KR20210121113A - 비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석 - Google Patents

비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석 Download PDF

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KR20210121113A
KR20210121113A KR1020217026724A KR20217026724A KR20210121113A KR 20210121113 A KR20210121113 A KR 20210121113A KR 1020217026724 A KR1020217026724 A KR 1020217026724A KR 20217026724 A KR20217026724 A KR 20217026724A KR 20210121113 A KR20210121113 A KR 20210121113A
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deaminase
cas9
amino acid
fusion protein
terminal fragment
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Korean (ko)
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이안 슬레이메이커
제이슨 마이클 제크
니콜 고델리
이 유
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빔 테라퓨틱스, 인크.
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    • C12N15/62—DNA sequences coding for fusion proteins
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80—Hydrolases (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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    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/04—Libraries containing only organic compounds
    • C40B40/10—Libraries containing peptides or polypeptides, or derivatives thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)

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  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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KR1020217026724A 2019-01-31 2020-01-31 비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석 Pending KR20210121113A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962799702P 2019-01-31 2019-01-31
US62/799,702 2019-01-31
PCT/US2020/016285 WO2020160514A1 (en) 2019-01-31 2020-01-31 Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors

Publications (1)

Publication Number Publication Date
KR20210121113A true KR20210121113A (ko) 2021-10-07

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KR1020217026724A Pending KR20210121113A (ko) 2019-01-31 2020-01-31 비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석

Country Status (8)

Country Link
US (1) US20220098572A1 (enExample)
EP (1) EP3918078A4 (enExample)
JP (2) JP7646552B2 (enExample)
KR (1) KR20210121113A (enExample)
CN (1) CN114040977B (enExample)
AU (1) AU2020215730A1 (enExample)
CA (1) CA3127493A1 (enExample)
WO (1) WO2020160514A1 (enExample)

Families Citing this family (13)

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US20180250424A1 (en) 2014-10-10 2018-09-06 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP3799603A4 (en) 2018-05-11 2022-03-02 Beam Therapeutics, Inc. METHODS OF PATHOGENIC AMINO ACID SUBSTITUTION USING PROGRAMMABLE BASE EDITOR SYSTEMS
US12454694B2 (en) 2018-09-07 2025-10-28 Beam Therapeutics Inc. Compositions and methods for improving base editing
IL296024A (en) 2020-03-04 2022-10-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for modulating a genome
WO2021222318A1 (en) 2020-04-28 2021-11-04 The Broad Institute, Inc. Targeted base editing of the ush2a gene
CN116134141A (zh) * 2020-09-04 2023-05-16 国立大学法人神户大学 包含小型化胞苷脱氨酶的双链dna修饰用复合体
WO2022120520A1 (en) * 2020-12-07 2022-06-16 Institute Of Zoology, Chinese Academy Of Sciences Engineered cas effector proteins and methods of use thereof
CN113308451B (zh) * 2020-12-07 2023-07-25 中国科学院动物研究所 工程化的Cas效应蛋白及其使用方法
KR20240004276A (ko) * 2021-03-26 2024-01-11 빔 테라퓨틱스, 인크. 아데노신 데아미나아제 변이체 및 이의 용도
WO2023283092A1 (en) * 2021-07-06 2023-01-12 Prime Medicine, Inc. Compositions and methods for efficient genome editing
MX2024002927A (es) 2021-09-08 2024-05-29 Flagship Pioneering Innovations Vi Llc Metodos y composiciones para modular un genoma.
US20250361530A1 (en) * 2022-05-14 2025-11-27 The Regents Of The University Of California Genome editing systems for multiplexing point mutation introduction in living cells
WO2025119306A1 (zh) * 2023-12-06 2025-06-12 北京齐禾生科生物科技有限公司 优化工程化t细胞的碱基编辑系统及其应用

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015089486A2 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
US11053481B2 (en) * 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
AU2015364286B2 (en) * 2014-12-20 2021-11-04 Arc Bio, Llc Compositions and methods for targeted depletion, enrichment, and partitioning of nucleic acids using CRISPR/Cas system proteins
EP3250689B1 (en) * 2015-01-28 2020-11-04 The Regents of The University of California Methods and compositions for labeling a single-stranded target nucleic acid
US10392607B2 (en) * 2015-06-03 2019-08-27 The Regents Of The University Of California Cas9 variants and methods of use thereof
JP7107683B2 (ja) * 2015-06-18 2022-07-27 ザ・ブロード・インスティテュート・インコーポレイテッド オフターゲット効果を低下させるｃｒｉｓｐｒ酵素突然変異
EP3350203A4 (en) * 2015-09-17 2019-06-26 The Regents of The University of California POLYPEPTIDES OF CAS9 VARIANT WITH INTERNAL INSERTIONS
US12043852B2 (en) * 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
GB2568182A (en) * 2016-08-03 2019-05-08 Harvard College Adenosine nucleobase editors and uses thereof
WO2018074979A1 (en) * 2016-10-17 2018-04-26 Nanyang Technological University Truncated crispr-cas proteins for dna targeting
WO2018119359A1 (en) * 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11268082B2 (en) 2017-03-23 2022-03-08 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
US12297436B2 (en) 2017-05-18 2025-05-13 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
EP3645054A4 (en) 2017-06-26 2021-03-31 The Broad Institute, Inc. CRISPR / CAS-ADENINE DESAMINASE COMPOSITIONS, TARGETED NUCLEIC ACID EDITING SYSTEMS AND METHODS

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US20220098572A1 (en) 2022-03-31
CN114040977A (zh) 2022-02-11
JP2022519507A (ja) 2022-03-24
EP3918078A1 (en) 2021-12-08
EP3918078A4 (en) 2022-12-14
JP2025102784A (ja) 2025-07-08
CN114040977B (zh) 2024-09-03
JP7646552B2 (ja) 2025-03-17
AU2020215730A1 (en) 2021-07-29
WO2020160514A1 (en) 2020-08-06
CA3127493A1 (en) 2020-08-06

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