Title:
ADENINE BASE EDITOR FUSION PROTEIN FREE OF LIMIT BY PAM, AND APPLICATION
Document Type and Number:
WIPO Patent Application WO/2023/015759
Kind Code:
A1
Abstract:
An adenine base editor fusion protein free of limit by PAM, and an application. Provided is a mutant polypeptide. From N-terminus to C-terminus, the polypeptide sequentially comprises a SpRY(D10A) N-terminal fragment, a TadA8e fragment, and a SpRY(D10A) C-terminal fragment. A fusion protein containing the mutant polypeptide can target a whole genome and broaden the editable range of the genome; the present invention can more efficiently cause base conversion from A:T to G:C, and has great application potential, comprising, but not limited to, simulation or repair of pathogenic sites of genetic disorders; moreover, the present invention reduces off-target in the transcriptome level, and achieves a mutant form having both high efficiency and low off-target.
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Inventors:
MA XU (CN)
CAO XIAOFANG (CN)
JIN XIAOHUA (CN)
CAO XIAOFANG (CN)
JIN XIAOHUA (CN)
Application Number:
PCT/CN2021/131279
Publication Date:
February 16, 2023
Filing Date:
November 17, 2021
Export Citation:
Assignee:
NATIONAL RES INSTITUTE FOR FAMILY PLANNING (CN)
International Classes:
C12N9/22; A61K38/50; A61K48/00; A61P3/00; C07K19/00; C12N9/78; C12N15/62
Domestic Patent References:
| WO2021151065A2 | 2021-07-29 | |||
| WO2021151085A2 | 2021-07-29 |
Foreign References:
| CN113699135A | 2021-11-26 | |||
| CN112143753A | 2020-12-29 | |||
| CN112048493A | 2020-12-08 |
Other References:
ZHENG JITAN, YAN NA′NA; ZUO ERWEI: "Research advancement in CRISPR-based genome editing technologies", JOURNAL OF FUJIAN MEDICAL UNIVERSITY, FUJIAN MEDICAL UNIVERSITY, CN, vol. 55, no. 3, 28 June 2021 (2021-06-28), CN , pages 187 - 191, XP093035328, ISSN: 1672-4194
YAN DAQI, REN BIN, LIU LANG, YAN FANG, LI SHAOFANG, WANG GUIRONG, SUN WENXIAN, ZHOU XUEPING, ZHOU HUANBIN: "High-efficiency and multiplex adenine base editing in plants using new TadA variants", MOLECULAR PLANT, vol. 14, no. 5, 1 May 2021 (2021-05-01), pages 722 - 731, XP093006106, ISSN: 1674-2052, DOI: 10.1016/j.molp.2021.02.007
YAN DAQI, REN BIN, LIU LANG, YAN FANG, LI SHAOFANG, WANG GUIRONG, SUN WENXIAN, ZHOU XUEPING, ZHOU HUANBIN: "High-efficiency and multiplex adenine base editing in plants using new TadA variants", MOLECULAR PLANT, vol. 14, no. 5, 1 May 2021 (2021-05-01), pages 722 - 731, XP093006106, ISSN: 1674-2052, DOI: 10.1016/j.molp.2021.02.007
Attorney, Agent or Firm:
BEIJING WANHUIDA LAW FIRM (CN)
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