Title:
BASE EDITING SYSTEM FOR ACHIEVING A-TO-C AND/OR A-TO-T BASE MUTATION AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2023/024089
Kind Code:
A1
Abstract:
Disclosed is a base editing system for achieving A-to-C and/or A-to-T base mutation and the use thereof. A base editor is constructed by means of fusing 3-methyladenine glycosidase with adenosine deaminase and Cas9 nuclease with impaired catalytic activity, which achieves adenine-based transversion for the first time. It is found through experimental comparison that AXBE, which is constructed by means of fusing mouse-derived 3-methyladenine glycosidase with adenosine deaminase TadA-8e derived from E coli and Cas9n with impaired catalytic activity derived from Streptococcus pyogenes, has the best effect of catalyzing the transversion of adenine. The use of the base editing system in the gene therapy, cell therapy, human disease model production, and crop genetic breeding, etc. is promoted.
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Inventors:
CHEN LIANG (CN)
LI DALI (CN)
HONG MENGJIA (CN)
LUAN CHANGMING (CN)
LI DALI (CN)
HONG MENGJIA (CN)
LUAN CHANGMING (CN)
Application Number:
PCT/CN2021/115084
Publication Date:
March 02, 2023
Filing Date:
August 27, 2021
Export Citation:
Assignee:
UNIV EAST CHINA NORMAL (CN)
International Classes:
C12N15/10; A61K48/00; C12N9/22; C12N9/78
Domestic Patent References:
| WO2022006226A1 | 2022-01-06 | |||
| WO2019147073A1 | 2019-08-01 | |||
| WO2021158921A2 | 2021-08-12 |
Foreign References:
| CN111148833A | 2020-05-12 | |||
| CN110835634A | 2020-02-25 | |||
| CN112979821A | 2021-06-18 | |||
| CN110214183A | 2019-09-06 |
Other References:
GAUDELLI NICOLE M.; KOMOR ALEXIS C.; REES HOLLY A.; PACKER MICHAEL S.; BADRAN AHMED H.; BRYSON DAVID I.; LIU DAVID R.: "Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage", NATURE, NATURE PUBLISHING GROUP UK, LONDON, vol. 551, no. 7681, 23 November 2017 (2017-11-23), London, pages 464 - 471, XP037336615, ISSN: 0028-0836, DOI: 10.1038/nature24644
KIM JIN-SOO: "Precision genome engineering through adenine and cytosine base editing", NATURE PLANTS, NATURE PUBLISHING GROUP UK, LONDON, vol. 4, no. 3, 26 February 2018 (2018-02-26), London , pages 148 - 151, XP036442899, DOI: 10.1038/s41477-018-0115-z
KIM JIN-SOO: "Precision genome engineering through adenine and cytosine base editing", NATURE PLANTS, NATURE PUBLISHING GROUP UK, LONDON, vol. 4, no. 3, 26 February 2018 (2018-02-26), London , pages 148 - 151, XP036442899, DOI: 10.1038/s41477-018-0115-z
Attorney, Agent or Firm:
BEIJING JUNHUI INTELLECTUAL PROPERTY AGENT OFFICE (ORDINARY PARTNERSHIP) (CN)
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