WO2023036189A1 - Adénine déaminase, éditeur de base adénine la contenant, et ses applications - Google Patents
Adénine déaminase, éditeur de base adénine la contenant, et ses applications Download PDFInfo
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- WO2023036189A1 WO2023036189A1 PCT/CN2022/117592 CN2022117592W WO2023036189A1 WO 2023036189 A1 WO2023036189 A1 WO 2023036189A1 CN 2022117592 W CN2022117592 W CN 2022117592W WO 2023036189 A1 WO2023036189 A1 WO 2023036189A1
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- adenine
- amino acid
- editing
- deaminase
- base
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- 229960000643 adenine Drugs 0.000 title claims abstract description 60
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 title claims abstract description 59
- 229930024421 Adenine Natural products 0.000 title claims abstract description 58
- 108010052875 Adenine deaminase Proteins 0.000 title claims abstract description 38
- 125000003275 alpha amino acid group Chemical group 0.000 claims abstract description 14
- 150000001413 amino acids Chemical class 0.000 claims abstract description 13
- 101710163270 Nuclease Proteins 0.000 claims abstract description 9
- 125000000539 amino acid group Chemical group 0.000 claims description 22
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- 229910052757 nitrogen Inorganic materials 0.000 claims description 2
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- UBDHSURDYAETAL-UHFFFAOYSA-N 8-aminonaphthalene-1,3,6-trisulfonic acid Chemical compound OS(=O)(=O)C1=CC(S(O)(=O)=O)=C2C(N)=CC(S(O)(=O)=O)=CC2=C1 UBDHSURDYAETAL-UHFFFAOYSA-N 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 abstract description 52
- 230000035772 mutation Effects 0.000 abstract description 29
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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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- 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
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
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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
- C12N15/70—Vectors or expression systems specially adapted for E. coli
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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
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- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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Abstract
Adénine déaminase, éditeur de base adénine la contenant, et ses applications, L'adénine désaminase possède une ou plusieurs différences d'acides aminés aux positions 29, 84, 108 et 145, comprenant la séquence d'acides aminés telle que représentée dans la SEQ ID NO : 1. L'éditeur de base adénine comprend une nucléase et une adénine désaminase, et peut efficacement saper la liaison non spécifique de l'adénine désaminase aux bases du substrat, et réduire une fenêtre d'édition à 1 ou 2 bases tout en maintenant une activité d'édition élevée. Les changements de poche structurelle causés par les mutations empêchent également l'adénine désaminase de reconnaître la cytosine comme substrat, et finalement éliminent complètement les événements indépendants d'édition de la cytosine présents dans l'éditeur de base adénine (ABE). De plus, le taux d'indel reste faible, la sécurité est améliorée et les applications dans les traitements médicaux précis, la création de modèles de maladies animales, la sélection génétique des cultures, etc. peuvent être améliorées, ce qui possède une grande valeur d'application.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202111044206.0 | 2021-09-07 | ||
| CN202111044206.0A CN115772512A (zh) | 2021-09-07 | 2021-09-07 | 腺嘌呤脱氨酶、包含其的腺嘌呤碱基编辑器及其应用 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| WO2023036189A1 true WO2023036189A1 (fr) | 2023-03-16 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PCT/CN2022/117592 WO2023036189A1 (fr) | 2021-09-07 | 2022-09-07 | Adénine déaminase, éditeur de base adénine la contenant, et ses applications |
Country Status (2)
| Country | Link |
|---|---|
| CN (1) | CN115772512A (fr) |
| WO (1) | WO2023036189A1 (fr) |
Citations (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110407945A (zh) * | 2019-06-14 | 2019-11-05 | 上海科技大学 | 一种腺嘌呤碱基编辑工具及其用途 |
| CN111328290A (zh) * | 2017-06-26 | 2020-06-23 | 博德研究所 | 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法 |
| CN112143753A (zh) * | 2020-09-17 | 2020-12-29 | 中国农业科学院植物保护研究所 | 一套腺嘌呤碱基编辑器及其相关生物材料与应用 |
| US20210130827A1 (en) * | 2019-10-30 | 2021-05-06 | Pairwise Plants Services, Inc. | Type v crispr-cas base editors and methods of use thereof |
| US20210130805A1 (en) * | 2019-02-13 | 2021-05-06 | Beam Therapeutics Inc. | Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence |
| WO2021158921A2 (fr) * | 2020-02-05 | 2021-08-12 | The Broad Institute, Inc. | Éditeurs de base d'adénine et leurs utilisations |
| CN115247162A (zh) * | 2021-04-27 | 2022-10-28 | 华东师范大学 | 一种腺嘌呤碱基编辑用融合蛋白及其应用 |
-
2021
- 2021-09-07 CN CN202111044206.0A patent/CN115772512A/zh active Pending
-
2022
- 2022-09-07 WO PCT/CN2022/117592 patent/WO2023036189A1/fr unknown
Patent Citations (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111328290A (zh) * | 2017-06-26 | 2020-06-23 | 博德研究所 | 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法 |
| US20210130805A1 (en) * | 2019-02-13 | 2021-05-06 | Beam Therapeutics Inc. | Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence |
| CN110407945A (zh) * | 2019-06-14 | 2019-11-05 | 上海科技大学 | 一种腺嘌呤碱基编辑工具及其用途 |
| US20210130827A1 (en) * | 2019-10-30 | 2021-05-06 | Pairwise Plants Services, Inc. | Type v crispr-cas base editors and methods of use thereof |
| WO2021158921A2 (fr) * | 2020-02-05 | 2021-08-12 | The Broad Institute, Inc. | Éditeurs de base d'adénine et leurs utilisations |
| CN112143753A (zh) * | 2020-09-17 | 2020-12-29 | 中国农业科学院植物保护研究所 | 一套腺嘌呤碱基编辑器及其相关生物材料与应用 |
| CN115247162A (zh) * | 2021-04-27 | 2022-10-28 | 华东师范大学 | 一种腺嘌呤碱基编辑用融合蛋白及其应用 |
Non-Patent Citations (2)
| Title |
|---|
| CHEN LIANG, ZHANG SHUN, XUE NIANNIAN, HONG MENGJIA, ZHANG XIAOHUI, ZHANG DAN, YANG JING, BAI SIJIA, HUANG YIFAN, MENG HAOWEI, WU H: "Engineering precise adenine base editor with infinitesimal rates of bystander mutations and off-target editing", BIORXIV, 13 August 2022 (2022-08-13), XP093045158, Retrieved from the Internet <URL:https://www.biorxiv.org/content/10.1101/2022.08.12.503700v1.full.pdf> [retrieved on 20230509], DOI: 10.1101/2022.08.12.503700 * |
| LI JIANAN, YU WENXIA, HUANG SHISHENG, WU SUSU, LI LIPING, ZHOU JIANKUI, CAO YU, HUANG XINGXU, QIAO YUNBO: "Structure-guided engineering of adenine base editor with minimized RNA off-targeting activity", NATURE COMMUNICATIONS, vol. 12, no. 1, XP093045160, DOI: 10.1038/s41467-021-22519-z * |
Also Published As
| Publication number | Publication date |
|---|---|
| CN115772512A (zh) | 2023-03-10 |
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