KR102712986B1 - 조작된 표적 특이적 염기 편집기 - Google Patents

조작된 표적 특이적 염기 편집기 Download PDF

Info

Publication number
KR102712986B1
KR102712986B1 KR1020217008090A KR20217008090A KR102712986B1 KR 102712986 B1 KR102712986 B1 KR 102712986B1 KR 1020217008090 A KR1020217008090 A KR 1020217008090A KR 20217008090 A KR20217008090 A KR 20217008090A KR 102712986 B1 KR102712986 B1 KR 102712986B1
Authority
KR
South Korea
Prior art keywords
leu
dna
ala
gly
glu
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.)
Active
Application number
KR1020217008090A
Other languages
English (en)
Korean (ko)
Other versions
KR20210049124A (ko
Inventor
프리드리히 파우저
제프리 씨. 밀러
에드워드 레바
Original Assignee
상가모 테라퓨틱스, 인코포레이티드
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 상가모 테라퓨틱스, 인코포레이티드 filed Critical 상가모 테라퓨틱스, 인코포레이티드
Publication of KR20210049124A publication Critical patent/KR20210049124A/ko
Application granted granted Critical
Publication of KR102712986B1 publication Critical patent/KR102712986B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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)
    • C12Y305/04001Cytosine deaminase (3.5.4.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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)
    • C12Y305/04002Adenine deaminase (3.5.4.2)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181Peptide nucleic acid, PNA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2521/00Reaction characterised by the enzymatic activity
    • C12Q2521/30Phosphoric diester hydrolysing, i.e. nuclease
    • C12Q2521/307Single strand endonuclease
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2521/00Reaction characterised by the enzymatic activity
    • C12Q2521/50Other enzymatic activities
    • C12Q2521/539Deaminase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2522/00Reaction characterised by the use of non-enzymatic proteins
    • C12Q2522/10Nucleic acid binding proteins
    • C12Q2522/101Single or double stranded nucleic acid binding proteins

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020217008090A 2018-08-23 2019-08-20 조작된 표적 특이적 염기 편집기 Active KR102712986B1 (ko)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862721903P 2018-08-23 2018-08-23
US62/721,903 2018-08-23
US201862753696P 2018-10-31 2018-10-31
US62/753,696 2018-10-31
US201962817153P 2019-03-12 2019-03-12
US62/817,153 2019-03-12
US201962867565P 2019-06-27 2019-06-27
US62/867,565 2019-06-27
PCT/US2019/047172 WO2020041249A1 (en) 2018-08-23 2019-08-20 Engineered target specific base editors

Publications (2)

Publication Number Publication Date
KR20210049124A KR20210049124A (ko) 2021-05-04
KR102712986B1 true KR102712986B1 (ko) 2024-10-07

Family

ID=69583877

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217008090A Active KR102712986B1 (ko) 2018-08-23 2019-08-20 조작된 표적 특이적 염기 편집기

Country Status (10)

Country Link
US (1) US11834686B2 (enExample)
EP (1) EP3841204A4 (enExample)
JP (1) JP7541508B2 (enExample)
KR (1) KR102712986B1 (enExample)
CN (1) CN112867792A (enExample)
AU (1) AU2019326408A1 (enExample)
CA (1) CA3109592A1 (enExample)
IL (1) IL280951B2 (enExample)
SG (1) SG11202101801RA (enExample)
WO (1) WO2020041249A1 (enExample)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019326408A1 (en) 2018-08-23 2021-03-11 Sangamo Therapeutics, Inc. Engineered target specific base editors
JP7059965B2 (ja) * 2019-02-21 2022-04-26 株式会社豊田中央研究所 エピゲノム状態を制御する融合タンパク質及びその利用
WO2021155166A1 (en) * 2020-01-31 2021-08-05 University Of Massachusetts A cas9-pdbd base editor platform with improved targeting range and specificity
CN111518794B (zh) * 2020-04-13 2023-05-16 中山大学 基于激活诱导性胞苷脱氨酶的诱导突变蛋白的制备和用途
CN111690720B (zh) * 2020-06-16 2021-06-15 山东舜丰生物科技有限公司 利用修饰的单链核酸进行靶核酸检测的方法
CA3187135A1 (en) * 2020-06-30 2022-01-06 Pairwise Plants Services, Inc. Compositions, systems, and methods for base diversification
US20230340486A1 (en) * 2020-07-27 2023-10-26 The Children’S Hospital Of Philadelphia In utero and postnatal gene editing and therapy for treatment of monogenic diseases, including mucopolysaccharidosis type 1h and other disorders
CN116261594A (zh) * 2020-09-25 2023-06-13 桑格摩生物治疗股份有限公司 用于核碱基编辑的锌指融合蛋白
CA3196347A1 (en) * 2020-10-23 2022-04-28 Jonathan Thon Compositions and methods related to megakaryocyte-derived extracellular vesicles for treating myeloproliferative neoplasms
CN117015552A (zh) * 2020-10-23 2023-11-07 斯特姆生物公司 用于治疗骨髓增殖性肿瘤的与巨核细胞来源的细胞外囊泡相关的组合物和方法
IL303506A (en) * 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
CA3218511A1 (en) 2021-05-10 2022-11-17 Sqz Biotechnologies Company Methods for delivering genome editing molecules to the nucleus or cytosol of a cell and uses thereof
WO2022271548A2 (en) * 2021-06-23 2022-12-29 Massachusetts Institute Of Technology Compositions, methods and systems for the delivery of gene editing material to cells
AU2022317661B2 (en) * 2021-07-26 2026-04-23 Dana-Farber Cancer Institute, Inc. Small molecule inhibition of deubiquitinating enzyme josephin domain containing 1 (josd1) as a targeted therapy for leukemias with mutant janus kinase 2 (jak2)
US20240417755A1 (en) * 2021-09-27 2024-12-19 Vor Biopharma Inc. Fusion polypeptides for genetic editing and methods of use thereof
US20240415979A1 (en) * 2021-10-23 2024-12-19 Strm.Bio Incorporated Gene editing to treat myeloproliferative neoplasms
JP2025504606A (ja) * 2021-12-22 2025-02-13 サンガモ セラピューティクス, インコーポレイテッド 核酸塩基編集のための新規ジンクフィンガー融合タンパク質
CN116497002A (zh) * 2022-01-19 2023-07-28 中国科学院动物研究所 工程化的CasX核酸酶、效应蛋白及其用途
EP4482855A4 (en) * 2022-02-22 2026-03-04 Peter Biotherapeutics Inc GENE MODIFICATION TECHNOLOGIES
JP2023131616A (ja) * 2022-03-09 2023-09-22 国立大学法人広島大学 Dna編集システム、並びに、それを用いた標的dnaの編集方法及び標的dnaが編集された細胞の製造方法
KR20230139064A (ko) * 2022-03-25 2023-10-05 기초과학연구원 식물세포 소기관 dna의 아데닌을 구아닌으로 염기 교정
CN119923471A (zh) * 2022-09-21 2025-05-02 基础科学研究院 植物细胞器dna的碱基编辑
JP2025534400A (ja) * 2022-09-30 2025-10-15 北京昌平▲実▼▲験▼室 二本鎖dnaデアミナーゼを用いたフットプリンティングによりゲノムワイドdna結合タンパク質結合部位を決定するための方法
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
JP2026501648A (ja) * 2022-12-30 2026-01-16 北京大学 塩基エディターシステムおよびその使用方法
WO2024155727A1 (en) * 2023-01-17 2024-07-25 The Broad Institute, Inc. Systems and methods for targeted continuous genome mutagenesis
WO2025259055A1 (ko) * 2024-06-13 2025-12-18 주식회사 엣진 징크 핑거 단백질 및 닉카제를 포함하는 신규한 염기 에디터

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170073670A1 (en) 2014-03-05 2017-03-16 National University Corporation Kobe University Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
WO2017070633A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
WO2018119354A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Gene editing of pcsk9
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection

Family Cites Families (97)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5928638A (en) 1996-06-17 1999-07-27 Systemix, Inc. Methods for gene transfer
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
ES2341926T3 (es) 1998-03-02 2010-06-29 Massachusetts Institute Of Technology Poliproteinas con dedos de cinc que tienen enlazadores mejorados.
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
AU776576B2 (en) 1999-12-06 2004-09-16 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US6689558B2 (en) 2000-02-08 2004-02-10 Sangamo Biosciences, Inc. Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US7923542B2 (en) 2000-04-28 2011-04-12 Sangamo Biosciences, Inc. Libraries of regulatory sequences, methods of making and using same
AU2001255870B2 (en) 2000-04-28 2006-07-13 Sangamo Biosciences, Inc. Databases of regulatory sequences; methods of making and using same
ATE353361T1 (de) 2000-04-28 2007-02-15 Sangamo Biosciences Inc Gezielten modifikation der chromatinstruktur
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Cnnについての亜鉛フィンガー結合ドメイン
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
WO2003087341A2 (en) 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
WO2003080809A2 (en) 2002-03-21 2003-10-02 Sangamo Biosciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
EP2806025B1 (en) 2002-09-05 2019-04-03 California Institute of Technology Use of zinc finger nucleases to stimulate gene targeting
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
AU2005232665B2 (en) 2004-04-08 2010-05-13 Sangamo Therapeutics, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
US7253273B2 (en) 2004-04-08 2007-08-07 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
AU2005287278B2 (en) 2004-09-16 2011-08-04 Sangamo Biosciences, Inc. Compositions and methods for protein production
CA2607104A1 (en) 2005-05-05 2006-11-16 The Arizona Board Of Regents On Behalf Of The University Of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
GB0607063D0 (en) 2006-04-07 2006-05-17 Cellcentric Ltd Compositions and methods for epigenetic modification of nucleic acid sequences in vivo
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
EP2027262B1 (en) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variant foki cleavage half-domains
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
CA2720903C (en) 2008-04-14 2019-01-15 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
US9394531B2 (en) 2008-05-28 2016-07-19 Sangamo Biosciences, Inc. Compositions for linking DNA-binding domains and cleavage domains
WO2009151591A2 (en) 2008-06-10 2009-12-17 Sangamo Biosciences, Inc. Methods and compositions for generation of bax- and bak-deficient cell lines
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
EP2352369B1 (en) 2008-12-04 2017-04-26 Sangamo BioSciences, Inc. Genome editing in rats using zinc-finger nucleases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
WO2011072246A2 (en) 2009-12-10 2011-06-16 Regents Of The University Of Minnesota Tal effector-mediated dna modification
ES2751916T3 (es) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
HRP20200254T1 (hr) 2010-05-03 2020-05-29 Sangamo Therapeutics, Inc. Pripravci za povezivanje modula cink fingera
AU2011281062B2 (en) 2010-07-21 2015-01-22 Board Of Regents, The University Of Texas System Methods and compositions for modification of a HLA locus
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA3099582A1 (en) 2011-10-27 2013-05-02 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US9255250B2 (en) 2012-12-05 2016-02-09 Sangamo Bioscience, Inc. Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
CN105940013B (zh) 2013-12-09 2020-03-27 桑格摩生物科学股份有限公司 用于治疗血友病的方法和组合物
EP3099801B1 (en) 2014-01-31 2020-03-18 Factor Bioscience Inc. Synthetic rna for use in the treatment of dystrophic epidermolysis bullosa
WO2015164748A1 (en) 2014-04-24 2015-10-29 Sangamo Biosciences, Inc. Engineered transcription activator like effector (tale) proteins
US20170198268A1 (en) 2014-07-09 2017-07-13 Gen9, Inc. Compositions and Methods for Site-Directed DNA Nicking and Cleaving
US10190106B2 (en) 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
AU2016261927B2 (en) 2015-05-12 2022-04-07 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
KR20250141836A (ko) * 2015-11-04 2025-09-29 페이트 세러퓨틱스, 인코포레이티드 만능 세포의 유전자 조작
JP6923205B2 (ja) 2015-11-27 2021-08-18 国立大学法人神戸大学 標的化したdna配列の核酸塩基を特異的に変換する、単子葉植物のゲノム配列の変換方法、及びそれに用いる分子複合体
EP3389677B1 (en) 2015-12-18 2024-06-26 Sangamo Therapeutics, Inc. Targeted disruption of the t cell receptor
KR20180101442A (ko) 2016-02-02 2018-09-12 상가모 테라퓨틱스, 인코포레이티드 Dna-결합 도메인 및 절단 도메인을 연결시키기 위한 조성물
CA3209273A1 (en) 2016-06-02 2017-12-07 Sigma-Aldrich Co. Llc Using programmable dna binding proteins to enhance targeted genome modification
KR102455249B1 (ko) 2016-08-24 2022-10-17 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US20190255106A1 (en) * 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
WO2018119243A1 (en) 2016-12-21 2018-06-28 University Of Florida Research Foundation, Incorporated Ccr2+hematopoietic stem cells mediate t cell activation in adoptive cell therapy
CN106916852B (zh) * 2017-04-13 2020-12-04 上海科技大学 一种碱基编辑系统及其构建和应用方法
EP3630849A4 (en) 2017-05-25 2021-01-13 The General Hospital Corporation BIPARTITE BASIC EDITOR (BBE) AND TYPE II-C-CAS9 ZINC FINGER EDITOR ARCHITECTURES
AU2019326408A1 (en) 2018-08-23 2021-03-11 Sangamo Therapeutics, Inc. Engineered target specific base editors
US20250011748A1 (en) 2020-01-28 2025-01-09 The Broad Institute, Inc. Base editors, compositions, and methods for modifying the mitochondrial genome
JP2024502630A (ja) 2021-01-12 2024-01-22 マーチ セラピューティクス, インコーポレイテッド コンテキスト依存性二本鎖dna特異的デアミナーゼ及びその使用

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170073670A1 (en) 2014-03-05 2017-03-16 National University Corporation Kobe University Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
WO2017070633A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
WO2018119354A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Gene editing of pcsk9
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection

Also Published As

Publication number Publication date
IL280951A (en) 2021-04-29
AU2019326408A1 (en) 2021-03-11
IL280951B1 (en) 2024-04-01
EP3841204A4 (en) 2022-05-18
CN112867792A (zh) 2021-05-28
EP3841204A1 (en) 2021-06-30
WO2020041249A1 (en) 2020-02-27
JP2021536229A (ja) 2021-12-27
CA3109592A1 (en) 2020-02-27
SG11202101801RA (en) 2021-03-30
IL280951B2 (en) 2024-08-01
JP7541508B2 (ja) 2024-08-28
KR20210049124A (ko) 2021-05-04
US11834686B2 (en) 2023-12-05
US20200063114A1 (en) 2020-02-27

Similar Documents

Publication Publication Date Title
KR102712986B1 (ko) 조작된 표적 특이적 염기 편집기
JP6875362B2 (ja) ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
EP3504327B1 (en) Engineered target specific nucleases
US10538787B2 (en) Compositions for linking DNA-binding domains and cleavage domains
CN111954540A (zh) 工程化靶标特异性核酸酶
CN110869497A (zh) 修饰囊性纤维化跨膜传导调节蛋白(cftr)基因的方法和组合物
RU2782794C2 (ru) Сконструированные мишень-специфические нуклеазы
HK40069273A (en) Engineered target specific nucleases
HK40011095B (en) Engineered target specific nucleases
NZ791740A (en) Engineered target specific nucleases

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20210318

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20220819

Comment text: Request for Examination of Application

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

Comment text: Notification of reason for refusal

Patent event date: 20231031

Patent event code: PE09021S01D

PE0701 Decision of registration

Patent event code: PE07011S01D

Comment text: Decision to Grant Registration

Patent event date: 20240627

GRNT Written decision to grant
PR0701 Registration of establishment

Comment text: Registration of Establishment

Patent event date: 20240927

Patent event code: PR07011E01D

PR1002 Payment of registration fee

Payment date: 20240930

End annual number: 3

Start annual number: 1

PG1601 Publication of registration