Title:
ADENINE BASE EDITOR HAVING INCREASED THYMINE-CYTOSINE SEQUENCE-SPECIFIC CYTOSINE EDITING ACTIVITY, AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2022/119295
Kind Code:
A1
Abstract:
The present invention relates to: an adenine base editor having increased thymine-cytosine sequence-specific cytosine base editing activity; a composition for thymine-cytosine sequence-specific cytosine base editing, comprising the base editor and a guide RNA; a cytosine editing method; and a cytosine base editing kit. An adenine base editor having increased T-C sequence-specific cytosine editing activity, according to the present invention, is prepared by introducing a P48R mutation into an adenosine deaminase, has an operation range that is more sophisticated than a conventional cytosine base editor, allows cytosine editing only if thymine is positioned right in front of cytosine, thereby enabling elaborate editing even if there is a plurality of cytosines within the range, enables cytosine to be substituted with thymine by adding UGI, and enables cytosine to be substituted with guanine if UGI is not added. Therefore, the cytosine base editing composition comprising a cytosine base editor and a sgRNA, according to the present invention, can be effectively used in the fields of gene therapy or new crop development which requires precise editing of only cytosine in all living organisms including humans, plants and bacteria.
Inventors:
BAE SANGSU (KR)
JEONG YOU KYEONG (KR)
LEE SEOKHOON (KR)
WOO JAE-SUNG (KR)
JEONG YOU KYEONG (KR)
LEE SEOKHOON (KR)
WOO JAE-SUNG (KR)
Application Number:
PCT/KR2021/017954
Publication Date:
June 09, 2022
Filing Date:
December 01, 2021
Export Citation:
Assignee:
IUCF HYU (KR)
UNIV KOREA RES & BUS FOUND (KR)
UNIV KOREA RES & BUS FOUND (KR)
International Classes:
C12N9/78; C12N15/10; C12N15/113; C12N15/90
Domestic Patent References:
| WO2018027078A1 | 2018-02-08 | |||
| WO2020168132A1 | 2020-08-20 |
Foreign References:
| KR20190044157A | 2019-04-30 | |||
| US20200308571A1 | 2020-10-01 |
Other References:
KIM HEON SEOK; JEONG YOU KYEONG; HUR JUNHO K; KIM JIN-SOO; BAE SANGSU: "Adenine base editors catalyze cytosine conversions in human cells", NATURE BIOTECHNOLOGY, vol. 37, no. 10, 23 September 2019 (2019-09-23), New York, pages 1145 - 1148, XP036897240, ISSN: 1087-0156, DOI: 10.1038/s41587-019-0254-4
JEONG YOU KYEONG, LEE SEOKHOON, HWANG GUE-HO, HONG SUNG-AH, PARK SE-EUN, KIM JIN-SOO, WOO JAE-SUNG, BAE SANGSU: "Precise adenine base editors that exhibit minimized cytosine catalysis", RESEARCH SQUARE, 1 September 2020 (2020-09-01), pages 1 - 15, XP055936198, DOI: 10.21203/rs.3.rs-84912/v1
JEONG YOU KYEONG; LEE SEOKHOON; HWANG GUE-HO; HONG SUNG-AH; PARK SE-EUN; KIM JIN-SOO; WOO JAE-SUNG; BAE SANGSU: "Adenine base editor engineering reduces editing of bystander cytosines", NATURE BIOTECHNOLOGY, vol. 39, no. 11, 1 July 2021 (2021-07-01), New York, pages 1426 - 1433, XP037616226, ISSN: 1087-0156, DOI: 10.1038/s41587-021-00943-2
BAE SANG-SOO; WOO JAE-SUNG; JUNG YOO-KYUNG; SEOK-HOON LEE: "Development of Ultra-Precision Gene Base Correction Technology", 7 July 2021 (2021-07-07), Korea, pages 1 - 2, XP009538121, Retrieved from the Internet [retrieved on 20220809]
JEONG YOU KYEONG, LEE SEOKHOON, HWANG GUE-HO, HONG SUNG-AH, PARK SE-EUN, KIM JIN-SOO, WOO JAE-SUNG, BAE SANGSU: "Precise adenine base editors that exhibit minimized cytosine catalysis", RESEARCH SQUARE, 1 September 2020 (2020-09-01), pages 1 - 15, XP055936198, DOI: 10.21203/rs.3.rs-84912/v1
JEONG YOU KYEONG; LEE SEOKHOON; HWANG GUE-HO; HONG SUNG-AH; PARK SE-EUN; KIM JIN-SOO; WOO JAE-SUNG; BAE SANGSU: "Adenine base editor engineering reduces editing of bystander cytosines", NATURE BIOTECHNOLOGY, vol. 39, no. 11, 1 July 2021 (2021-07-01), New York, pages 1426 - 1433, XP037616226, ISSN: 1087-0156, DOI: 10.1038/s41587-021-00943-2
BAE SANG-SOO; WOO JAE-SUNG; JUNG YOO-KYUNG; SEOK-HOON LEE: "Development of Ultra-Precision Gene Base Correction Technology", 7 July 2021 (2021-07-07), Korea, pages 1 - 2, XP009538121, Retrieved from the Internet
Attorney, Agent or Firm:
ISIS IP LAW LLC (KR)
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