KR20210124280A - 표적-이탈 탈아미노화가 감소된 핵염기 편집기 및 이를 이용하여 핵염기 표적 서열을 변형시키는 방법 - Google Patents

표적-이탈 탈아미노화가 감소된 핵염기 편집기 및 이를 이용하여 핵염기 표적 서열을 변형시키는 방법 Download PDF

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KR20210124280A
KR20210124280A KR1020217026727A KR20217026727A KR20210124280A KR 20210124280 A KR20210124280 A KR 20210124280A KR 1020217026727 A KR1020217026727 A KR 1020217026727A KR 20217026727 A KR20217026727 A KR 20217026727A KR 20210124280 A KR20210124280 A KR 20210124280A
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South Korea
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cytidine
deaminase
base editor
fusion protein
amino acid
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Korean (ko)
Inventor
니콜 고델리
이 유
이안 슬레이메이커
제이슨 마이클 제크
승주 이
데이비드 에이. 본
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빔 테라퓨틱스, 인크.
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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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    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2497Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing N- glycosyl compounds (3.2.2)
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    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
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    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005Cytidine deaminase (3.5.4.5)
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
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    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004Adenosine deaminase (3.5.4.4)

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  • Mycology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
KR1020217026727A 2019-01-31 2020-01-31 표적-이탈 탈아미노화가 감소된 핵염기 편집기 및 이를 이용하여 핵염기 표적 서열을 변형시키는 방법 Pending KR20210124280A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962799702P 2019-01-31 2019-01-31
US62/799,702 2019-01-31
US201962835456P 2019-04-17 2019-04-17
US62/835,456 2019-04-17
US201962941569P 2019-11-27 2019-11-27
US62/941,569 2019-11-27
PCT/US2020/016288 WO2020160517A1 (en) 2019-01-31 2020-01-31 Nucleobase editors having reduced off-target deamination and methods of using same to modify a nucleobase target sequence

Publications (1)

Publication Number Publication Date
KR20210124280A true KR20210124280A (ko) 2021-10-14

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KR1020217026727A Pending KR20210124280A (ko) 2019-01-31 2020-01-31 표적-이탈 탈아미노화가 감소된 핵염기 편집기 및 이를 이용하여 핵염기 표적 서열을 변형시키는 방법

Country Status (8)

Country Link
US (1) US20220136012A1 (https=)
EP (1) EP3918077A4 (https=)
JP (2) JP7600122B2 (https=)
KR (1) KR20210124280A (https=)
CN (2) CN114072509B (https=)
AU (1) AU2020216484A1 (https=)
CA (1) CA3127494A1 (https=)
WO (1) WO2020160517A1 (https=)

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EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2020215730A1 (en) 2019-01-31 2021-07-29 Beam Therapeutics Inc. Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors
CN120174005A (zh) 2019-02-13 2025-06-20 比姆医疗股份有限公司 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
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IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
EP4313118A4 (en) * 2021-03-26 2025-06-18 Beam Therapeutics Inc. ADENOSINE DEAMINASE VARIANTS AND THEIR USES
KR20240007651A (ko) * 2021-04-16 2024-01-16 빔 테라퓨틱스, 인크. 간세포의 유전적 변형
WO2022261509A1 (en) * 2021-06-11 2022-12-15 The Broad Institute, Inc. Improved cytosine to guanine base editors
US20240352439A1 (en) * 2021-09-03 2024-10-24 The University Of Chicago Polypeptides and methods for modifying nucleic acids
WO2023086953A1 (en) * 2021-11-11 2023-05-19 Beam Therapeutics Inc. Compositions and methods for the treatment of hereditary angioedema (hae)
CN114686456B (zh) * 2022-05-10 2023-02-17 中山大学 基于双分子脱氨酶互补的碱基编辑系统及其应用
EP4540378A1 (en) * 2022-06-20 2025-04-23 CRISPR Therapeutics AG Base editing proteins and uses thereof
EP4605542A1 (en) * 2022-10-19 2025-08-27 DNA Script Methods and products for removal of uracil containing polynucleotides
CN117384885B (zh) 2022-12-15 2025-06-10 中国科学院遗传与发育生物学研究所 一种碱基编辑器及其应用
CN118475691B (zh) * 2023-02-28 2025-07-22 锐正基因(苏州)有限公司 用于碱基编辑的脱氨酶
WO2024240223A1 (en) 2023-05-24 2024-11-28 Accuredit Therapeutics (Suzhou) Co., Ltd. Deaminases and variants thereof for use in base editing
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CN120400115A (zh) 2025-08-01
CA3127494A1 (en) 2020-08-06
JP2022521460A (ja) 2022-04-08
WO2020160517A1 (en) 2020-08-06
EP3918077A1 (en) 2021-12-08
EP3918077A4 (en) 2023-03-29
AU2020216484A1 (en) 2021-07-29
CN114072509A (zh) 2022-02-18
JP2025041661A (ja) 2025-03-26
JP7779988B2 (ja) 2025-12-03
JP7600122B2 (ja) 2024-12-16
CN114072509B (zh) 2025-04-15
US20220136012A1 (en) 2022-05-05

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