CN114206395B - 使用可编程碱基编辑器系统编辑单核苷酸多态性的方法 - Google Patents
使用可编程碱基编辑器系统编辑单核苷酸多态性的方法 Download PDFInfo
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- CN114206395B CN114206395B CN202080052684.3A CN202080052684A CN114206395B CN 114206395 B CN114206395 B CN 114206395B CN 202080052684 A CN202080052684 A CN 202080052684A CN 114206395 B CN114206395 B CN 114206395B
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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
- 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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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- 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
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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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- 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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- 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
- 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)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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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)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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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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- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
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- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
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- Medicinal Chemistry (AREA)
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- Biophysics (AREA)
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- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202510277023.5A CN120346348A (zh) | 2019-05-21 | 2020-05-20 | 使用可编程碱基编辑器系统编辑单核苷酸多态性的方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962850919P | 2019-05-21 | 2019-05-21 | |
| US62/850,919 | 2019-05-21 | ||
| PCT/US2020/033807 WO2020236936A1 (en) | 2019-05-21 | 2020-05-20 | Methods of editing a single nucleotide polymorphism using programmable base editor systems |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202510277023.5A Division CN120346348A (zh) | 2019-05-21 | 2020-05-20 | 使用可编程碱基编辑器系统编辑单核苷酸多态性的方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN114206395A CN114206395A (zh) | 2022-03-18 |
| CN114206395B true CN114206395B (zh) | 2025-03-28 |
Family
ID=73459015
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202080052684.3A Active CN114206395B (zh) | 2019-05-21 | 2020-05-20 | 使用可编程碱基编辑器系统编辑单核苷酸多态性的方法 |
| CN202510277023.5A Pending CN120346348A (zh) | 2019-05-21 | 2020-05-20 | 使用可编程碱基编辑器系统编辑单核苷酸多态性的方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202510277023.5A Pending CN120346348A (zh) | 2019-05-21 | 2020-05-20 | 使用可编程碱基编辑器系统编辑单核苷酸多态性的方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20220387622A1 (enExample) |
| EP (1) | EP3972654A4 (enExample) |
| JP (1) | JP7679311B2 (enExample) |
| KR (1) | KR20220010540A (enExample) |
| CN (2) | CN114206395B (enExample) |
| AU (1) | AU2020279751A1 (enExample) |
| CA (1) | CA3140093A1 (enExample) |
| WO (1) | WO2020236936A1 (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL3765615T3 (pl) | 2018-03-14 | 2023-11-13 | Arbor Biotechnologies, Inc. | Nowe enzymy i układy crispr ukierunkowane na dna |
| WO2019217942A1 (en) | 2018-05-11 | 2019-11-14 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| WO2019222555A1 (en) | 2018-05-16 | 2019-11-21 | Arbor Biotechnologies, Inc. | Novel crispr-associated systems and components |
| EP3850088A4 (en) | 2018-09-07 | 2023-07-19 | Beam Therapeutics, Inc. | Compositions and methods for improving base editing |
| KR20210055733A (ko) | 2018-09-07 | 2021-05-17 | 빔 테라퓨틱스, 인크. | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
| EP3924479A4 (en) * | 2019-02-13 | 2023-01-25 | Beam Therapeutics, Inc. | ADENOSINE DEAMINASE BASE EDITORS AND METHODS OF USING THEM TO MODIFY A NUCLEOBASE IN A TARGET SEQUENCE |
| WO2020168135A1 (en) | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| US12600971B2 (en) | 2019-02-13 | 2026-04-14 | Beam Therapeutics Inc. | Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence |
| US12594301B2 (en) | 2019-09-27 | 2026-04-07 | Beam Therapeutics Inc. | Compositions and methods for treatment of liquid cancers |
| AU2021347359A1 (en) | 2020-09-25 | 2023-05-18 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| EP4396207A4 (en) * | 2021-09-03 | 2025-07-16 | Univ Chicago | POLYPEPTIDES AND METHODS FOR MODIFYING NUCLEIC ACIDS |
| EP4404942A4 (en) * | 2021-09-26 | 2026-06-17 | Wave Life Sciences Ltd | Compositions for the Editing of Transcripts MECP2 and Related Processes |
| WO2023140694A1 (ko) * | 2022-01-24 | 2023-07-27 | 주식회사 툴젠 | 스트렙토코커스 피오게네스 유래 cas9 변이체 |
| CA3266991A1 (en) * | 2022-09-08 | 2024-03-14 | The University Court Of The University Of Edinburgh | TREATMENT OF RETT SYNDROME |
| WO2024102811A2 (en) * | 2022-11-08 | 2024-05-16 | The Board Of Trustees Of The Leland Stanford Junior University | Muscle-specific base editors for correction of mutations causing- dilated cardiomyopathy |
| CN117965505A (zh) * | 2023-06-28 | 2024-05-03 | 微光基因(苏州)有限公司 | 工程化的腺苷脱氨酶及碱基编辑器 |
| JP2026524490A (ja) * | 2023-07-07 | 2026-07-22 | ミリアド ジェネティクス,インコーポレーテッド | 乳がんについての多祖先ポリジェニックリスク評価 |
| WO2025199339A2 (en) * | 2024-03-20 | 2025-09-25 | Shape Therapeutics Inc. | Mecp2-targeting engineered guide rnas and polynucleotides |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105793425A (zh) * | 2013-06-17 | 2016-07-20 | 布罗德研究所有限公司 | 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用 |
| CN108291218A (zh) * | 2015-07-15 | 2018-07-17 | 新泽西鲁特格斯州立大学 | 核酸酶非依赖性靶向基因编辑平台及其用途 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201510327TA (en) * | 2013-06-17 | 2016-01-28 | Broad Inst Inc | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
| WO2018027078A1 (en) * | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| RU2019133002A (ru) * | 2017-03-24 | 2021-04-26 | Зе Юнивёрсити Корт Оф Зе Юнивёрсити Оф Эдинбург | ЭКСПРЕССИОННЫЕ КАССЕТЫ MeCP2 |
| IL316453A (en) * | 2017-10-06 | 2024-12-01 | Univ Oregon Health & Science | RNA editing agents and methods |
| CA3082251A1 (en) * | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| EP3699267A4 (en) | 2017-10-17 | 2021-10-27 | Kyoto University | METHOD OF MANUFACTURING AN ARTIFICIAL NEUROMUSCULAR END PLATE FROM PLURIPOTENT STEM CELLS |
| KR102853088B1 (ko) * | 2018-05-11 | 2025-09-02 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법 |
-
2020
- 2020-05-20 JP JP2021568855A patent/JP7679311B2/ja active Active
- 2020-05-20 EP EP20809005.0A patent/EP3972654A4/en active Pending
- 2020-05-20 KR KR1020217041287A patent/KR20220010540A/ko active Pending
- 2020-05-20 CN CN202080052684.3A patent/CN114206395B/zh active Active
- 2020-05-20 CN CN202510277023.5A patent/CN120346348A/zh active Pending
- 2020-05-20 CA CA3140093A patent/CA3140093A1/en active Pending
- 2020-05-20 WO PCT/US2020/033807 patent/WO2020236936A1/en not_active Ceased
- 2020-05-20 US US17/612,879 patent/US20220387622A1/en active Pending
- 2020-05-20 AU AU2020279751A patent/AU2020279751A1/en active Pending
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105793425A (zh) * | 2013-06-17 | 2016-07-20 | 布罗德研究所有限公司 | 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用 |
| CN108291218A (zh) * | 2015-07-15 | 2018-07-17 | 新泽西鲁特格斯州立大学 | 核酸酶非依赖性靶向基因编辑平台及其用途 |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2022533673A (ja) | 2022-07-25 |
| KR20220010540A (ko) | 2022-01-25 |
| CN114206395A (zh) | 2022-03-18 |
| CN120346348A (zh) | 2025-07-22 |
| WO2020236936A1 (en) | 2020-11-26 |
| AU2020279751A1 (en) | 2021-12-02 |
| EP3972654A4 (en) | 2024-07-03 |
| CA3140093A1 (en) | 2020-11-26 |
| JP7679311B2 (ja) | 2025-05-19 |
| US20220387622A1 (en) | 2022-12-08 |
| EP3972654A1 (en) | 2022-03-30 |
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