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

Info

Publication number
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
Authority
KR
South Korea
Prior art keywords
deaminase
cas9
amino acid
fusion protein
terminal fragment
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
KR1020217026724A
Other languages
English (en)
Korean (ko)
Inventor
이안 슬레이메이커
제이슨 마이클 제크
니콜 고델리
이 유
Original Assignee
빔 테라퓨틱스, 인크.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 빔 테라퓨틱스, 인크. filed Critical 빔 테라퓨틱스, 인크.
Publication of KR20210121113A publication Critical patent/KR20210121113A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • 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)
    • C—CHEMISTRY; METALLURGY
    • 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
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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

Family

ID=71842366

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217026724A Pending KR20210121113A (ko) 2019-01-31 2020-01-31 비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석

Country Status (7)

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

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
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
CA3194019A1 (en) * 2020-09-04 2022-03-10 National University Corporation Kobe University Miniaturized cytidine deaminase-containing complex for modifying double-stranded dna
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CN116601293A (zh) * 2020-12-07 2023-08-15 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
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
CA3214494A1 (en) * 2021-03-26 2022-09-29 Beam Therapeutics Inc. Adenosine deaminase variants and uses thereof
WO2022221699A1 (en) * 2021-04-16 2022-10-20 Beam Therapeutics, Inc. Genetic modification of hepatocytes
WO2023283092A1 (en) * 2021-07-06 2023-01-12 Prime Medicine, Inc. Compositions and methods for efficient genome editing
JP2024533311A (ja) 2021-09-08 2024-09-12 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー ゲノムを調節するための方法及び組成物
CN117925585A (zh) * 2022-04-07 2024-04-26 尧唐(上海)生物科技有限公司 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途
WO2025119307A1 (zh) * 2023-12-06 2025-06-12 北京齐禾生科生物科技有限公司 优化的qbe碱基编辑系统及其应用

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150165054A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CA2971444A1 (en) * 2014-12-20 2016-06-23 Arc Bio, Llc Compositions and methods for targeted depletion, enrichment, and partitioning of nucleic acids using crispr/cas system proteins
TWI813532B (zh) * 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的ｃｒｉｓｐｒ酶突變
WO2017048969A1 (en) * 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
JP7109784B2 (ja) * 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
WO2018119359A1 (en) * 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
CA3057192A1 (en) * 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
EP3625342B1 (en) * 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
AU2018290843B2 (en) * 2017-06-26 2025-04-24 Massachusetts Institute Of Technology CRISPR/Cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing

Also Published As

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

Similar Documents

Publication Publication Date Title
KR20210121113A (ko) 비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석
JP7657711B2 (ja) 核酸塩基編集システムを送達するための組成物および方法
KR102852347B1 (ko) 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 아미노산을 치환하는 방법
JP7717684B2 (ja) 新規核酸塩基エディター及びその使用方法
CN114040970B (zh) 使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法，包括遗传性疾病的治疗
JP7803917B2 (ja) 脂質ナノ粒子を用いるインビトロでのｍｒｎａの送達方法
US20230086199A1 (en) Systems and methods for evaluating cas9-independent off-target editing of nucleic acids
JP2025037975A (ja) アルファ－１アンチトリプシン不全を治療するための組成物および方法
CN116497067A (zh) 治疗血红素病变的组合物和方法
JP2025087691A (ja) 糖原病１ａ型を治療するための組成物および方法
KR20210023830A (ko) 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 돌연변이를 억제하는 방법
US20250333718A1 (en) Context-specific adenine base editors and uses thereof
JP2024511621A (ja) 新規ｃｒｉｓｐｒ酵素、方法、システム、及びそれらの使用
CN120586100A (zh) 用于治疗乙型肝炎的组合物和方法
JP2023545497A (ja) グリコーゲン貯蔵症１ａ型を治療するための組成物及び方法
JP2025533556A (ja) 合成ポリペプチド及びその用途
KR20240012377A (ko) 염기 편집기의 자기-불활성화용 조성물 및 방법
JP2024546841A (ja) 新規ｃｒｉｓｐｒ酵素、方法、システム、及びそれらの使用
KR20240007651A (ko) 간세포의 유전적 변형

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

R15-X000 Change to inventor requested

St.27 status event code: A-3-3-R10-R15-oth-X000

R16-X000 Change to inventor recorded

St.27 status event code: A-3-3-R10-R16-oth-X000

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

D22 Grant of ip right intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D22-EXM-PE0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701