JP7642531B2 - プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 - Google Patents
プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 Download PDFInfo
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- JP7642531B2 JP7642531B2 JP2021513763A JP2021513763A JP7642531B2 JP 7642531 B2 JP7642531 B2 JP 7642531B2 JP 2021513763 A JP2021513763 A JP 2021513763A JP 2021513763 A JP2021513763 A JP 2021513763A JP 7642531 B2 JP7642531 B2 JP 7642531B2
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- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
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- 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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- C12N15/09—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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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
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- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024183249A JP2025026840A (ja) | 2018-05-11 | 2024-10-18 | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862670539P | 2018-05-11 | 2018-05-11 | |
| US201862670521P | 2018-05-11 | 2018-05-11 | |
| US62/670,521 | 2018-05-11 | ||
| US62/670,539 | 2018-05-11 | ||
| US201862780890P | 2018-12-17 | 2018-12-17 | |
| US62/780,890 | 2018-12-17 | ||
| PCT/US2019/031897 WO2019217942A1 (en) | 2018-05-11 | 2019-05-11 | Methods of substituting pathogenic amino acids using programmable base editor systems |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024183249A Division JP2025026840A (ja) | 2018-05-11 | 2024-10-18 | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021523737A JP2021523737A (ja) | 2021-09-09 |
| JP2021523737A5 JP2021523737A5 (https=) | 2022-05-19 |
| JP7642531B2 true JP7642531B2 (ja) | 2025-03-10 |
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Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021513763A Active JP7642531B2 (ja) | 2018-05-11 | 2019-05-11 | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
| JP2024183249A Pending JP2025026840A (ja) | 2018-05-11 | 2024-10-18 | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024183249A Pending JP2025026840A (ja) | 2018-05-11 | 2024-10-18 | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US12133884B2 (https=) |
| EP (1) | EP3799603A4 (https=) |
| JP (2) | JP7642531B2 (https=) |
| KR (2) | KR20250134703A (https=) |
| CN (2) | CN121555430A (https=) |
| AU (2) | AU2019265019B2 (https=) |
| CA (1) | CA3100019A1 (https=) |
| WO (1) | WO2019217942A1 (https=) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
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| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors 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 |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| 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 |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| EP3847254A4 (en) | 2018-09-07 | 2022-08-10 | Beam Therapeutics Inc. | COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM |
| WO2020051562A2 (en) | 2018-09-07 | 2020-03-12 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| 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 |
| CN120174005A (zh) | 2019-02-13 | 2025-06-20 | 比姆医疗股份有限公司 | 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞 |
| CA3128755C (en) | 2019-02-13 | 2024-06-04 | Beam Therapeutics Inc. | Compositions and methods for treating hemoglobinopathies |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| EP4034138A4 (en) | 2019-09-27 | 2024-07-31 | Beam Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| IL292517A (en) * | 2019-12-17 | 2022-06-01 | Sigma Aldrich Co Llc | Genome editing in Bacteroidetes |
| WO2021151085A2 (en) | 2020-01-24 | 2021-07-29 | The General Hospital Corporation | Crispr-cas enzymes with enhanced on-target activity |
| WO2021151073A2 (en) | 2020-01-24 | 2021-07-29 | The General Hospital Corporation | Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants |
| WO2021163587A1 (en) * | 2020-02-13 | 2021-08-19 | Beam Therapeutics Inc. | Compositions and methods for engraftment of base edited cells |
| US12416001B2 (en) * | 2020-02-14 | 2025-09-16 | Ohio State Innovation Foundation | Nucleobase editors and methods of use thereof |
| WO2021183504A1 (en) * | 2020-03-11 | 2021-09-16 | North Carolina State University | Compositions, methods, and systems for genome editing technology |
| EP4143315A1 (en) | 2020-04-28 | 2023-03-08 | The Broad Institute Inc. | <smallcaps/>? ? ?ush2a? ? ? ? ?targeted base editing of thegene |
| IL297761A (en) | 2020-05-08 | 2022-12-01 | Broad Inst Inc | Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence |
| US20230279438A1 (en) * | 2020-05-13 | 2023-09-07 | Inserm (Institut National De La Santé Et La Recherche Médicale) | Base editing approaches for the treatment of betahemoglobinopathies |
| CA3196831A1 (en) | 2020-09-25 | 2022-03-31 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| WO2022119294A1 (ko) * | 2020-12-01 | 2022-06-09 | 한양대학교 산학협력단 | 사이토신 교정 활성이 제거된 아데닌 염기교정 유전자가위 및 이의 용도 |
| JP2024502348A (ja) * | 2021-01-05 | 2024-01-18 | ホライズン・ディスカバリー・リミテッド | Tracr不含 V型Casシステム用のガイドRNAデザインおよび複合体 |
| WO2022150372A1 (en) * | 2021-01-05 | 2022-07-14 | Horizon Discovery Ltd. | Guide rna designs and complexes for type v cas systems |
| WO2022206986A1 (en) * | 2021-04-02 | 2022-10-06 | Shanghaitech University | Gene therapy for treating beta-hemoglobinopathies |
| AU2022272250A1 (en) * | 2021-05-14 | 2023-11-30 | Beam Therapeutics Inc. | Compositions and methods for treating transthyretin amyloidosis |
| JP7125727B1 (ja) * | 2021-09-07 | 2022-08-25 | 国立大学法人千葉大学 | 核酸配列改変用組成物および核酸配列の標的部位を改変する方法 |
| WO2023056139A1 (en) * | 2021-10-01 | 2023-04-06 | The Regents Of The University Of California | Compositions and methods for treating a β-thalassemia disease |
| WO2024018056A1 (en) * | 2022-07-22 | 2024-01-25 | Institut National de la Santé et de la Recherche Médicale | Base editing approaches for correcting the ivs2-1 (g>a) mutation in patients suffering from βeta-thalassemia |
| AU2024301706A1 (en) * | 2023-07-21 | 2026-02-05 | Crispr Therapeutics Ag | Modulating expression of alas1 (5'-aminolevulinate synthase 1) gene |
| CN120813698A (zh) * | 2023-09-22 | 2025-10-17 | 北京大学现代农业研究院 | 改进的基因组编辑方法 |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2016528890A (ja) | 2013-07-09 | 2016-09-23 | 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 | CRISPR/Cas系を用いるゲノム編集の治療用の使用 |
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