JP7541508B2 - 操作された標的特異的な塩基エディター - Google Patents

操作された標的特異的な塩基エディター Download PDF

Info

Publication number
JP7541508B2
JP7541508B2 JP2021510000A JP2021510000A JP7541508B2 JP 7541508 B2 JP7541508 B2 JP 7541508B2 JP 2021510000 A JP2021510000 A JP 2021510000A JP 2021510000 A JP2021510000 A JP 2021510000A JP 7541508 B2 JP7541508 B2 JP 7541508B2
Authority
JP
Japan
Prior art keywords
dna
domain
editing
base
nickase
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
JP2021510000A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021536229A (ja
JP2021536229A5 (https=
JPWO2020041249A5 (https=
Inventor
フリードリック・ファウザー
ジェフリー・シー・ミラー
エドワード・リバー
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sangamo Therapeutics Inc
Original Assignee
Sangamo Therapeutics Inc
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 Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of JP2021536229A publication Critical patent/JP2021536229A/ja
Publication of JP2021536229A5 publication Critical patent/JP2021536229A5/ja
Publication of JPWO2020041249A5 publication Critical patent/JPWO2020041249A5/ja
Application granted granted Critical
Publication of JP7541508B2 publication Critical patent/JP7541508B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N15/10—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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • 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)
    • C12Y305/04001—Cytosine deaminase (3.5.4.1)
    • 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)
    • C12Y305/04002—Adenine deaminase (3.5.4.2)
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q2521/00—Reaction characterised by the enzymatic activity
    • C12Q2521/30—Phosphoric diester hydrolysing, i.e. nuclease
    • C12Q2521/307—Single strand endonuclease
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q2521/00—Reaction characterised by the enzymatic activity
    • C12Q2521/50—Other enzymatic activities
    • C12Q2521/539—Deaminase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q2522/00—Reaction characterised by the use of non-enzymatic proteins
    • C12Q2522/10—Nucleic acid binding proteins
    • C12Q2522/101—Single 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)
JP2021510000A 2018-08-23 2019-08-20 操作された標的特異的な塩基エディター Active JP7541508B2 (ja)

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 (4)

Publication Number Publication Date
JP2021536229A JP2021536229A (ja) 2021-12-27
JP2021536229A5 JP2021536229A5 (https=) 2022-08-29
JPWO2020041249A5 JPWO2020041249A5 (https=) 2022-08-29
JP7541508B2 true JP7541508B2 (ja) 2024-08-28

Family

ID=69583877

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021510000A Active JP7541508B2 (ja) 2018-08-23 2019-08-20 操作された標的特異的な塩基エディター

Country Status (10)

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

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7541508B2 (ja) 2018-08-23 2024-08-28 サンガモ セラピューティクス， インコーポレイテッド 操作された標的特異的な塩基エディター
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
EP4097233A4 (en) * 2020-01-31 2024-02-28 University of Massachusetts CAS9-PDBD CORE 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
EP4172328A1 (en) 2020-06-30 2023-05-03 Pairwise Plants Services, Inc. Compositions, systems, and methods for base diversification
WO2022027035A1 (en) * 2020-07-27 2022-02-03 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
KR20230074519A (ko) * 2020-09-25 2023-05-30 상가모 테라퓨틱스, 인코포레이티드 핵염기 편집을 위한 아연 핑거 융합 단백질
WO2022087505A1 (en) * 2020-10-23 2022-04-28 Strm.Bio Incorporated 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
CR20230305A (es) * 2020-12-11 2023-11-10 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación
WO2022240846A1 (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
WO2023009982A1 (en) * 2021-07-26 2023-02-02 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
WO2023070122A1 (en) * 2021-10-23 2023-04-27 Strm.Bio Incorporated Gene editing to treat myeloproliferative neoplasms
CN118591626A (zh) * 2021-12-22 2024-09-03 桑格摩生物治疗股份有限公司 用于核碱基编辑的新型锌指融合蛋白
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
US20260035677A1 (en) * 2022-02-22 2026-02-05 Peter Biotherapeutics, Inc. Technologies for genetic modification
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
KR20240041835A (ko) * 2022-09-21 2024-04-01 기초과학연구원 식물세포 소기관 dna의 염기 교정
EP4594528A1 (en) * 2022-09-30 2025-08-06 Beijing Changping Laboratory Methods of determining genome-wide dna binding protein binding sites by footprinting with double stranded dna deaminase
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
KR20250177381A (ko) * 2024-06-13 2025-12-23 주식회사 엣진 징크 핑거 단백질 및 닉카제를 포함하는 신규한 염기 에디터

Citations (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090068164A1 (en) 2005-05-05 2009-03-12 The Ariz Bd Of Regents On Behald Of The Univ Of Az Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
JP2009532053A (ja) 2006-04-07 2009-09-10 セルセントリック・リミテッド インヴィヴォでの核酸配列のエピジェネティック修飾用組成物および方法
US20100047805A1 (en) 2008-08-22 2010-02-25 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
WO2015133554A1 (ja) 2014-03-05 2015-09-11 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ゲノム配列の改変方法及びそれに用いる分子複合体
US20160177278A1 (en) 2014-12-22 2016-06-23 University Of Massachusetts Cas9-DNA Targeting Unit Chimeras
WO2017070633A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
WO2017090761A1 (ja) 2015-11-27 2017-06-01 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
US20170198268A1 (en) 2014-07-09 2017-07-13 Gen9, Inc. Compositions and Methods for Site-Directed DNA Nicking and Cleaving
WO2017209809A1 (en) 2016-06-02 2017-12-07 Sigma-Aldrich Co. Llc Using programmable dna binding proteins to enhance targeted genome modification
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
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
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
WO2018218166A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Using split deaminases to limit unwanted off-target base editor deamination

Family Cites Families (87)

* 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
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6242568B1 (en) 1994-01-18 2001-06-05 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
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
ATE524545T1 (de) 1994-08-20 2011-09-15 Gendaq Ltd Verbesserungen bei oder im zusammenhang mit bindeproteinen zur erkennung von dna
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
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (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
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics 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
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
CA2394850C (en) 1999-12-06 2012-02-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
KR20020086508A (ko) 2000-02-08 2002-11-18 상가모 바이오사이언스 인코포레이티드 약물 발견용 세포
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
EP1276859B1 (en) 2000-04-28 2007-02-07 Sangamo Biosciences Inc. Targeted modification of chromatin structure
US7923542B2 (en) 2000-04-28 2011-04-12 Sangamo Biosciences, Inc. Libraries of regulatory sequences, methods of making and using same
US7217509B2 (en) 2000-04-28 2007-05-15 Sangamo Biosciences, Inc. Databases of regulatory sequences; methods of making and using same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith 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
US20050267061A1 (en) 2004-04-08 2005-12-01 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
EP1476547B1 (en) 2002-01-23 2006-12-06 The University of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl 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
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
JP2008506359A (ja) 2004-04-08 2008-03-06 サンガモ バイオサイエンシズ インコーポレイテッド ジンクフィンガータンパク質による神経因性疼痛の処置
KR20070060115A (ko) 2004-09-16 2007-06-12 상가모 바이오사이언스 인코포레이티드 단백질 생산을 위한 조성물 및 방법
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
WO2008133938A2 (en) 2007-04-26 2008-11-06 Sangamo Biosciences, Inc. Targeted integration into the ppp1r12c locus
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
EP2281050B1 (en) 2008-04-14 2014-04-02 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
KR101802393B1 (ko) 2008-06-10 2017-11-28 상가모 테라퓨틱스, 인코포레이티드 Ｂａｘ- 및 ｂａｋ-결함 세포주들의 제조 방법 및 조성물
EP3156494B8 (en) 2008-12-04 2018-09-19 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
HUE041436T2 (hu) 2009-12-10 2019-05-28 Univ Minnesota Tal-effektor-közvetített DNS-módosítás
EP3594333B1 (en) 2010-02-08 2023-11-01 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US8771985B2 (en) 2010-04-26 2014-07-08 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using zinc-finger nucleases
PT2566972T (pt) 2010-05-03 2020-03-02 Sangamo Biosciences Inc Composições para a ligação de módulos de dedo de zinco
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド 新規のｄｎａ結合タンパク質及びその使用
CA2993567C (en) 2010-07-21 2022-06-28 Sangamo Biosciences, Inc. Methods and compositions for modification of a t-cell receptor gene
AU2012312260B2 (en) 2011-09-21 2017-08-31 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
JP6188703B2 (ja) 2011-10-27 2017-08-30 サンガモ セラピューティクス， インコーポレイテッド Ｈｐｒｔ遺伝子座を修飾するための方法および組成物
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2014089212A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
CN105121648B (zh) 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
AU2013359262C1 (en) 2012-12-12 2021-05-13 Massachusetts Institute Of Technology CRISPR-Cas component systems, methods and compositions for sequence manipulation
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
CA2894684A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
AU2014227653B2 (en) 2013-03-15 2017-04-20 The General Hospital Corporation Using RNA-guided foki nucleases (RFNs) to increase specificity for RNA-guided genome editing
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
DK3080274T3 (da) 2013-12-09 2020-08-31 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til genom-manipulation
ES2939542T3 (es) 2014-01-31 2023-04-24 Factor Bioscience Inc Métodos y productos para la producción y la administración de ácido nucleico
WO2015164748A1 (en) 2014-04-24 2015-10-29 Sangamo Biosciences, Inc. Engineered transcription activator like effector (tale) proteins
MX382223B (es) 2015-05-12 2025-03-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
JP6928604B2 (ja) * 2015-11-04 2021-09-01 フェイト セラピューティクス，インコーポレイテッド 万能性細胞のゲノム改変
AU2016369490C1 (en) 2015-12-18 2021-12-23 Sangamo Therapeutics, Inc. Targeted disruption of the T cell receptor
EP3411056A4 (en) 2016-02-02 2019-10-02 Sangamo Therapeutics, Inc. COMPOUNDS FOR NETWORKING DNA BINDING DOMAINS AND SPLITTING DOMAINS
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
JP7682604B2 (ja) * 2016-09-07 2025-05-26 フラッグシップ パイオニアリング イノベーションズ ブイ， インコーポレイテッド 遺伝子発現をモジュレートするための方法および組成物
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
JP7541508B2 (ja) 2018-08-23 2024-08-28 サンガモ セラピューティクス， インコーポレイテッド 操作された標的特異的な塩基エディター
CA3166153A1 (en) 2020-01-28 2021-08-05 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 (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090068164A1 (en) 2005-05-05 2009-03-12 The Ariz Bd Of Regents On Behald Of The Univ Of Az Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
JP2009532053A (ja) 2006-04-07 2009-09-10 セルセントリック・リミテッド インヴィヴォでの核酸配列のエピジェネティック修飾用組成物および方法
US20100047805A1 (en) 2008-08-22 2010-02-25 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
WO2015133554A1 (ja) 2014-03-05 2015-09-11 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ゲノム配列の改変方法及びそれに用いる分子複合体
US20170198268A1 (en) 2014-07-09 2017-07-13 Gen9, Inc. Compositions and Methods for Site-Directed DNA Nicking and Cleaving
US20160177278A1 (en) 2014-12-22 2016-06-23 University Of Massachusetts Cas9-DNA Targeting Unit Chimeras
WO2017070633A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
WO2017090761A1 (ja) 2015-11-27 2017-06-01 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
WO2017209809A1 (en) 2016-06-02 2017-12-07 Sigma-Aldrich Co. Llc Using programmable dna binding proteins to enhance targeted genome modification
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
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
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
WO2018218166A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Using split deaminases to limit unwanted off-target base editor deamination
WO2018218206A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Bipartite base editor (bbe) architectures and type-ii-c-cas9 zinc finger editing

Also Published As

Publication number Publication date
EP3841204A1 (en) 2021-06-30
JP2021536229A (ja) 2021-12-27
SG11202101801RA (en) 2021-03-30
US20200063114A1 (en) 2020-02-27
EP3841204A4 (en) 2022-05-18
IL280951B2 (en) 2024-08-01
AU2019326408A1 (en) 2021-03-11
US11834686B2 (en) 2023-12-05
KR102712986B1 (ko) 2024-10-07
KR20210049124A (ko) 2021-05-04
CA3109592A1 (en) 2020-02-27
IL280951A (en) 2021-04-29
IL280951B1 (en) 2024-04-01
CN112867792A (zh) 2021-05-28
WO2020041249A1 (en) 2020-02-27

Similar Documents

Publication Publication Date Title
JP7541508B2 (ja) 操作された標的特異的な塩基エディター
US20240229079A9 (en) Engineered target specific nucleases
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
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

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220819

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220819

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230905

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20231204

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240202

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240304

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20240502

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20240618

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240628

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20240816

R150 Certificate of patent or registration of utility model

Ref document number: 7541508

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150