JP7518620B2 - 操作されたｃａｓ９ヌクレアーゼ - Google Patents

操作されたｃａｓ９ヌクレアーゼ Download PDF

Info

Publication number
JP7518620B2
JP7518620B2 JP2019567686A JP2019567686A JP7518620B2 JP 7518620 B2 JP7518620 B2 JP 7518620B2 JP 2019567686 A JP2019567686 A JP 2019567686A JP 2019567686 A JP2019567686 A JP 2019567686A JP 7518620 B2 JP7518620 B2 JP 7518620B2
Authority
JP
Japan
Prior art keywords
polypeptide
cas9
target
dna
selection
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
JP2019567686A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020524497A (ja
JP2020524497A5 (enExample
Inventor
バレット イーサン スタインバーグ
デレク セルチオーネ
Original Assignee
エディタス・メディシン，インコーポレイテッド
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 エディタス・メディシン，インコーポレイテッド filed Critical エディタス・メディシン，インコーポレイテッド
Publication of JP2020524497A publication Critical patent/JP2020524497A/ja
Publication of JP2020524497A5 publication Critical patent/JP2020524497A5/ja
Priority to JP2023165873A priority Critical patent/JP7695315B2/ja
Application granted granted Critical
Publication of JP7518620B2 publication Critical patent/JP7518620B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • 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
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2019567686A 2017-06-09 2018-06-08 操作されたｃａｓ９ヌクレアーゼ Active JP7518620B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023165873A JP7695315B2 (ja) 2017-06-09 2023-09-27 操作されたｃａｓ９ヌクレアーゼ

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762517811P 2017-06-09 2017-06-09
US62/517,811 2017-06-09
US201862665388P 2018-05-01 2018-05-01
US62/665,388 2018-05-01
PCT/US2018/036695 WO2018227114A1 (en) 2017-06-09 2018-06-08 Engineered cas9 nucleases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023165873A Division JP7695315B2 (ja) 2017-06-09 2023-09-27 操作されたｃａｓ９ヌクレアーゼ

Publications (3)

Publication Number Publication Date
JP2020524497A JP2020524497A (ja) 2020-08-20
JP2020524497A5 JP2020524497A5 (enExample) 2021-07-26
JP7518620B2 true JP7518620B2 (ja) 2024-07-18

Family

ID=62779085

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019567686A Active JP7518620B2 (ja) 2017-06-09 2018-06-08 操作されたｃａｓ９ヌクレアーゼ
JP2023165873A Active JP7695315B2 (ja) 2017-06-09 2023-09-27 操作されたｃａｓ９ヌクレアーゼ

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023165873A Active JP7695315B2 (ja) 2017-06-09 2023-09-27 操作されたｃａｓ９ヌクレアーゼ

Country Status (9)

Country Link
US (3) US10428319B2 (enExample)
EP (1) EP3635104A1 (enExample)
JP (2) JP7518620B2 (enExample)
KR (1) KR102746733B1 (enExample)
CN (1) CN110997908A (enExample)
AU (1) AU2018279829B2 (enExample)
CA (1) CA3065813A1 (enExample)
MX (1) MX2019014640A (enExample)
WO (1) WO2018227114A1 (enExample)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
WO2018129368A2 (en) 2017-01-06 2018-07-12 Editas Medicine, Inc. Methods of assessing nuclease cleavage
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
CN110997908A (zh) 2017-06-09 2020-04-10 爱迪塔斯医药公司 工程化的cas9核酸酶
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CA3084733A1 (en) 2017-12-14 2019-06-20 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant cone-rod dystrophy
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
CN113661392B (zh) 2019-02-05 2025-05-27 卡蒂亚生物公司 有效靶向基因组操纵剂的增强选择
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
CN114206376A (zh) 2019-07-11 2022-03-18 阿伯生物技术公司 新型crispr dna靶向酶及系统
CN112538471B (zh) * 2020-12-28 2023-12-12 南方医科大学 一种CRISPR SpCas9(K510A)突变体及其应用
MX2023009036A (es) * 2021-02-01 2023-10-30 Epsilen Bio S R L Silenciamiento de genes.
EP4286401A1 (en) * 2022-06-03 2023-12-06 Technische Universität Dresden Use of an artificial protein or of a nucleic acid encoding the artificial protein for providing a functional ribonucleoprotein complex
WO2024147677A1 (ko) * 2023-01-04 2024-07-11 고려대학교 산학협력단 2차 대사산물 증가를 위한 유전자 조작된 cas9 시스템과 이를 이용한 2차 대사산물 증진 방법
CN117866926B (zh) * 2024-03-07 2024-08-16 珠海舒桐医疗科技有限公司 一种CRISPR-FrCas9蛋白突变体及应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016205613A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzyme mutations reducing off-target effects
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases

Family Cites Families (180)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
US7476500B1 (en) 2001-03-19 2009-01-13 President And Fellows Of Harvard College In vivo selection system for enzyme activity
EP2325332B1 (en) 2005-08-26 2012-10-31 DuPont Nutrition Biosciences ApS Use of CRISPR associated genes (CAS)
US8546553B2 (en) 2008-07-25 2013-10-01 University Of Georgia Research Foundation, Inc. Prokaryotic RNAi-like system and methods of use
JP5908725B2 (ja) 2008-08-22 2016-04-26 サンガモ バイオサイエンシーズ， インコーポレイテッド 標的一本鎖開裂および標的組込みのための方法、並びに組成物
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
WO2010054108A2 (en) 2008-11-06 2010-05-14 University Of Georgia Research Foundation, Inc. Cas6 polypeptides and methods of use
EP2526199A4 (en) 2010-01-22 2013-08-07 Scripps Research Inst PROCESS FOR PRODUCING ZINC FINGERUCLEASES WITH CHANGED EFFECT
EP3202903B1 (en) 2010-12-22 2020-02-12 President and Fellows of Harvard College Continuous directed evolution
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
WO2013188638A2 (en) 2012-06-15 2013-12-19 The Regents Of The University Of California Endoribonucleases and methods of use thereof
CN116622704A (zh) 2012-07-25 2023-08-22 布罗德研究所有限公司 可诱导的dna结合蛋白和基因组干扰工具及其应用
HK1207111A1 (en) 2012-08-03 2016-01-22 加利福尼亚大学董事会 Methods and compositions for controlling gene expression by rna processing
EP4397759A3 (en) 2012-10-23 2024-10-09 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
US20160010154A1 (en) 2012-11-30 2016-01-14 The Parkinson's Institute Screening assays for therapeutics for parkinson's disease
DK3363902T3 (da) 2012-12-06 2020-01-02 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
EP3064585B1 (en) 2012-12-12 2020-02-05 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
PT2931898E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
MX380562B (es) 2012-12-12 2025-03-12 Broad Inst Inc Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias.
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN105209621B (zh) 2012-12-12 2021-05-25 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
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
KR102955556B1 (ko) 2012-12-12 2026-04-22 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
IL308158A (en) 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
WO2014113493A1 (en) 2013-01-16 2014-07-24 Emory University Cas9-nucleic acid complexes and uses related thereto
ES2749624T3 (es) 2013-03-14 2020-03-23 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
CN105408483A (zh) 2013-03-15 2016-03-16 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
EP4286517A3 (en) 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
EP2997146A4 (en) 2013-05-15 2017-04-26 Sangamo BioSciences, Inc. Methods and compositions for treatment of a genetic condition
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
JP6608807B2 (ja) 2013-05-29 2019-11-20 セレクティス Ｒｎａガイドｃａｓヌクレアーゼ系を用いることによって免疫療法のためにｔ細胞を操作するための方法
DK3004349T3 (en) 2013-05-29 2018-06-06 Cellectis Sa A method for producing precise DNA cleavage using CAS9 nickase activity
EP3004339B1 (en) 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
EP3603679B1 (en) 2013-06-04 2022-08-10 President and Fellows of Harvard College Rna-guided transcriptional regulation
WO2014197748A2 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
ES2777217T3 (es) 2013-06-17 2020-08-04 Broad Inst Inc Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
SG11201510327TA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
KR20260047646A (ko) 2013-07-09 2026-04-08 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 멀티플렉스 rna-가이드된 게놈 조작
CN105517579B (zh) 2013-07-10 2019-11-15 哈佛大学校长及研究员协会 用于RNA向导的基因调节和编辑的正交Cas9蛋白
EP3022304B1 (en) 2013-07-19 2018-12-26 Larix Biosciences LLC Methods and compositions for producing double allele knock outs
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
US20150044772A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
KR20160097338A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도
CA2932439A1 (en) 2013-12-12 2015-06-18 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
CA2932948A1 (en) 2013-12-13 2015-06-18 Cellectis New method of selection of algal-transformed cells using nuclease
EP3089989B1 (en) 2013-12-31 2020-06-24 The Regents of The University of California Cas9 crystals and methods of use thereof
SG11201608701QA (en) 2014-04-18 2016-11-29 Editas Medicine Inc Crispr-cas-related methods, compositions and components for cancer immunotherapy
US9970001B2 (en) 2014-06-05 2018-05-15 Sangamo Therapeutics, Inc. Methods and compositions for nuclease design
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3180426B1 (en) 2014-08-17 2019-12-25 The Broad Institute, Inc. Genome editing using cas9 nickases
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
EP3221457B1 (en) 2014-11-21 2019-03-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
CN107208070B (zh) 2014-11-26 2021-09-07 技术创新动力基金（以色列）有限合伙公司 细菌基因的靶向消除
CN107667171A (zh) * 2014-12-16 2018-02-06 丹尼斯科美国公司 真菌基因组修饰系统及使用方法
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
WO2016112242A1 (en) 2015-01-08 2016-07-14 President And Fellows Of Harvard College Split cas9 proteins
US11208638B2 (en) 2015-01-12 2021-12-28 The Regents Of The University Of California Heterodimeric Cas9 and methods of use thereof
AU2016211517B2 (en) 2015-01-28 2021-05-13 Caribou Biosciences, Inc. CRISPR hybrid DNA/RNA polynucleotides and methods of use
AU2016226077B2 (en) 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
EP4600366A3 (en) 2015-03-31 2025-10-22 SOHM, Inc. Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell
WO2016164797A1 (en) 2015-04-08 2016-10-13 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Activatable crispr/cas9 for spatial and temporal control of genome editing
EP3284749B1 (en) 2015-04-13 2024-08-14 The University of Tokyo Set of polypeptides exhibiting nuclease activity or nickase activity with dependence on light or in presence of drug or suppressing or activating expression of target gene
GB201506509D0 (en) 2015-04-16 2015-06-03 Univ Wageningen Nuclease-mediated genome editing
CN106011104B (zh) 2015-05-21 2019-09-27 清华大学 利用拆分Cas系统进行基因编辑和表达调控方法
US10392607B2 (en) 2015-06-03 2019-08-27 The Regents Of The University Of California Cas9 variants and methods of use thereof
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
US10648020B2 (en) 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
WO2017015015A1 (en) 2015-07-17 2017-01-26 Emory University Crispr-associated protein from francisella and uses related thereto
US10612011B2 (en) 2015-07-30 2020-04-07 President And Fellows Of Harvard College Evolution of TALENs
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US11008555B2 (en) 2015-09-17 2021-05-18 The Regents Of The University Of California Variant Cas9 polypeptides comprising internal insertions
WO2017064546A1 (en) 2015-09-24 2017-04-20 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
CA3001130A1 (en) 2015-10-16 2017-04-20 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions utilizing cpf1 for rna-guided gene editing
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
WO2017070632A2 (en) * 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
US11566052B2 (en) 2015-11-11 2023-01-31 Lonza Ltd. CRISPR-associated (Cas) proteins with reduced immunogenicity
WO2017099494A1 (ko) 2015-12-08 2017-06-15 기초과학연구원 Cpf1을 포함하는 유전체 교정용 조성물 및 그 용도
US20190264186A1 (en) 2016-01-22 2019-08-29 The Broad Institute Inc. Crystal structure of crispr cpf1
WO2017136335A1 (en) 2016-02-01 2017-08-10 The Regents Of The University Of California Self-inactivating endonuclease-encoding nucleic acids and methods of using the same
US9896696B2 (en) 2016-02-15 2018-02-20 Benson Hill Biosystems, Inc. Compositions and methods for modifying genomes
US20200291370A1 (en) 2016-03-18 2020-09-17 President And Fellows Of Harvard College Mutant Cas Proteins
WO2017181107A2 (en) 2016-04-16 2017-10-19 Ohio State Innovation Foundation Modified cpf1 mrna, modified guide rna, and uses thereof
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
CA3026110A1 (en) 2016-04-19 2017-11-02 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2017196768A1 (en) 2016-05-09 2017-11-16 President And Fellows Of Harvard College Self-targeting guide rnas in crispr system
WO2017197238A1 (en) 2016-05-12 2017-11-16 President And Fellows Of Harvard College Aav split cas9 genome editing and transcriptional regulation
EP3472311A4 (en) 2016-06-17 2020-03-04 Montana State University BIDIRECTIONAL TARGETING FOR GENOMEDITATION
AU2017283713B2 (en) 2016-06-17 2021-04-08 Massachusetts Institute Of Technology Type VI CRISPR orthologs and systems
US20190100745A1 (en) 2016-06-20 2019-04-04 Pioneer Hi-Bred International, Inc. Novel cas systems and methods of use
US10927383B2 (en) 2016-06-30 2021-02-23 Ethris Gmbh Cas9 mRNAs
AU2017302551B2 (en) 2016-07-26 2023-04-27 The General Hospital Corporation Variants of CRISPR from Prevotella and Francisella 1 (Cpf1)
US11352647B2 (en) 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
WO2018035250A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018049075A1 (en) 2016-09-07 2018-03-15 Flagship Pioneering, Inc. Methods and compositions for modulating gene expression
WO2018052247A1 (ko) 2016-09-13 2018-03-22 주식회사 툴젠 시토신 디아미나제에 의한 dna에서의 염기 교정 확인 방법
US12188059B2 (en) 2016-09-13 2025-01-07 The Broad Institute, Inc. Proximity-dependent biotinylation and uses thereof
CA3038982A1 (en) 2016-09-30 2018-04-05 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
JP2019532644A (ja) 2016-09-30 2019-11-14 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Ｒｎａ誘導型核酸修飾酵素及びその使用方法
WO2018068053A2 (en) 2016-10-07 2018-04-12 Integrated Dna Technologies, Inc. S. pyogenes cas9 mutant genes and polypeptides encoded by same
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
IT201600102542A1 (it) 2016-10-12 2018-04-12 Univ Degli Studi Di Trento Plasmide e sistema lentivirale contenente un circuito autolimitante della Cas9 che ne incrementa la sicurezza.
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
KR102662249B1 (ko) 2016-10-14 2024-05-03 더 제너럴 하스피탈 코포레이션 후성적으로 조절되는 부위-특이적 뉴클레아제
WO2018074979A1 (en) 2016-10-17 2018-04-26 Nanyang Technological University Truncated crispr-cas proteins for dna targeting
US11180778B2 (en) 2016-11-11 2021-11-23 The Regents Of The University Of California Variant RNA-guided polypeptides and methods of use
CA3044101A1 (en) 2016-11-22 2018-05-31 Integrated Dna Technologies, Inc. Crispr/cpf1 systems and methods
US11674128B2 (en) 2016-12-12 2023-06-13 Tsinghua University Engineering of a minimal SaCas9 CRISPR/Cas system for gene editing and transcriptional regulation optimized by enhanced guide RNA
WO2018209712A1 (en) 2017-05-19 2018-11-22 Tsinghua University Engineering of a minimal sacas9 crispr/cas system for gene editing and transcriptional regulation optimized by enhanced guide rna
US11242513B2 (en) 2016-12-14 2022-02-08 Wageningen Universiteit Thermostable Cas9 nucleases
WO2018112451A1 (en) 2016-12-16 2018-06-21 The Rockefeller University Compositions and methods using cas9 with enhanced spacer acquisition function
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
WO2018118968A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Emulsion-based screening methods
WO2018129368A2 (en) 2017-01-06 2018-07-12 Editas Medicine, Inc. Methods of assessing nuclease cleavage
IT201700016321A1 (it) * 2017-02-14 2018-08-14 Univ Degli Studi Di Trento Mutanti di cas9 ad alta specificita' e loro applicazioni.
US11111492B2 (en) 2017-03-06 2021-09-07 Florida State University Research Foundation, Inc. Genome engineering methods using a cytosine-specific Cas9
GB2574561A (en) 2017-03-14 2019-12-11 Univ California Engineering CRISPR CAS9 immune stealth
MX2019011215A (es) 2017-03-24 2020-02-12 Curevac Ag Ácidos nucleicos que codifican proteínas asociadas a crispr y usos de los mismos.
WO2018183766A1 (en) 2017-03-29 2018-10-04 Editas Medicine, Inc. Selection methods
CN108690845B (zh) 2017-04-10 2021-04-27 中国科学院动物研究所 基因组编辑系统和方法
US20180362590A1 (en) 2017-04-14 2018-12-20 Synthetic Genomics, Inc. Polypeptides with type v crispr activity and uses thereof
WO2018195545A2 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
EP3612204A4 (en) 2017-04-21 2021-01-27 The General Hospital Corporation INDUCTIBLE, TUNING AND MULTIPLEX HUMAN GENE REGULATION USING CRISPR-CPF1
WO2018197495A1 (en) 2017-04-24 2018-11-01 Dupont Nutrition Biosciences Aps Novel anti-crispr genes and proteins and methods of use
US11692184B2 (en) 2017-05-16 2023-07-04 The Regents Of The University Of California Thermostable RNA-guided endonucleases and methods of use thereof
US11371030B2 (en) 2017-05-31 2022-06-28 The University Of Tokyo Modified Cas9 protein and use thereof
US20200140835A1 (en) 2017-06-06 2020-05-07 The General Hospital Corporation Engineered CRISPR-Cas9 Nucleases
CN110997908A (zh) 2017-06-09 2020-04-10 爱迪塔斯医药公司 工程化的cas9核酸酶
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
US11441135B2 (en) 2017-07-07 2022-09-13 Toolgen Incorporated Target-specific CRISPR mutant
WO2019018041A1 (en) 2017-07-21 2019-01-24 Arizona Board Of Regents On Behalf Of Arizona State University MODULATION OF THE IMMUNE RESPONSE OF A HOST SPECIFIC TO HUMAN CAS9
JP2020532961A (ja) 2017-08-15 2020-11-19 インテリア セラピューティクス，インコーポレイテッド メッセンジャーリボ核酸（ｍＲＮＡ）をコードする安定化された核酸
WO2019040650A1 (en) 2017-08-23 2019-02-28 The General Hospital Corporation GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY
EP3676378A4 (en) 2017-08-31 2021-06-09 The New York Genome Center PROCEDURES AND COMPOSITIONS USING CRIMP-CPF1 AND PAIRED GUIDE-CRISPR-RNA FOR PROGRAMMABLE GENOMIC DELETIONS
JP6628387B2 (ja) 2017-09-05 2020-01-08 国立大学法人 東京大学 改変されたＣａｓ９タンパク質及びその用途
WO2019051419A1 (en) 2017-09-08 2019-03-14 University Of North Texas Health Science Center Engineered cas9 variants
WO2019060469A2 (en) 2017-09-19 2019-03-28 Massachusetts Institute Of Technology CAS9 STREPTOCOCCUS CANIS AS A GENOMIC ENGINEERING PLATFORM WITH NEW PAM SPECIFICITY
CA3111479A1 (en) 2017-09-26 2019-04-04 The Board Of Trustees Of The University Of Illinois Crispr/cas system and method for genome editing and modulating transcription
GB201716590D0 (en) 2017-10-10 2017-11-22 Univ Wageningen Thermostable cas9 nucleases with reduced off-target activity
WO2019083532A1 (en) 2017-10-26 2019-05-02 Excision Biotherapeutics Inc IDENTIFICATION OF GENE EDITION PROTEINS AND GENE EXCISION (TYPE CAS9 AND ARGONAUTE) FROM OTHER ORGANISMS
EP3704254A4 (en) 2017-11-01 2021-09-01 The Regents of The University of California CAS12C COMPOSITIONS AND METHOD OF USE
WO2019089808A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Class 2 crispr/cas compositions and methods of use
CN117487776A (zh) 2017-11-01 2024-02-02 加利福尼亚大学董事会 Casz组合物和使用方法
WO2019089804A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Casy compositions and methods of use
US20200255829A1 (en) 2017-11-02 2020-08-13 Arbor Biotechnologies, Inc. Novel crispr-associated transposon systems and components
MA50579A (fr) 2017-11-09 2020-09-16 Crispr Therapeutics Ag Systèmes crispr/cas ou crispr/cpf1 à auto-inactivation (sin) et leurs utilisations
EP3707252A1 (en) 2017-11-10 2020-09-16 Novozymes A/S Temperature-sensitive cas9 protein
JP7423520B2 (ja) 2017-11-16 2024-01-29 アストラゼネカ・アクチエボラーグ Ｃａｓ９ベースノックイン方針の効力を改善するための組成物及び方法
US20230193242A1 (en) 2017-12-22 2023-06-22 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
WO2019126762A2 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
EP3728576A4 (en) 2017-12-22 2021-11-24 The Broad Institute, Inc. CAS12B SYSTEMS, METHODS AND COMPOSITIONS FOR SPECIFIC EDITING OF RNA BASES
WO2019126774A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2019168953A1 (en) 2018-02-27 2019-09-06 President And Fellows Of Harvard College Evolved cas9 variants and uses thereof
DK3765616T3 (da) 2018-03-14 2023-08-21 Arbor Biotechnologies Inc Nye enzymer og systemer målrettet crispr dna og rna
PL3765615T3 (pl) 2018-03-14 2023-11-13 Arbor Biotechnologies, Inc. Nowe enzymy i układy crispr ukierunkowane na dna
JP7550648B2 (ja) 2018-03-19 2024-09-13 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016205613A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzyme mutations reducing off-target effects
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Science，2016年，Vol. 351, Issue 6268，pp. 84-88

Also Published As

Publication number Publication date
EP3635104A1 (en) 2020-04-15
KR20200016892A (ko) 2020-02-17
JP2020524497A (ja) 2020-08-20
WO2018227114A1 (en) 2018-12-13
CN110997908A (zh) 2020-04-10
AU2018279829A1 (en) 2020-01-02
CA3065813A1 (en) 2018-12-13
US10428319B2 (en) 2019-10-01
JP2024001075A (ja) 2024-01-09
US11098297B2 (en) 2021-08-24
US20180355332A1 (en) 2018-12-13
US20200056164A1 (en) 2020-02-20
US20220177861A1 (en) 2022-06-09
US12297466B2 (en) 2025-05-13
JP7695315B2 (ja) 2025-06-18
AU2018279829B2 (en) 2024-01-04
MX2019014640A (es) 2020-10-05
KR102746733B1 (ko) 2024-12-24

Similar Documents

Publication Publication Date Title
JP7695315B2 (ja) 操作されたｃａｓ９ヌクレアーゼ
AU2021236446B2 (en) CRISPR-Cas-related methods, compositions and components for cancer immunotherapy
US12606834B2 (en) Methods and compositions for efficient delivery of nucleic acids and rna-based antimicrobials
US20230018543A1 (en) Crispr/cas-mediated gene conversion
US12359223B2 (en) Systems and methods for modulating chromosomal rearrangements
US12264331B2 (en) Systems and methods for one-shot guide RNA (ogRNA) targeting of endogenous and source DNA
EP4047092B1 (en) Cas9 fusion molecules, gene editing systems, and methods of use thereof
EP3443088B1 (en) Grna fusion molecules, gene editing systems, and methods of use thereof
EP3981876A1 (en) Crispr/cas-related methods and compositions for treating sickle cell disease
CA3040030A1 (en) Self-limiting cas9 circuitry for enhanced safety (slices) plasmid and lentiviral system thereof
WO2019090175A1 (en) Novel crispr-associated transposon systems and components
US20210047633A1 (en) Selection methods
US20240417707A1 (en) Compositions and methods for improved genome editing with nme2cas9 and nme2-smucas9 variants
WO2025029727A2 (en) Compositions, methods, and systems for dna modification
HK1254074B (en) Methods and compositions for efficient delivery of nucleic acids and rna-based antimicrobials

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210604

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210604

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220627

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220829

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20221221

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230309

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20230529

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230927

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231002

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20231023

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231222

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240314

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240527

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: 20240610

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20240705

R150 Certificate of patent or registration of utility model

Ref document number: 7518620

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150