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1. WO2022119294 - ADENINE BASE EDITOR LACKING CYTOSINE EDITING ACTIVITY AND USE THEREOF

Publication Number WO/2022/119294
Publication Date 09.06.2022
International Application No. PCT/KR2021/017952
International Filing Date 01.12.2021
IPC
C12N 9/78 2006.1
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NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
78acting on carbon to nitrogen bonds other than peptide bonds (3.5)
C12N 15/90 2006.1
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
C12N 15/10 2006.1
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C12N 15/113 2010.1
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
CPC
C12N 15/10
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
10Processes for the isolation, preparation or purification of DNA or RNA
C12N 15/113
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-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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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
C12N 9/78
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes; Proenzymes; Compositions thereof
14Hydrolases (3)
78acting on carbon to nitrogen bonds other than peptide bonds (3.5)
Applicants
  • 한양대학교 산학협력단 IUCF-HYU (INDUSTRY-UNIVERSITY COOPERATION FOUNDATION HANYANG UNIVERSITY) [KR]/[KR]
  • 고려대학교 산학협력단 KOREA UNIVERSITY RESEARCH AND BUSINESS FOUNDATION [KR]/[KR]
Inventors
  • 배상수 BAE, Sangsu
  • 정유경 JEONG, You Kyeong
  • 이석훈 LEE, Seokhoon
  • 우재성 WOO, Jae-Sung
Agents
  • 특허법인(유한)아이시스 ISIS IP LAW LLC
Priority Data
10-2020-016602801.12.2020KR
10-2021-006109912.05.2021KR
Publication Language Korean (ko)
Filing Language Korean (KO)
Designated States
Title
(EN) ADENINE BASE EDITOR LACKING CYTOSINE EDITING ACTIVITY AND USE THEREOF
(FR) ÉDITEUR DE BASE D'ADÉNINE DÉPOURVU D'ACTIVITÉ D'ÉDITION DE CYTOSINE ET SON UTILISATION
(KO) 사이토신 교정 활성이 제거된 아데닌 염기교정 유전자가위 및 이의 용도
Abstract
(EN) 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.
(FR) La présente invention concerne un éditeur de base d'adénine dépourvu d'activité d'édition de cytosine, une composition d'édition de base d'adénine comprenant l'éditeur de base d'adénine et de l'ARNsg, un procédé d'édition de base d'adénine et un kit d'édition de base d'adénine. Ont été identifiées dans la présente invention quatre mutations (V106W, D108Q, F148A et F149A) d'adénosine désaminase qui augmente la spécificité de l'édition d'adénine par réduction de l'efficacité d'édition de base de cytosine, et les résultats d'un test à l'aide de variants ABE construits par l'introduction de chacune des mutations dans divers ABE dans une autre version améliorée par rapport à l'ABEmax ont confirmé que les mutations réduisent remarquablement les effets d'édition de la cytosine. Par conséquent, au fur et à mesure que l'éditeur de base d'adénine selon la présente invention peut éditer plus spécifiquement de l'adénine, une composition d'édition de base d'adénine comprenant l'éditeur et l'ARNsg sera avantageusement utilisée dans le domaine de la thérapie génique qui devrait modifier avec précision uniquement l'adénine dans tous les organismes vivants y compris les êtres humains, les plantes et les bactéries, ou dans le domaine de la mise au point de nouvelles cultures.
(KO) 본 발명은 사이토신 교정 활성이 제거된 아데닌 염기교정 유전자가위, 상기 아데닌 염기교정 유전자가위 및 sgRNA를 포함하는 아데닌 염기교정용 조성물, 아데닌 염기교정 방법 및 아데닌 염기교정용 키트에 관한 것이다. 본 발명에서는 사이토신 염기교정 효율을 감소시켜 아데닌 교정에 대한 특이성을 증가시키는 아데노신 탈아미노화 효소 내 4가지 돌연변이 (V106W, D108Q, F148A, F149A)를 확인하였고, ABEmax 보다 더욱 개량된 버전의 다양한 ABEs에 상기 각 돌연변이가 도입된 ABE 변이체를 제작하여 테스트한 결과 상기 돌연변이들이 사이토신 교정 효과를 현저히 감소시키는 것을 확인하였다. 이에, 본 발명에 따른 아데닌 염기교정 유전자가위는 더욱 정교한 아데닌의 교정이 가능한바, 상기 유전자가위와 sgRNA를 포함하는 아데닌 염기교정용 조성물은 인간, 식물, 박테리아를 포함한 모든 생명체에서 아데닌만을 정확하게 교정해야 하는 유전자 치료 분야 또는 새로운 작물 개발 분야 등에서 유용하게 활용 가능할 것이다.
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