CN116656676A - 一对用于编辑人pcsk9核酸序列的辅助引导rna/引导rna - Google Patents
一对用于编辑人pcsk9核酸序列的辅助引导rna/引导rna Download PDFInfo
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- CN116656676A CN116656676A CN202211043562.5A CN202211043562A CN116656676A CN 116656676 A CN116656676 A CN 116656676A CN 202211043562 A CN202211043562 A CN 202211043562A CN 116656676 A CN116656676 A CN 116656676A
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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
- 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
- C12N15/1137—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 against enzymes
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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
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- C12N15/111—General methods applicable to biologically active non-coding 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
- 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
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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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
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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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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/503—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from viruses
- C12N9/506—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from viruses derived from RNA viruses
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- 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)
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- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/22—Cysteine endopeptidases (3.4.22)
- C12Y304/22044—Nuclear-inclusion-a endopeptidase (3.4.22.44)
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- 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)
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- 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)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/50—Fusion polypeptide containing protease site
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/85—Fusion polypeptide containing an RNA binding domain
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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
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- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21111—Aqualysin 1 (3.4.21.111)
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- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
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- General Engineering & Computer Science (AREA)
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- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Mycology (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN2019074577 | 2019-02-02 | ||
| CNPCT/CN2019/074577 | 2019-02-02 | ||
| CN202080026986.3A CN113661248B (zh) | 2019-02-02 | 2020-02-03 | 基因编辑中非预期突变的抑制 |
| PCT/CN2020/074218 WO2020156575A1 (en) | 2019-02-02 | 2020-02-03 | Inhibition of unintended mutations in gene editing |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202080026986.3A Division CN113661248B (zh) | 2019-02-02 | 2020-02-03 | 基因编辑中非预期突变的抑制 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN116656676A true CN116656676A (zh) | 2023-08-29 |
Family
ID=71841894
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202211043562.5A Pending CN116656676A (zh) | 2019-02-02 | 2020-02-03 | 一对用于编辑人pcsk9核酸序列的辅助引导rna/引导rna |
| CN202211057011.4A Pending CN116355966A (zh) | 2019-02-02 | 2020-02-03 | 一种融合蛋白在遗传编辑的用途 |
| CN202080026986.3A Active CN113661248B (zh) | 2019-02-02 | 2020-02-03 | 基因编辑中非预期突变的抑制 |
| CN202211042699.9A Pending CN116286802A (zh) | 2019-02-02 | 2020-02-03 | 一种包含支架的引导rna及利用其的遗传编辑的方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202211057011.4A Pending CN116355966A (zh) | 2019-02-02 | 2020-02-03 | 一种融合蛋白在遗传编辑的用途 |
| CN202080026986.3A Active CN113661248B (zh) | 2019-02-02 | 2020-02-03 | 基因编辑中非预期突变的抑制 |
| CN202211042699.9A Pending CN116286802A (zh) | 2019-02-02 | 2020-02-03 | 一种包含支架的引导rna及利用其的遗传编辑的方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (3) | US11384353B2 (https=) |
| EP (1) | EP3918083A4 (https=) |
| JP (3) | JP7518549B2 (https=) |
| KR (1) | KR20210126042A (https=) |
| CN (4) | CN116656676A (https=) |
| AU (3) | AU2020214090B2 (https=) |
| BR (1) | BR112021015092A2 (https=) |
| CA (1) | CA3128283A1 (https=) |
| IL (1) | IL285194A (https=) |
| SG (1) | SG11202108303SA (https=) |
| WO (1) | WO2020156575A1 (https=) |
| ZA (2) | ZA202105895B (https=) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN118995709A (zh) * | 2024-08-23 | 2024-11-22 | 东北农业大学 | 一种提高基因编辑效率的重组型pegRNA及其应用 |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN117120621A (zh) * | 2020-11-12 | 2023-11-24 | 上海科技大学 | 提高效率和准确性的基因组编辑 |
| CN112575014B (zh) * | 2020-12-11 | 2022-04-01 | 安徽省农业科学院水稻研究所 | 一种碱基编辑器SpCas9-LjCDAL1及其构建和应用 |
| CN115161316B (zh) * | 2021-04-02 | 2025-12-23 | 上海科技大学 | 一种引导编辑工具、融合rna及其用途 |
| WO2022206986A1 (en) * | 2021-04-02 | 2022-10-06 | Shanghaitech University | Gene therapy for treating beta-hemoglobinopathies |
| EP4330389A4 (en) * | 2021-04-30 | 2025-04-30 | Alnylam Pharmaceuticals, Inc. | RISC REORIENTATION FOR RNA EDITING |
| US12123006B2 (en) * | 2021-05-18 | 2024-10-22 | Shanghaitech University | Base editing tool and use thereof |
| WO2023283092A1 (en) * | 2021-07-06 | 2023-01-12 | Prime Medicine, Inc. | Compositions and methods for efficient genome editing |
| WO2023088440A1 (en) * | 2021-11-18 | 2023-05-25 | Correctsequence Therapeutics | Regeneration of surface antigen-negative cells |
| EP4477759A4 (en) * | 2022-02-08 | 2025-12-31 | Toolgen Inc | A method for predicting off-target outcomes that can occur in a genome editing process using a primary editing system. |
| CN119013310A (zh) * | 2022-02-17 | 2024-11-22 | 正序(上海)生物科技有限公司 | 具有改善的编辑精确度的突变的胞嘧啶脱氨酶 |
| GB2634837A (en) | 2022-06-07 | 2025-04-23 | Scribe Therapeutics Inc | Compositions and methods for the targeting of PCSK9 |
| WO2024012435A1 (en) | 2022-07-11 | 2024-01-18 | CorrectSequence Therapeutics Co., Ltd | Gene editing systems and methods for treating hereditary angioedema |
| CA3265664A1 (en) | 2022-08-25 | 2024-02-29 | Life Edit Therapeutics, Inc. | CHEMICAL MODIFICATION OF GUIDE RNA WITH A LOCKED NUCLEIC ACID FOR RNA-GUIDED NUCLEASE-MEDIATED GENE EDITING |
| WO2024088401A1 (en) * | 2022-10-28 | 2024-05-02 | CorrectSequence Therapeutics Co., Ltd | Gene editing systems and methods for reducing immunogenicity and graft versus host response |
| KR20250126155A (ko) | 2022-11-04 | 2025-08-22 | 라이프 에디트 테라퓨틱스, 인크. | 내부 삽입 부위를 갖는 진화된 아데닌 데아미나제 및 rna-가이드 뉴클레아제 융합 단백질 및 사용 방법 |
| WO2024109745A1 (en) * | 2022-11-21 | 2024-05-30 | CorrectSequence Therapeutics Co., Ltd | Gene editing systems and methods for treating hbv infection |
| WO2024112957A1 (en) * | 2022-11-23 | 2024-05-30 | Amber Bio Inc. | Gene-modifying endonucleases |
| EP4634383A1 (en) | 2022-12-16 | 2025-10-22 | Life Edit Therapeutics, Inc. | Guide rnas that target foxp3 gene and methods of use |
| WO2024127370A1 (en) | 2022-12-16 | 2024-06-20 | LifeEDIT Therapeutics, Inc. | Guide rnas that target trac gene and methods of use |
| WO2024178397A2 (en) | 2023-02-24 | 2024-08-29 | Elevatebio Technologies, Inc. | Modified immune effector cells and methods of use |
| CN116286740A (zh) * | 2023-02-24 | 2023-06-23 | 四川大学华西医院 | 碱基编辑器和引导编辑器及构建与应用 |
| WO2025022367A2 (en) | 2023-07-27 | 2025-01-30 | Life Edit Therapeutics, Inc. | Rna-guided nucleases and active fragments and variants thereof and methods of use |
| WO2025061147A1 (en) * | 2023-09-21 | 2025-03-27 | Shanghaitech University | Rna editing systems of high efficiency and specificity |
| AU2024357861A1 (en) | 2023-10-12 | 2026-04-23 | Renagade Therapeutics Management Inc. | Nickase-retron template-based precision editing system and methods of use |
| WO2025083619A1 (en) | 2023-10-18 | 2025-04-24 | Life Edit Therapeutics, Inc. | Rna-guided nucleases and acive fragments and variants thereof and methods of use |
| CN117092084B (zh) * | 2023-10-20 | 2024-01-12 | 浙江迪福润丝生物科技有限公司 | Wnv蛋白酶抑制剂的筛选方法和抑制效果评价方法 |
| WO2025140399A1 (en) * | 2023-12-26 | 2025-07-03 | Correctsequence Therapeutics (Shanghai) Co., Ltd | Gene editing systems and methods for treating cardiovascular disease |
| WO2025174908A1 (en) | 2024-02-12 | 2025-08-21 | Life Edit Therapeutics, Inc. | Novel rna-guided nucleases and proteins for polymerase editing |
| WO2025185623A1 (en) * | 2024-03-04 | 2025-09-12 | Shanghaitech University | Virus-like particles for gene editing |
| WO2026003754A1 (en) | 2024-06-25 | 2026-01-02 | Life Edit Therapeutics, Inc. | Novel reverse transcriptases and uses thereof |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPS6445314A (en) | 1987-04-16 | 1989-02-17 | Res Corp Technologies Inc | Antitumoral |
| US8288094B2 (en) | 2008-04-09 | 2012-10-16 | Institut Pasteur | APOBEC3 mediated DNA editing |
| AU2010215761B2 (en) * | 2009-02-23 | 2017-04-06 | Cytomx Therapeutics, Inc | Proproteins and methods of use thereof |
| US20150165054A1 (en) * | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| EP3080259B1 (en) * | 2013-12-12 | 2023-02-01 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| CN105886498A (zh) | 2015-05-13 | 2016-08-24 | 沈志荣 | CRISPR-Cas9特异性敲除人PCSK9基因的方法以及用于特异性靶向PCSK9基因的sgRNA |
| IL310721B2 (en) * | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| EP3530737A4 (en) * | 2016-09-13 | 2020-04-29 | Toolgen Incorporated | METHOD FOR IDENTIFYING DNA BASE EDITING USING CYTOSINE DEAMINASE |
| US11192929B2 (en) * | 2016-12-08 | 2021-12-07 | Regents Of The University Of Minnesota | Site-specific DNA base editing using modified APOBEC enzymes |
| CN108342387B (zh) | 2017-01-24 | 2021-09-24 | 谭旭 | Pcsk9抑制剂类降血脂药的递送系统和生物制剂 |
| KR20240116572A (ko) * | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| WO2018208998A1 (en) * | 2017-05-10 | 2018-11-15 | The Regents Of The University Of California | Directed editing of cellular rna via nuclear delivery of crispr/cas9 |
| EP3630849A4 (en) * | 2017-05-25 | 2021-01-13 | The General Hospital Corporation | BIPARTITE BASIC EDITOR (BBE) AND TYPE II-C-CAS9 ZINC FINGER EDITOR ARCHITECTURES |
| CN109136272A (zh) | 2017-06-15 | 2019-01-04 | 中山大学 | 用于特异性修复人hbb基因突变的碱基编辑系统、方法、试剂盒及其在人生殖系中的应用 |
| BR112019028146A2 (pt) * | 2017-07-31 | 2020-07-07 | Sigma-Aldrich Co. Llc | rna guia sintético para sistemas ativadores de crispr/cas |
| CN109021111B (zh) * | 2018-02-23 | 2021-12-07 | 上海科技大学 | 一种基因碱基编辑器 |
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2020
- 2020-02-03 CN CN202211043562.5A patent/CN116656676A/zh active Pending
- 2020-02-03 SG SG11202108303SA patent/SG11202108303SA/en unknown
- 2020-02-03 JP JP2021544682A patent/JP7518549B2/ja active Active
- 2020-02-03 CN CN202211057011.4A patent/CN116355966A/zh active Pending
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Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN118995709A (zh) * | 2024-08-23 | 2024-11-22 | 东北农业大学 | 一种提高基因编辑效率的重组型pegRNA及其应用 |
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| CN113661248A (zh) | 2021-11-16 |
| WO2020156575A1 (en) | 2020-08-06 |
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| AU2020214090A1 (en) | 2021-09-16 |
| US20230094769A1 (en) | 2023-03-30 |
| CN116355966A (zh) | 2023-06-30 |
| JP2026012727A (ja) | 2026-01-27 |
| KR20210126042A (ko) | 2021-10-19 |
| CN116286802A (zh) | 2023-06-23 |
| CN113661248B (zh) | 2022-09-16 |
| JP2022526695A (ja) | 2022-05-26 |
| ZA202105895B (en) | 2024-07-31 |
| BR112021015092A2 (pt) | 2022-10-11 |
| IL285194A (en) | 2021-09-30 |
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| US20240026342A1 (en) | 2024-01-25 |
| CA3128283A1 (en) | 2020-08-06 |
| EP3918083A1 (en) | 2021-12-08 |
| EP3918083A4 (en) | 2023-03-29 |
| US11840685B2 (en) | 2023-12-12 |
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