KR102746733B1 - 조작된 cas9 뉴클레아제 - Google Patents

조작된 cas9 뉴클레아제 Download PDF

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KR102746733B1
KR102746733B1 KR1020197038409A KR20197038409A KR102746733B1 KR 102746733 B1 KR102746733 B1 KR 102746733B1 KR 1020197038409 A KR1020197038409 A KR 1020197038409A KR 20197038409 A KR20197038409 A KR 20197038409A KR 102746733 B1 KR102746733 B1 KR 102746733B1
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바렛 이탄 스타인버그
데렉 썰치온
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에디타스 메디신, 인코포레이티드
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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • 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)
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KR1020197038409A 2017-06-09 2018-06-08 조작된 cas9 뉴클레아제 Active KR102746733B1 (ko)

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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

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KR20200016892A KR20200016892A (ko) 2020-02-17
KR102746733B1 true KR102746733B1 (ko) 2024-12-24

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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
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
AU2015330699B2 (en) 2014-10-10 2021-12-02 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
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
MX2019014640A (es) 2017-06-09 2020-10-05 Editas Medicine Inc Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas.
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
CA3084733A1 (en) 2017-12-14 2019-06-20 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant cone-rod dystrophy
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
EP3921638A4 (en) 2019-02-05 2022-08-24 AMMR Joint Venture EXPANDED CHOICE OF EFFICIENT TARGETED GENE MANIPULATORS
EP3996738A4 (en) * 2019-07-11 2023-07-26 Arbor Biotechnologies, Inc. NOVEL ENZYMES AND SYSTEMS TARGETING CRISPR DNA
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
CA3207166A1 (en) * 2021-02-01 2022-08-04 Angelo Leone Lombardo Gene silencing
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
KR20240109932A (ko) * 2023-01-04 2024-07-12 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
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
KR102350405B1 (ko) 2014-12-16 2022-01-11 다니스코 유에스 인크. 진균 게놈 변형 시스템 및 사용 방법

Family Cites Families (181)

* 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
DK2341149T3 (en) 2005-08-26 2017-02-27 Dupont Nutrition Biosci Aps Use of CRISPR-associated genes (Cas)
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
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
EP2526199A4 (en) 2010-01-22 2013-08-07 Scripps Research Inst PROCESS FOR PRODUCING ZINC FINGERUCLEASES WITH CHANGED EFFECT
CA2825370A1 (en) 2010-12-22 2012-06-28 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
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
WO2013188638A2 (en) 2012-06-15 2013-12-19 The Regents Of The University Of California Endoribonucleases and methods of use thereof
AU2013293270B2 (en) 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
WO2014022702A2 (en) 2012-08-03 2014-02-06 The Regents Of The University Of California Methods and compositions for controlling gene expression by rna processing
AU2013335451C1 (en) 2012-10-23 2024-07-04 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
PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
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
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
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
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
CA2898184A1 (en) 2013-01-16 2014-07-24 Emory University Cas9-nucleic acid complexes and uses related thereto
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
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
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
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
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
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
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
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
AU2014273082B2 (en) 2013-05-29 2018-11-08 Cellectis A method for producing precise DNA cleavage using Cas9 nickase activity
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
EP3004339B1 (en) 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
SG10201913068PA (en) 2013-06-04 2020-02-27 Harvard College Rna-guided transcriptional regulation
US9267135B2 (en) 2013-06-04 2016-02-23 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
CN105683379A (zh) 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
MX374532B (es) 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
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
ES2929143T3 (es) 2013-07-09 2022-11-25 Harvard College Ingeniería genómica guiada por ARN múltiplex
EP3666892A1 (en) 2013-07-10 2020-06-17 President and Fellows of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
JP6482546B2 (ja) 2013-07-19 2019-03-13 ラリクス・バイオサイエンス・リミテッド・ライアビリティ・カンパニーＬａｒｉｘ Ｂｉｏｓｃｉｅｎｃｅ， Ｌｌｃ 二重対立遺伝子ノックアウトを生成するための方法および組成物
US11306328B2 (en) 2013-07-26 2022-04-19 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
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
WO2015089427A1 (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
JP6712948B2 (ja) 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法
WO2015086798A2 (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
WO2015161276A2 (en) 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
WO2015188056A1 (en) 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
EP4464338A3 (en) 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
KR102531016B1 (ko) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물
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
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
WO2016112242A1 (en) 2015-01-08 2016-07-14 President And Fellows Of Harvard College Split cas9 proteins
EP3245232B1 (en) 2015-01-12 2021-04-21 The Regents of The University of California Heterodimeric cas9 and methods of use thereof
US9650617B2 (en) 2015-01-28 2017-05-16 Pioneer Hi-Bred International. Inc. CRISPR hybrid DNA/RNA polynucleotides and methods of use
KR20230156800A (ko) 2015-03-03 2023-11-14 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
JP2018513681A (ja) 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのＤＮＡ配列の標的化組み込みのためのＣａｓ９レトロウイルスインテグラーゼおよびＣａｓ９レコンビナーゼ系
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
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
WO2016196655A1 (en) 2015-06-03 2016-12-08 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
US10648020B2 (en) 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
US9790490B2 (en) 2015-06-18 2017-10-17 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
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
TWI906646B (zh) * 2015-06-18 2025-12-01 美商博得學院股份有限公司 降低脫靶效應的ｃｒｉｓｐｒ酶突變
WO2017015015A1 (en) 2015-07-17 2017-01-26 Emory University Crispr-associated protein from francisella and uses related thereto
WO2017019895A1 (en) 2015-07-30 2017-02-02 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
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
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
IL310721B2 (en) * 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
EP3374501B1 (en) 2015-11-11 2023-07-12 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
JP6914274B2 (ja) 2016-01-22 2021-08-04 ザ・ブロード・インスティテュート・インコーポレイテッド Ｃｒｉｓｐｒｃｐｆ１の結晶構造
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
AU2017257274B2 (en) 2016-04-19 2023-07-13 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
WO2017196768A1 (en) 2016-05-09 2017-11-16 President And Fellows Of Harvard College Self-targeting guide rnas in crispr system
US20190345483A1 (en) 2016-05-12 2019-11-14 President And Fellows Of Harvard College AAV Split Cas9 Genome Editing and Transcriptional Regulation
US20190323038A1 (en) 2016-06-17 2019-10-24 Montana State Univesity Bidirectional targeting for genome editing
CN109642231A (zh) 2016-06-17 2019-04-16 博德研究所 Vi型crispr直向同源物和系统
WO2017222773A1 (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
CN109790527A (zh) 2016-07-26 2019-05-21 通用医疗公司 普氏菌属和弗朗西斯氏菌属的CRISPR1(Cpf1)的变体
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
EP3500967A1 (en) 2016-08-17 2019-06-26 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
EP4485466A3 (en) 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
US12188059B2 (en) 2016-09-13 2025-01-07 The Broad Institute, Inc. Proximity-dependent biotinylation and uses thereof
EP3530737A4 (en) 2016-09-13 2020-04-29 Toolgen Incorporated METHOD FOR IDENTIFYING DNA BASE EDITING USING CYTOSINE DEAMINASE
MX2019003674A (es) 2016-09-30 2021-01-08 Univ California Enzimas modificadoras de ácido nucleico guiadas por arn y métodos de uso de estas.
BR112019006388A2 (pt) 2016-09-30 2019-06-25 Univ California enzimas modificadoras de ácido nucleico guiadas por rna e métodos de uso das mesmas
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
KR20260019012A (ko) 2016-10-07 2026-02-09 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Ｓ. 피오게네스 ｃａｓ９ 돌연변이 유전자 및 이에 의해 암호화되는 폴리펩티드
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.
KR20190067209A (ko) 2016-10-14 2019-06-14 더 제너럴 하스피탈 코포레이션 후성적으로 조절되는 부위-특이적 뉴클레아제
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
SG10201913505WA (en) 2016-10-17 2020-02-27 Univ Nanyang Tech Truncated crispr-cas proteins for dna targeting
EP3538561A4 (en) 2016-11-11 2020-10-21 The Regents of The University of California RNA-GUIDED VARIANT POLYPEPTIDES AND THEIR METHODS OF USE
KR20190082318A (ko) 2016-11-22 2019-07-09 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Crispr/cpf1 시스템 및 방법
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
BR112019012155A2 (pt) 2016-12-14 2019-11-12 Wageningen Universiteit uso de pelo menos uma molécula-guia de rna e uma proteína cas, método de ligação, clivagem, marcação ou modificação de um polinucleotídeo alvo de fita dupla, célula transformada, e, complexo de nucleoproteína
US11377645B2 (en) 2016-12-16 2022-07-05 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
US20200087658A1 (en) 2016-12-19 2020-03-19 Editas Medicine, Inc. Emulsion-based screening methods
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
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
WO2018170015A1 (en) 2017-03-14 2018-09-20 The Regents Of The University Of California Engineering crispr cas9 immune stealth
SG11201906297QA (en) 2017-03-24 2019-10-30 Curevac Ag Nucleic acids encoding crispr-associated proteins and uses thereof
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
WO2018191715A2 (en) 2017-04-14 2018-10-18 Synthetic Genomics, Inc. Polypeptides with type v crispr activity and uses thereof
US11591589B2 (en) 2017-04-21 2023-02-28 The General Hospital Corporation Variants of Cpf1 (Cas12a) with altered PAM specificity
WO2018195540A1 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Inducible, tunable, 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
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
WO2018221685A1 (ja) 2017-05-31 2018-12-06 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
US20200140835A1 (en) 2017-06-06 2020-05-07 The General Hospital Corporation Engineered CRISPR-Cas9 Nucleases
MX2019014640A (es) 2017-06-09 2020-10-05 Editas Medicine Inc Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas.
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
SG11201913852RA (en) 2017-07-07 2020-01-30 Toolgen Inc 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
CA3072960A1 (en) 2017-08-15 2019-02-21 Intellia Therapeutics, Inc. Stabilized nucleic acids encoding messenger ribonucleic acid (mrna)
CA3073448A1 (en) 2017-08-23 2019-02-28 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
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
EP3680331A4 (en) 2017-09-05 2021-06-09 The University Of Tokyo MODIFIED CAS9 PROTEIN AND USE OF IT
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
EP3688159A4 (en) 2017-09-26 2021-10-20 The Board of Trustees of the University of Illinois CRISPR / CAS SYSTEM AND PROCESS FOR GENOME EDITING AND TRANSCRIPT MODULATION
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
AU2018358051B2 (en) 2017-11-01 2025-01-09 The Regents Of The University Of California CasZ compositions and methods of use
US11970719B2 (en) 2017-11-01 2024-04-30 The Regents Of The University Of California Class 2 CRISPR/Cas compositions and methods of use
WO2019089796A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Cas12c compositions and methods of use
WO2019089804A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Casy compositions and methods of use
WO2019090175A1 (en) 2017-11-02 2019-05-09 Arbor Biotechnologies, Inc. Novel crispr-associated transposon systems and components
EP3707155A2 (en) 2017-11-09 2020-09-16 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
CN120310773A (zh) 2017-11-16 2025-07-15 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
EP3728576A4 (en) 2017-12-22 2021-11-24 The Broad Institute, Inc. CAS12B SYSTEMS, METHODS AND COMPOSITIONS FOR SPECIFIC EDITING OF RNA BASES
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
US20210079366A1 (en) 2017-12-22 2021-03-18 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
EP3727469A4 (en) 2017-12-22 2021-12-01 The Broad Institute, Inc. NEW CRISPR ENZYMES AND SYSTEMS
US20220307001A1 (en) 2018-02-27 2022-09-29 President And Fellows Of Harvard College Evolved cas9 variants and uses thereof
WO2019178427A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
WO2019178428A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna and rna targeting enzymes and systems
MA52074A (fr) 2018-03-19 2021-01-27 Bayer Healthcare Llc Nouveaux systèmes d'endonucléase à arn programmable et leurs utilisations

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102350405B1 (ko) 2014-12-16 2022-01-11 다니스코 유에스 인크. 진균 게놈 변형 시스템 및 사용 방법
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Nature. Vol. 529, No. 7587, 1-24(2016)
Science. Vol. 351, No. 6268, 84-88(2016)

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