JP7708660B2 - Ｒｎａ誘導型ヌクレアーゼ、その活性な断片とバリアント、ならびに利用法 - Google Patents
Ｒｎａ誘導型ヌクレアーゼ、その活性な断片とバリアント、ならびに利用法Info
- Publication number
- JP7708660B2 JP7708660B2 JP2021518031A JP2021518031A JP7708660B2 JP 7708660 B2 JP7708660 B2 JP 7708660B2 JP 2021518031 A JP2021518031 A JP 2021518031A JP 2021518031 A JP2021518031 A JP 2021518031A JP 7708660 B2 JP7708660 B2 JP 7708660B2
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Classifications
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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
- C12N15/87—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
- 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
- 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
- C12N15/11—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
-
- 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
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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
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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
- C12N15/87—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
-
- 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]
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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
- C12N2310/10—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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
-
- 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
- C12Q2521/00—Reaction characterised by the enzymatic activity
- C12Q2521/30—Phosphoric diester hydrolysing, i.e. nuclease
- C12Q2521/301—Endonuclease
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Mycology (AREA)
- Medicinal Chemistry (AREA)
- Cell Biology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025113206A JP2025133875A (ja) | 2018-06-05 | 2025-07-03 | Ｒｎａ誘導型ヌクレアーゼ、その活性な断片とバリアント、ならびに利用法 |
Applications Claiming Priority (19)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862680853P | 2018-06-05 | 2018-06-05 | |
| US201862680859P | 2018-06-05 | 2018-06-05 | |
| US201862680862P | 2018-06-05 | 2018-06-05 | |
| US201862680845P | 2018-06-05 | 2018-06-05 | |
| US201862680846P | 2018-06-05 | 2018-06-05 | |
| US201862680863P | 2018-06-05 | 2018-06-05 | |
| US62/680,862 | 2018-06-05 | ||
| US62/680,853 | 2018-06-05 | ||
| US62/680,859 | 2018-06-05 | ||
| US62/680,846 | 2018-06-05 | ||
| US62/680,863 | 2018-06-05 | ||
| US62/680,845 | 2018-06-05 | ||
| US201862686901P | 2018-06-19 | 2018-06-19 | |
| US62/686,901 | 2018-06-19 | ||
| US201962805041P | 2019-02-13 | 2019-02-13 | |
| US201962805045P | 2019-02-13 | 2019-02-13 | |
| US62/805,045 | 2019-02-13 | ||
| US62/805,041 | 2019-02-13 | ||
| PCT/US2019/035373 WO2019236566A1 (en) | 2018-06-05 | 2019-06-04 | Rna-guided nucleases and active fragments and variants thereof and methods of use |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025113206A Division JP2025133875A (ja) | 2018-06-05 | 2025-07-03 | Ｒｎａ誘導型ヌクレアーゼ、その活性な断片とバリアント、ならびに利用法 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021526853A JP2021526853A (ja) | 2021-10-11 |
| JP2021526853A5 JP2021526853A5 (https=) | 2022-06-10 |
| JPWO2019236566A5 JPWO2019236566A5 (https=) | 2022-06-10 |
| JP7708660B2 true JP7708660B2 (ja) | 2025-07-15 |
Family
ID=67002401
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021518031A Active JP7708660B2 (ja) | 2018-06-05 | 2019-06-04 | Ｒｎａ誘導型ヌクレアーゼ、その活性な断片とバリアント、ならびに利用法 |
| JP2025113206A Pending JP2025133875A (ja) | 2018-06-05 | 2025-07-03 | Ｒｎａ誘導型ヌクレアーゼ、その活性な断片とバリアント、ならびに利用法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025113206A Pending JP2025133875A (ja) | 2018-06-05 | 2025-07-03 | Ｒｎａ誘導型ヌクレアーゼ、その活性な断片とバリアント、ならびに利用法 |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US11162114B2 (https=) |
| EP (2) | EP3802807B1 (https=) |
| JP (2) | JP7708660B2 (https=) |
| KR (1) | KR20210089629A (https=) |
| CN (1) | CN113015797A (https=) |
| AU (2) | AU2019282149B2 (https=) |
| BR (1) | BR112020024863A2 (https=) |
| CA (1) | CA3102840A1 (https=) |
| DK (1) | DK3802807T3 (https=) |
| ES (1) | ES3011849T3 (https=) |
| FI (1) | FI3802807T3 (https=) |
| IL (2) | IL279222B2 (https=) |
| MX (1) | MX2020013124A (https=) |
| PL (1) | PL3802807T3 (https=) |
| PT (1) | PT3802807T (https=) |
| SG (1) | SG11202011975WA (https=) |
| TW (1) | TW202532650A (https=) |
| WO (1) | WO2019236566A1 (https=) |
| ZA (1) | ZA202008045B (https=) |
Families Citing this family (55)
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| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| JP7109784B2 (ja) | 2015-10-23 | 2022-08-01 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 遺伝子編集のための進化したＣａｓ９蛋白質 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| JP2020510038A (ja) | 2017-03-09 | 2020-04-02 | プレジデント アンド フェローズ オブ ハーバード カレッジ | がんワクチン |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | プレジデント アンド フェローズ オブ ハーバード カレッジ | シトシンからグアニンへの塩基編集因子 |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| 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 |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3797160A1 (en) | 2018-05-23 | 2021-03-31 | The Broad Institute Inc. | Base editors and uses thereof |
| EP3823633A4 (en) | 2018-06-29 | 2023-05-03 | Editas Medicine, Inc. | SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| JP7657726B2 (ja) | 2019-03-19 | 2025-04-07 | ザ ブロード インスティテュート，インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| KR20220062289A (ko) | 2019-08-12 | 2022-05-16 | 라이프에디트 테라퓨틱스, 인크. | Rna-가이드된 뉴클레아제 및 그의 활성 단편 및 변이체 및 사용 방법 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| KR102776469B1 (ko) * | 2019-12-10 | 2025-03-05 | 인스크립타 인코포레이티드 | 신규 mad 뉴클레아제 |
| US20230203463A1 (en) * | 2019-12-30 | 2023-06-29 | LifeEDIT Therapeutics, Inc. | Rna-guided nucleases and active fragments and variants thereof and methods of use |
| TW202208626A (zh) * | 2020-04-24 | 2022-03-01 | 美商生命編輯公司 | Rna引導核酸酶及其活性片段與變體，以及使用方法 |
| WO2021222318A1 (en) * | 2020-04-28 | 2021-11-04 | The Broad Institute, Inc. | Targeted base editing of the ush2a gene |
| DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
| GB202007943D0 (en) | 2020-05-27 | 2020-07-08 | Snipr Biome Aps | Products & methods |
| CA3174356A1 (en) | 2020-07-01 | 2022-01-06 | Santosh NARAYAN | Compositions and methods for cellular reprogramming using circular rna |
| JP2023534693A (ja) | 2020-07-15 | 2023-08-10 | ライフエディット セラピューティクス，インコーポレイティド | ウラシル安定化タンパク質並びにその活性断片及びバリアント並びに使用方法 |
| WO2022027035A1 (en) * | 2020-07-27 | 2022-02-03 | The Children's Hospital Of Philadelphia | In utero and postnatal gene editing and therapy for treatment of monogenic diseases, including mucopolysaccharidosis type 1h and other disorders |
| TWI910226B (zh) | 2020-09-11 | 2026-01-01 | 美商生命編輯治療學公司 | Ｄｎａ修飾酶及活性片段，及其變異體與使用方法 |
| CA3202977A1 (en) | 2020-12-22 | 2022-06-30 | Chroma Medicine, Inc. | Compositions and methods for epigenetic editing |
| CN117295817A (zh) | 2021-03-22 | 2023-12-26 | 生命编辑制药股份有限公司 | Dna修饰酶及其活性片段和变体以及使用方法 |
| AU2022290278A1 (en) | 2021-06-11 | 2024-01-04 | LifeEDIT Therapeutics, Inc. | Rna polymerase iii promoters and methods of use |
| CA3244778A1 (en) * | 2022-02-23 | 2023-08-31 | Metagenomi Therapeutics, Inc. | SYSTEMS AND METHODS FOR TRANSPOSING CARGO NUCLEOTID SEQUENCES |
| CN117925585A (zh) * | 2022-04-07 | 2024-04-26 | 尧唐(上海)生物科技有限公司 | 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途 |
| EP4519293A1 (en) | 2022-05-01 | 2025-03-12 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of pcsk9 expression |
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| TW202424186A (zh) | 2022-08-25 | 2024-06-16 | 美商生命編輯治療學公司 | Rna引導核酸酶中介基因編輯用之具鎖核酸引導 rna之化學修飾 |
| CN115820636B (zh) * | 2022-09-02 | 2024-06-11 | 华南农业大学 | 靶向TP53基因的sgRNA及其应用 |
| KR20250110209A (ko) | 2022-09-23 | 2025-07-18 | 엔크로마 바이오, 인크. | Hbv 유전자 발현의 에피제네틱 조절을 위한 조성물 및 방법 |
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| IL324634A (en) | 2023-05-15 | 2026-01-01 | Nchroma Bio Inc | Compositions and methods for epigenetic regulation of hbv gene expression |
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| TW202546224A (zh) | 2024-02-12 | 2025-12-01 | 美商生命編輯治療學公司 | 新穎的ｒｎａ引導之核酸酶及用於聚合酶編輯之蛋白質 |
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Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2017155717A1 (en) | 2016-03-11 | 2017-09-14 | Pioneer Hi-Bred International, Inc. | Novel cas9 systems and methods of use |
| WO2017155714A1 (en) | 2016-03-11 | 2017-09-14 | Pioneer Hi-Bred International, Inc. | Novel cas9 systems and methods of use |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
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