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

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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
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polypeptide
cas9
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selection
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JP2020524497A (ja
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バレット イーサン スタインバーグ
デレク セルチオーネ
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エディタス・メディシン，インコーポレイテッド
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    • 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

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (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)

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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)

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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

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JP2020524497A JP2020524497A (ja) 2020-08-20
JP2020524497A5 JP2020524497A5 (https=) 2021-07-26
JP7518620B2 true JP7518620B2 (ja) 2024-07-18

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

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3004370B1 (en) * 2013-06-05 2024-08-21 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
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2018119010A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Assessing nuclease cleavage
EP3565907B1 (en) 2017-01-06 2022-05-04 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
WO2018227114A1 (en) 2017-06-09 2018-12-13 Editas Medicine, Inc. Engineered cas9 nucleases
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
MA51186A (fr) 2017-12-14 2020-10-21 Bayer Healthcare Llc Matériels et procédés pour traiter la dystrophie des cônes et des bâtonnets autosomique dominante
WO2019144061A1 (en) 2018-01-19 2019-07-25 Duke University Genome engineering with crispr-cas systems in eukaryotes
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED 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
AU2020311438A1 (en) * 2019-07-11 2021-12-16 Arbor Biotechnologies, Inc. Novel CRISPR DNA targeting enzymes and systems
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
KR20230152689A (ko) * 2021-02-01 2023-11-03 엡실렌 바이오 에스.알.엘. 유전자 침묵
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 (182)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith 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
ES2398918T3 (es) 2005-08-26 2013-03-22 Dupont Nutrition Biosciences Aps Un método y una ordenación para soportar verticalmente elementos de resistencia eléctrica pendientes
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
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
WO2011091324A2 (en) 2010-01-22 2011-07-28 The Scripps Research Institute Methods of generating zinc finger nucleases having altered activity
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
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
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
WO2013188638A2 (en) 2012-06-15 2013-12-19 The Regents Of The University Of California Endoribonucleases and methods of use thereof
KR102530118B1 (ko) 2012-07-25 2023-05-08 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
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
EP4357457B1 (en) 2012-10-23 2024-10-16 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
WO2014085830A2 (en) 2012-11-30 2014-06-05 The Parkinson's Institute Screening assays for therapeutics for parkinson's disease
EP3363902B1 (en) 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014093718A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
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
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
DK2931897T3 (en) 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS
AU2013359262C1 (en) 2012-12-12 2021-05-13 Massachusetts Institute Of Technology CRISPR-Cas component systems, methods and compositions for sequence manipulation
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
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
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
CA2895155C (en) 2012-12-17 2021-07-06 President And Fellows Of Harvard College Rna-guided human genome engineering
EP3919505B1 (en) 2013-01-16 2023-08-30 Emory University Uses of cas9-nucleic acid complexes
MX374090B (es) 2013-03-14 2025-03-05 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos al ácido nucleico.
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
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
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
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
JP2016522679A (ja) 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
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
US11414695B2 (en) 2013-05-29 2022-08-16 Agilent Technologies, Inc. Nucleic acid enrichment using Cas9
US20160122774A1 (en) 2013-05-29 2016-05-05 Cellectis A method for producing precise dna cleavage using cas9 nickase activity
ES2883131T3 (es) 2013-05-29 2021-12-07 Cellectis Métodos para la modificación de células T para inmunoterapia utilizando el sistema de nucleasa CAS guiado por ARN
US11685935B2 (en) 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
EP4596565A3 (en) 2013-06-04 2025-11-05 President And Fellows Of Harvard College Rna-guided transcriptional regulation
EP3004370B1 (en) 2013-06-05 2024-08-21 Duke University Rna-guided gene editing and gene regulation
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
KR20160019553A (ko) 2013-06-17 2016-02-19 더 브로드 인스티튜트, 인코퍼레이티드 유사분열 후 세포의 질병 및 장애를 표적화하고 모델링하기 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3011030B1 (en) 2013-06-17 2023-11-08 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
JP7120717B2 (ja) 2013-07-09 2022-08-17 プレジデント アンド フェローズ オブ ハーバード カレッジ 多重ｒｎａ誘導型ゲノム編集
WO2015006294A2 (en) 2013-07-10 2015-01-15 President And Fellows Of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US9663782B2 (en) 2013-07-19 2017-05-30 Larix Bioscience Llc Methods and compositions for producing double allele knock outs
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
WO2015021426A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase 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
AU2014361834B2 (en) 2013-12-12 2020-10-22 Massachusetts Institute Of Technology CRISPR-Cas systems and methods for altering expression of gene products, structural information and inducible modular Cas enzymes
MX2016007325A (es) 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
EP3080275B1 (en) 2013-12-13 2020-01-15 Cellectis Method of selection of transformed diatoms using nuclease
US9963689B2 (en) 2013-12-31 2018-05-08 The Regents Of The University Of California Cas9 crystals and methods of use thereof
CA2945335A1 (en) 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
EP3152319A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3686279B1 (en) 2014-08-17 2023-01-04 The Broad Institute, Inc. Genome editing using cas9 nickases
CA2963820A1 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
ES2731437T3 (es) 2014-11-21 2019-11-15 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida mediante el uso de pares de ARN guías
WO2016084088A1 (en) 2014-11-26 2016-06-02 Ramot At Tel-Aviv University Ltd. Targeted elimination of bacterial genes
CA2971187C (en) 2014-12-16 2023-10-24 Danisco Us Inc. Fungal genome modification systems and methods of use
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
WO2016114972A1 (en) 2015-01-12 2016-07-21 The Regents Of The University Of California Heterodimeric cas9 and methods of use thereof
EP3250691B9 (en) 2015-01-28 2023-08-02 Caribou Biosciences, Inc. Crispr hybrid dna/rna polynucleotides and methods of use
EP3265559B1 (en) 2015-03-03 2021-01-06 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
KR20250171479A (ko) 2015-03-31 2025-12-08 소흠, 인코포레이티드 세포 또는 유기체의 게놈으로의 ＤＮＡ 서열의 표적화 혼입을 위한 Ｃａｓ 9 레트로바이러스 인테그라제 시스템 및 Ｃａｓ 9 재조합효소 시스템
US11214779B2 (en) 2015-04-08 2022-01-04 University of Pittsburgh—of the Commonwealth System of Higher Education Activatable CRISPR/CAS9 for spatial and temporal control of genome editing
JP6892642B2 (ja) 2015-04-13 2021-06-23 国立大学法人 東京大学 光依存的に又は薬物存在下でヌクレアーゼ活性若しくはニッカーゼ活性を示す、又は標的遺伝子の発現を抑制若しくは活性化するポリペプチドのセット
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
EP3303634B1 (en) 2015-06-03 2023-08-30 The Regents of The University of California Cas9 variants and methods of use thereof
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
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
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
CA3012607A1 (en) 2015-06-18 2016-12-22 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
WO2017048969A1 (en) 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
US20190048340A1 (en) 2015-09-24 2019-02-14 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
US20190083656A1 (en) 2015-10-16 2019-03-21 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions utilizing cpf1 for rna-guided gene editing
CN109153980B (zh) 2015-10-22 2023-04-14 布罗德研究所有限公司 Vi-b型crispr酶和系统
JP7109784B2 (ja) * 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
WO2017081288A1 (en) 2015-11-11 2017-05-18 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
WO2017127807A1 (en) 2016-01-22 2017-07-27 The Broad Institute Inc. Crystal structure of crispr cpf1
US11530421B2 (en) 2016-02-01 2022-12-20 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
US12065667B2 (en) 2016-04-16 2024-08-20 Ohio State Innovation Foundation Modified Cpf1 MRNA, modified guide RNA, and uses thereof
US20200263190A1 (en) 2016-04-19 2020-08-20 The Broad Institute, Inc. Novel crispr enzymes and systems
CN110382692A (zh) 2016-04-19 2019-10-25 博德研究所 新型crispr酶以及系统
US20190161743A1 (en) 2016-05-09 2019-05-30 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 GENOME EDITING
CA3028158A1 (en) 2016-06-17 2017-12-21 The Broad Institute, Inc. Type vi crispr orthologs and systems
CA3018430A1 (en) 2016-06-20 2017-12-28 Pioneer Hi-Bred International, Inc. Novel cas systems and methods of use
US10927383B2 (en) 2016-06-30 2021-02-23 Ethris Gmbh Cas9 mRNAs
WO2018022634A1 (en) 2016-07-26 2018-02-01 The General Hospital Corporation Variants of crispr from prevotella and francisella 1 (cpf1)
US12431216B2 (en) 2016-08-17 2025-09-30 Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
CN110312799A (zh) 2016-08-17 2019-10-08 博德研究所 新型crispr酶和系统
WO2018035388A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
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
WO2018053053A1 (en) 2016-09-13 2018-03-22 The Broad Institute, Inc. Proximity-dependent biotinylation and uses thereof
WO2018052247A1 (ko) 2016-09-13 2018-03-22 주식회사 툴젠 시토신 디아미나제에 의한 dna에서의 염기 교정 확인 방법
CA3038960A1 (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
JP7306696B2 (ja) 2016-09-30 2023-07-11 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
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
CN118726313A (zh) 2016-10-07 2024-10-01 综合Dna技术公司 化脓链球菌cas9突变基因和由其编码的多肽
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.
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
AU2017341926B2 (en) 2016-10-14 2022-06-30 The General Hospital Corporation Epigenetically regulated site-specific nucleases
SG10201913505WA (en) 2016-10-17 2020-02-27 Univ Nanyang Tech Truncated crispr-cas proteins for dna targeting
WO2018089664A1 (en) 2016-11-11 2018-05-17 The Regents Of The University Of California Variant rna-guided polypeptides and methods of use
WO2018098383A1 (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
JP7182545B2 (ja) 2016-12-14 2022-12-02 ヴァーヘニンゲン ユニヴェルシテット 熱安定性ｃａｓ９ヌクレアーゼ
WO2018109101A1 (en) 2016-12-14 2018-06-21 Wageningen Universiteit Thermostable cas9 nucleases
US11377645B2 (en) 2016-12-16 2022-07-05 The Rockefeller University Compositions and methods using Cas9 with enhanced spacer acquisition function
US20200087658A1 (en) 2016-12-19 2020-03-19 Editas Medicine, Inc. Emulsion-based screening methods
WO2018119010A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Assessing nuclease cleavage
EP3565907B1 (en) 2017-01-06 2022-05-04 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
EP3595694A4 (en) 2017-03-14 2021-06-09 The Regents of The University of California Engineering crispr cas9 immune stealth
AU2018240515B2 (en) 2017-03-24 2024-07-25 CureVac SE Nucleic acids encoding CRISPR-associated proteins and uses thereof
US20210047633A1 (en) 2017-03-29 2021-02-18 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
WO2018191715A2 (en) 2017-04-14 2018-10-18 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
CN110799205A (zh) 2017-04-21 2020-02-14 通用医疗公司 利用CRISPR-Cpf1的可诱导、可调和多重的人类基因调节
EP3615665B1 (en) 2017-04-24 2025-11-26 International N&H Denmark 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
EP3625338A4 (en) 2017-05-19 2021-01-20 Tsinghua University CONSTRUCTION OF A MINIMAL SACAS9-CRISPR / CAS SYSTEM FOR GENEDITATION AND TRANSCRIPTION REGULATION, OPTIMIZED BY EXTENDED GUIDE-RNA
JP6628385B6 (ja) 2017-05-31 2020-03-04 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
WO2018226855A1 (en) 2017-06-06 2018-12-13 The General Hospital Corporation Engineered crispr-cas9 nucleases
WO2018227114A1 (en) 2017-06-09 2018-12-13 Editas Medicine, Inc. Engineered cas9 nucleases
US11168322B2 (en) 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
CA3069296A1 (en) 2017-07-07 2019-01-10 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
EP3668980A1 (en) 2017-08-15 2020-06-24 Intellia Therapeutics, Inc. Stabilized nucleic acids encoding messenger ribonucleic acid (mrna)
CN118530967A (zh) 2017-08-23 2024-08-23 通用医疗公司 具有改变的PAM特异性的工程化的CRISPR-Cas9核酸酶
WO2019046540A1 (en) 2017-08-31 2019-03-07 New York Genome Center, Inc. METHODS AND COMPOSITIONS COMPRISING CRISPR-CPF1 AND APPROVED GUIDING CRISPR RNAES 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
US11713452B2 (en) 2017-09-08 2023-08-01 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
CN111373041B (zh) 2017-09-26 2024-10-11 伊利诺伊大学理事会 用于基因组编辑和调节转录的crispr/cas系统和方法
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
WO2019089804A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Casy compositions and methods 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
US20200339967A1 (en) 2017-11-01 2020-10-29 The Regents Of The University Of California Cas12c compositions and methods of use
JP2021503278A (ja) 2017-11-01 2021-02-12 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 ＣａｓＺ組成物及び使用方法
WO2019090175A1 (en) 2017-11-02 2019-05-09 Arbor Biotechnologies, Inc. Novel crispr-associated transposon systems and components
US20210363521A1 (en) 2017-11-09 2021-11-25 Vertex Pharmaceuticals Incorporated CRISPR/CAS Systems For Treatment of DMD
EP3707252A1 (en) 2017-11-10 2020-09-16 Novozymes A/S Temperature-sensitive cas9 protein
CN111448313A (zh) 2017-11-16 2020-07-24 阿斯利康(瑞典)有限公司 用于改善基于Cas9的敲入策略的有效性的组合物和方法
WO2019126709A1 (en) 2017-12-22 2019-06-27 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
WO2019126774A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Novel crispr enzymes and systems
EP3728576A4 (en) 2017-12-22 2021-11-24 The Broad Institute, Inc. CAS12B SYSTEMS, METHODS AND COMPOSITIONS FOR SPECIFIC EDITING OF RNA BASES
US20220307001A1 (en) 2018-02-27 2022-09-29 President And Fellows Of Harvard College Evolved cas9 variants and uses thereof
EP3765616B1 (en) 2018-03-14 2023-07-19 Arbor Biotechnologies, Inc. Novel crispr dna and rna targeting enzymes and systems
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

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