection |
| WO2019011118A1 (zh) * | 2017-07-14 | 2019-01-17 | 苏州克睿基因生物科技有限公司 | 一种基因编辑系统及基因编辑的方法 |
| WO2019183000A1 (en) * | 2018-03-19 | 2019-09-26 | University Of Massachusetts | Modified guide rnas for crispr genome editing |
| KR20210045360A (ko) * | 2018-05-16 | 2021-04-26 | 신테고 코포레이션 | 가이드 rna 설계 및 사용을 위한 방법 및 시스템 |
| WO2019237069A1 (en) * | 2018-06-08 | 2019-12-12 | Intellia Therapeutics, Inc. | Modified guide rnas for gene editing |
-
2021
- 2021-05-12 WO PCT/US2021/032038 patent/WO2021231606A2/en not_active Ceased
- 2021-05-12 AU AU2021271004A patent/AU2021271004A1/en active Pending
- 2021-05-12 CA CA3177463A patent/CA3177463A1/en active Pending
- 2021-05-12 JP JP2022568828A patent/JP2023526057A/ja active Pending
- 2021-05-12 CN CN202180047016.6A patent/CN115956120A/zh active Pending
- 2021-05-12 US US17/318,846 patent/US20210388348A1/en active Pending
- 2021-05-12 EP EP21803427.0A patent/EP4150091A4/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2021231606A3 (en) | 2021-12-16 |
| JP2023526057A (ja) | 2023-06-20 |
| EP4150091A4 (en) | 2025-07-23 |
| CA3177463A1 (en) | 2021-11-18 |
| WO2021231606A2 (en) | 2021-11-18 |
| AU2021271004A1 (en) | 2023-01-19 |
| US20210388348A1 (en) | 2021-12-16 |
| EP4150091A2 (en) | 2023-03-22 |
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| Date | Code | Title | Description |
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| PB01 | Publication | ||
| PB01 | Publication | ||
| SE01 | Entry into force of request for substantive examination | ||
| SE01 | Entry into force of request for substantive examination | ||
| CB03 | Change of inventor or designer information | ||
| CB03 | Change of inventor or designer information |
Inventor after: Eric Joseph Sentenheimer Inventor after: Shan Tianjian Inventor after: Guitari Daifei Inventor after: Zhang Han Inventor after: Amir mill Inventor after: Anastasia Kovorova Inventor after: Jonathan Kenneth Worth Inventor after: Nadia Amrani Inventor after: Chen Zexiang Inventor after: Hassler Matthew Inventor after: Dimas Echeveria Moreno Inventor after: Julia Francis Altman Inventor after: Scott Wolf Inventor before: Eric Joseph Sentenheimer Inventor before: Shan Tianjian Inventor before: Guitari Daifei Inventor before: Zhang Han Inventor before: Anastasia Kovorova Inventor before: Jonathan Kenneth Worth Inventor before: Nadia Amrani Inventor before: Chen Zexiang Inventor before: Hassler Matthew Inventor before: Dimas Echeveria Moreno Inventor before: Julia Francis Altman Inventor before: Scott Wolf |