CN120290559A - 修饰的cpf1引导rna - Google Patents
修饰的cpf1引导rna Download PDFInfo
- Publication number
- CN120290559A CN120290559A CN202510240123.0A CN202510240123A CN120290559A CN 120290559 A CN120290559 A CN 120290559A CN 202510240123 A CN202510240123 A CN 202510240123A CN 120290559 A CN120290559 A CN 120290559A
- Authority
- CN
- China
- Prior art keywords
- nucleic acid
- sequence
- crrna
- cpf1
- extension
- 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
Links
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
- C12N15/11—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
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
-
- 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
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
Landscapes
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (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)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762567123P | 2017-10-02 | 2017-10-02 | |
| US62/567,123 | 2017-10-02 | ||
| US201862617138P | 2018-01-12 | 2018-01-12 | |
| US62/617,138 | 2018-01-12 | ||
| US201862697327P | 2018-07-12 | 2018-07-12 | |
| US62/697,327 | 2018-07-12 | ||
| CN201880077637.7A CN111770994B (zh) | 2017-10-02 | 2018-10-02 | 修饰的cpf1引导rna |
| PCT/US2018/054027 WO2019070762A1 (en) | 2017-10-02 | 2018-10-02 | RNA GUIDE CPF1 MODIFIED |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201880077637.7A Division CN111770994B (zh) | 2017-10-02 | 2018-10-02 | 修饰的cpf1引导rna |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN120290559A true CN120290559A (zh) | 2025-07-11 |
Family
ID=64184173
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202510240123.0A Pending CN120290559A (zh) | 2017-10-02 | 2018-10-02 | 修饰的cpf1引导rna |
| CN201880077637.7A Active CN111770994B (zh) | 2017-10-02 | 2018-10-02 | 修饰的cpf1引导rna |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201880077637.7A Active CN111770994B (zh) | 2017-10-02 | 2018-10-02 | 修饰的cpf1引导rna |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US12606823B2 (https=) |
| EP (1) | EP3692154A1 (https=) |
| JP (2) | JP2020537540A (https=) |
| KR (2) | KR102907044B1 (https=) |
| CN (2) | CN120290559A (https=) |
| AU (2) | AU2018345683B2 (https=) |
| BR (1) | BR112020006601A2 (https=) |
| CA (1) | CA3077189A1 (https=) |
| IL (2) | IL273595B2 (https=) |
| MX (1) | MX2020003339A (https=) |
| SG (1) | SG11202003083PA (https=) |
| WO (1) | WO2019070762A1 (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201616590D0 (en) | 2016-09-29 | 2016-11-16 | Oxford Nanopore Technologies Limited | Method |
| EP3596217A1 (en) | 2017-03-14 | 2020-01-22 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| EP3622070A2 (en) | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| CN120290559A (zh) | 2017-10-02 | 2025-07-11 | 基因编辑有限公司 | 修饰的cpf1引导rna |
| US11268092B2 (en) | 2018-01-12 | 2022-03-08 | GenEdit, Inc. | Structure-engineered guide RNA |
| WO2019178426A1 (en) * | 2018-03-14 | 2019-09-19 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| EP3823633A4 (en) | 2018-06-29 | 2023-05-03 | Editas Medicine, Inc. | SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO |
| GB201905651D0 (en) * | 2019-04-24 | 2019-06-05 | Lightbio Ltd | Nucleic acid constructs and methods for their manufacture |
| GB201913997D0 (en) * | 2019-09-27 | 2019-11-13 | Oxford Nanopore Tech Ltd | Method |
| EP4076479A4 (en) * | 2019-12-18 | 2024-05-22 | Editas Medicine, Inc. | GENETICALLY MODIFIED CELLS FOR THERAPY |
| CN113234701B (zh) * | 2020-10-20 | 2022-08-16 | 珠海舒桐医疗科技有限公司 | 一种Cpf1蛋白及基因编辑系统 |
| CN116848240A (zh) * | 2020-12-07 | 2023-10-03 | 因思科瑞普特公司 | 核酸引导的切口酶编辑中的gRNA稳定 |
| IL316048A (en) * | 2022-04-13 | 2024-11-01 | Caribou Biosciences Inc | Therapeutic applications of V-type CRISPR systems |
| US20240167029A1 (en) * | 2022-06-27 | 2024-05-23 | University Of Massachusetts | Modified guide rnas for crispr genome editing |
| IL324634A (en) | 2023-05-15 | 2026-01-01 | Nchroma Bio Inc | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2025212120A1 (en) * | 2024-04-05 | 2025-10-09 | Arbor Biotechnologies, Inc. | Chemical modifications of guide rnas for crispr nucleases |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4863457A (en) | 1986-11-24 | 1989-09-05 | Lee David A | Drug delivery device |
| US5378475A (en) | 1991-02-21 | 1995-01-03 | University Of Kentucky Research Foundation | Sustained release drug delivery devices |
| US5443505A (en) | 1993-11-15 | 1995-08-22 | Oculex Pharmaceuticals, Inc. | Biocompatible ocular implants |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| US20140349400A1 (en) | 2013-03-15 | 2014-11-27 | Massachusetts Institute Of Technology | Programmable Modification of DNA |
| US10563225B2 (en) | 2013-07-26 | 2020-02-18 | President And Fellows Of Harvard College | Genome engineering |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| WO2016022866A1 (en) | 2014-08-07 | 2016-02-11 | Agilent Technologies, Inc. | Cis-blocked guide rna |
| EP3183358B1 (en) | 2014-08-19 | 2020-10-07 | President and Fellows of Harvard College | Rna-guided systems for probing and mapping of nucleic acids |
| US20170306306A1 (en) | 2014-10-24 | 2017-10-26 | Life Technologies Corporation | Compositions and Methods for Enhancing Homologous Recombination |
| US10900034B2 (en) | 2014-12-03 | 2021-01-26 | Agilent Technologies, Inc. | Guide RNA with chemical modifications |
| EP3889260A1 (en) | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| AU2016261358B2 (en) | 2015-05-11 | 2021-09-16 | Editas Medicine, Inc. | Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells |
| WO2016183402A2 (en) | 2015-05-13 | 2016-11-17 | President And Fellows Of Harvard College | Methods of making and using guide rna for use with cas9 systems |
| WO2017040511A1 (en) | 2015-08-31 | 2017-03-09 | Agilent Technologies, Inc. | Compounds and methods for crispr/cas-based genome editing by homologous recombination |
| US20180237800A1 (en) | 2015-09-21 | 2018-08-23 | The Regents Of The University Of California | Compositions and methods for target nucleic acid modification |
| US20190048340A1 (en) | 2015-09-24 | 2019-02-14 | Crispr Therapeutics Ag | Novel family of rna-programmable endonucleases and their uses in genome editing and other applications |
| DK3350327T3 (en) * | 2015-10-23 | 2019-01-21 | Caribou Biosciences Inc | CONSTRUCTED CRISPR CLASS-2-NUCLEIC ACID TARGETING-NUCLEIC ACID |
| WO2017099494A1 (ko) * | 2015-12-08 | 2017-06-15 | 기초과학연구원 | Cpf1을 포함하는 유전체 교정용 조성물 및 그 용도 |
| EP3443088B1 (en) | 2016-04-13 | 2024-09-18 | Editas Medicine, Inc. | Grna fusion molecules, gene editing systems, and methods of use thereof |
| JP7184648B2 (ja) | 2016-04-29 | 2022-12-06 | ビーエーエスエフ プラント サイエンス カンパニー ゲーエムベーハー | 標的核酸の改変のための改善された方法 |
| CN106244591A (zh) * | 2016-08-23 | 2016-12-21 | 苏州吉玛基因股份有限公司 | 修饰crRNA在CRISPR/Cpf1基因编辑系统中的应用 |
| JP2019517261A (ja) | 2016-06-01 | 2019-06-24 | カーヴェーエス ザート ソシエタス・ヨーロピアＫｗｓ Ｓａａｔ Ｓｅ | ゲノム操作のためのハイブリッド核酸配列 |
| KR20190089175A (ko) | 2016-11-18 | 2019-07-30 | 진에딧 인코포레이티드 | 표적 핵산 변형을 위한 조성물 및 방법 |
| CA3062165A1 (en) * | 2017-05-04 | 2018-11-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for gene editing in t cells using crispr/cpf1 |
| CN120290559A (zh) | 2017-10-02 | 2025-07-11 | 基因编辑有限公司 | 修饰的cpf1引导rna |
-
2018
- 2018-10-02 CN CN202510240123.0A patent/CN120290559A/zh active Pending
- 2018-10-02 AU AU2018345683A patent/AU2018345683B2/en active Active
- 2018-10-02 CA CA3077189A patent/CA3077189A1/en active Pending
- 2018-10-02 WO PCT/US2018/054027 patent/WO2019070762A1/en not_active Ceased
- 2018-10-02 US US16/753,293 patent/US12606823B2/en active Active
- 2018-10-02 CN CN201880077637.7A patent/CN111770994B/zh active Active
- 2018-10-02 JP JP2020539688A patent/JP2020537540A/ja active Pending
- 2018-10-02 KR KR1020207012638A patent/KR102907044B1/ko active Active
- 2018-10-02 MX MX2020003339A patent/MX2020003339A/es unknown
- 2018-10-02 BR BR112020006601-0A patent/BR112020006601A2/pt unknown
- 2018-10-02 EP EP18799900.8A patent/EP3692154A1/en active Pending
- 2018-10-02 KR KR1020257043505A patent/KR20260011199A/ko active Pending
- 2018-10-02 IL IL273595A patent/IL273595B2/en unknown
- 2018-10-02 SG SG11202003083PA patent/SG11202003083PA/en unknown
-
2024
- 2024-05-08 JP JP2024076160A patent/JP2024116119A/ja active Pending
-
2025
- 2025-04-11 AU AU2025202594A patent/AU2025202594A1/en active Pending
- 2025-09-10 IL IL323269A patent/IL323269A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP3692154A1 (en) | 2020-08-12 |
| AU2018345683A1 (en) | 2020-04-23 |
| US12606823B2 (en) | 2026-04-21 |
| US20200299689A1 (en) | 2020-09-24 |
| IL273595B2 (en) | 2026-02-01 |
| CN111770994B (zh) | 2025-03-18 |
| IL273595B1 (en) | 2025-10-01 |
| AU2018345683B2 (en) | 2025-05-08 |
| MX2020003339A (es) | 2020-11-06 |
| CA3077189A1 (en) | 2019-04-11 |
| SG11202003083PA (en) | 2020-05-28 |
| JP2020537540A (ja) | 2020-12-24 |
| WO2019070762A1 (en) | 2019-04-11 |
| IL273595A (en) | 2020-05-31 |
| BR112020006601A2 (pt) | 2020-12-08 |
| KR102907044B1 (ko) | 2026-01-02 |
| KR20260011199A (ko) | 2026-01-22 |
| AU2025202594A1 (en) | 2025-05-22 |
| IL323269A (en) | 2025-11-01 |
| KR20200106485A (ko) | 2020-09-14 |
| CN111770994A (zh) | 2020-10-13 |
| AU2025202594A9 (en) | 2025-09-04 |
| JP2024116119A (ja) | 2024-08-27 |
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