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Title:
ADENINE BASE EDITOR LACKING CYTOSINE EDITING ACTIVITY AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2022/119294
Kind Code:
A1
Abstract:
The present invention relates to an adenine base editor lacking cytosine editing activity, an adenine base editing composition comprising the adenine base editor and sgRNA, an adenine base editing method, and an adenine base editing kit. Identified in the present invention were four mutations (V106W, D108Q, F148A, and F149A) of adenosine deaminase that increase specificity of adenine editing by reducing cytosine base editing efficiency, and the results from a test using ABE variants constructed by introducing each of the mutations into various ABEs in a further improved version compared to ABEmax confirmed that the mutations remarkably reduce cytosine editing effects. Hence, as the adenine base editor according to the present invention can more elaborately edit adenine, an adenine base editing composition comprising the editor and sgRNA will be advantageously used in the gene therapy field which should accurately edit only adenine in all living organisms including humans, plants, and bacteria, or in the field of developing new crops.

Inventors:
BAE SANGSU (KR)
JEONG YOU KYEONG (KR)
LEE SEOKHOON (KR)
WOO JAE-SUNG (KR)
Application Number:
PCT/KR2021/017952
Publication Date:
June 09, 2022
Filing Date:
December 01, 2021
Export Citation:
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Assignee:
IUCF HYU (KR)
UNIV KOREA RES & BUS FOUND (KR)
International Classes:
C12N9/78; C12N15/10; C12N15/113; C12N15/90
Domestic Patent References:
WO2020168132A12020-08-20
WO2019217942A12019-11-14
Foreign References:
US20200308571A12020-10-01
US20190093099A12019-03-28
KR20190044157A2019-04-30
Other References:
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
ANONYMOUS: "Development of ultra-precise gene base editing technology", 7 July 2021 (2021-07-07), pages 1 - 3, XP055936206, Retrieved from the Internet
Attorney, Agent or Firm:
ISIS IP LAW LLC (KR)
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