AU2014281027A1 - Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation - Google Patents

Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation Download PDF

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Publication number
AU2014281027A1
AU2014281027A1 AU2014281027A AU2014281027A AU2014281027A1 AU 2014281027 A1 AU2014281027 A1 AU 2014281027A1 AU 2014281027 A AU2014281027 A AU 2014281027A AU 2014281027 A AU2014281027 A AU 2014281027A AU 2014281027 A1 AU2014281027 A1 AU 2014281027A1
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Prior art keywords
sequence
crispr
target
tracr
overhang
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Abandoned
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AU2014281027A
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Inventor
Patrick Hsu
Chie-yu LIN
Fei RAN
Feng Zhang
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Massachusetts Institute of Technology
Broad Institute Inc
Harvard University
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Massachusetts Institute of Technology
Broad Institute Inc
Harvard University
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Priority claimed from PCT/US2013/074667 external-priority patent/WO2014093622A2/en
Application filed by Massachusetts Institute of Technology, Broad Institute Inc, Harvard University filed Critical Massachusetts Institute of Technology
Publication of AU2014281027A1 publication Critical patent/AU2014281027A1/en
Assigned to MASSACHUSETTS INSTITUTE OF TECHNOLOGY, PRESIDENT AND FELLOWS OF HARVARD COLLEGE, THE BROAD INSTITUTE, INC. reassignment MASSACHUSETTS INSTITUTE OF TECHNOLOGY Amend patent request/document other than specification (104) Assignors: MASSACHUSETTS INSTITUTE OF TECHNOLOGY, PRESIDENT AND FELLOWS OF HARVARD COLLEGE, The Broad Institute Inc.
Abandoned legal-status Critical Current

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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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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]
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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]
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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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  • Animal Behavior & Ethology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
AU2014281027A 2013-06-17 2014-06-10 Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation Abandoned AU2014281027A1 (en)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201361836123P 2013-06-17 2013-06-17
US61/836,123 2013-06-17
US201361847537P 2013-07-17 2013-07-17
US61/847,537 2013-07-17
US201361862355P 2013-08-05 2013-08-05
US61/862,355 2013-08-05
US201361871301P 2013-08-28 2013-08-28
US61/871,301 2013-08-28
US201361915383P 2013-12-12 2013-12-12
PCT/US2013/074667 WO2014093622A2 (en) 2012-12-12 2013-12-12 Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
AUPCT/US2013/074667 2013-12-12
US61/915,383 2013-12-12
PCT/US2014/041803 WO2014204725A1 (en) 2013-06-17 2014-06-10 Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation

Publications (1)

Publication Number Publication Date
AU2014281027A1 true AU2014281027A1 (en) 2016-01-28

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AU2014281027A Abandoned AU2014281027A1 (en) 2013-06-17 2014-06-10 Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation

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US (3) US10711285B2 (cg-RX-API-DMAC7.html)
EP (2) EP4245853A3 (cg-RX-API-DMAC7.html)
JP (4) JP6665088B2 (cg-RX-API-DMAC7.html)
KR (1) KR20160034901A (cg-RX-API-DMAC7.html)
CN (1) CN105492611A (cg-RX-API-DMAC7.html)
AU (1) AU2014281027A1 (cg-RX-API-DMAC7.html)
CA (1) CA2915837A1 (cg-RX-API-DMAC7.html)
WO (1) WO2014204725A1 (cg-RX-API-DMAC7.html)

Families Citing this family (349)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
CA2850509C (en) 2011-10-14 2023-08-01 President And Fellows Of Harvard College Sequencing by structure assembly
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
CN110643600A (zh) * 2012-10-23 2020-01-03 基因工具股份有限公司 用于切割靶dna的系统及其用途
LT3138910T (lt) 2012-12-06 2017-11-10 Sigma-Aldrich Co. Llc Crispr pagrįstas genomo modifikavimas ir reguliavimas
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
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
EP3434776A1 (en) 2012-12-12 2019-01-30 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
SG10201704932UA (en) 2012-12-17 2017-07-28 Harvard College Rna-guided human genome engineering
EP2971184B1 (en) 2013-03-12 2019-04-17 President and Fellows of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
NZ712727A (en) * 2013-03-14 2017-05-26 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
HUE040575T2 (hu) 2013-04-16 2019-03-28 Regeneron Pharma A patkány genom célzott módosítása
US20140356956A1 (en) * 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
SG11201509962QA (en) 2013-06-04 2016-01-28 Harvard College Rna-guideded transcriptional 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
EP3825406A1 (en) 2013-06-17 2021-05-26 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
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
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
WO2014204725A1 (en) * 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
US10563225B2 (en) * 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
DE202014010413U1 (de) 2013-09-18 2015-12-08 Kymab Limited Zellen und Organismen
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
US9546384B2 (en) 2013-12-11 2017-01-17 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a mouse genome
EP4219699A1 (en) 2013-12-12 2023-08-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
AU2014361781B2 (en) 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
AU2014362248A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
WO2015089465A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
EP3110971B1 (en) 2014-02-27 2019-05-08 The Broad Institute Inc. T cell balance gene expression and methods of use thereof
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3116997B1 (en) 2014-03-10 2019-05-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015148863A2 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
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
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
JP2017536811A (ja) 2014-10-15 2017-12-14 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 多能性細胞の樹立又は維持のための方法及び組成物
EP3212788A2 (en) 2014-10-27 2017-09-06 The Broad Institute, Inc. Compositions, methods and use of synthetic lethal screening
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
HUE044907T2 (hu) 2014-11-21 2019-11-28 Regeneron Pharma Módszerek és készítmények célzott genetikai módosításhoz, párosított mutató RNS-ek használatával
EP3224381B1 (en) 2014-11-25 2019-09-04 The Brigham and Women's Hospital, Inc. Method of identifying a person having a predisposition to or afflicted with a cardiometabolic disease
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
CN116059378A (zh) 2014-12-10 2023-05-05 明尼苏达大学董事会 用于治疗疾病的遗传修饰的细胞、组织和器官
EP3230451B1 (en) 2014-12-12 2021-04-07 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
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
WO2016108926A1 (en) 2014-12-30 2016-07-07 The Broad Institute Inc. Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis
EP3242938B1 (en) * 2015-01-09 2020-01-08 Bio-Rad Laboratories, Inc. Detection of genome editing
EP3262193A2 (en) 2015-02-26 2018-01-03 The Broad Institute Inc. T cell balance gene expression, compositions of matters and methods of use thereof
US11261466B2 (en) 2015-03-02 2022-03-01 Sinai Health System Homologous recombination factors
CN107532161A (zh) * 2015-03-03 2018-01-02 通用医疗公司 具有改变的PAM特异性的工程化CRISPR‑Cas9核酸酶
CN104726449A (zh) * 2015-03-23 2015-06-24 国家纳米科学中心 一种用于预防和/或治疗HIV的CRISPR-Cas9系统及其制备方法和用途
CA2981715A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
KR20170135966A (ko) 2015-04-13 2017-12-08 맥스시티 인코포레이티드 게놈 dna를 변형시키기 위한 방법 및 조성물
EP4019975A1 (en) 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
EP3291679B1 (en) 2015-05-06 2021-12-15 SNIPR Technologies Limited Altering microbial populations & modifying microbiota
WO2016182893A1 (en) 2015-05-08 2016-11-17 Teh Broad Institute Inc. Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof
JP7030522B2 (ja) 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
JP2018516984A (ja) * 2015-05-29 2018-06-28 アジェノビア コーポレーション 細胞を標的にしたｈｐｖ処置のための組成物および方法
WO2016196499A1 (en) * 2015-05-29 2016-12-08 Clark Atlanta University Human cell lines mutant for zic2
JP6968416B2 (ja) 2015-06-01 2021-11-17 テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション Ｒｎａ誘導性の、ｈｉｖ感染の処置のための、方法および組成物
US11911415B2 (en) 2015-06-09 2024-02-27 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for improving transplantation
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
FI3430134T3 (fi) 2015-06-18 2023-01-13 Uusia CRISPR-entsyymejä ja järjestelmiä
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
KR102840885B1 (ko) 2015-06-18 2025-07-30 더 브로드 인스티튜트, 인코퍼레이티드 표적외 효과를 감소시키는 crispr 효소 돌연변이
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
EP3313989B1 (en) 2015-06-29 2024-12-25 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
WO2017011721A1 (en) 2015-07-15 2017-01-19 Rutgers, The State University Of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
US10166255B2 (en) 2015-07-31 2019-01-01 Regents Of The University Of Minnesota Intracellular genomic transplant and methods of therapy
EP3334484B1 (en) 2015-08-12 2024-12-25 The General Hospital Corporation Compositions that promote hypoxia or the hypoxia response for the treatment and prevention of mitochondrial dysfunction
US11214800B2 (en) 2015-08-18 2022-01-04 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
CN108291224A (zh) * 2015-09-10 2018-07-17 优美佳生物技术有限公司 用于治疗青光眼的方法和组合物
CA2999500A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
WO2017069958A2 (en) 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
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
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
WO2017074788A1 (en) 2015-10-27 2017-05-04 The Broad Institute Inc. Compositions and methods for targeting cancer-specific sequence variations
WO2017075478A2 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
WO2017075294A1 (en) 2015-10-28 2017-05-04 The Board Institute Inc. Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
WO2017075265A1 (en) 2015-10-28 2017-05-04 The Broad Institute, Inc. Multiplex analysis of single cell constituents
MX2018005611A (es) 2015-11-03 2018-11-09 Harvard College Metodo y aparato para la formacion de imagenes volumetricas de una matriz tridimensional que contiene acido nucleico.
WO2017081288A1 (en) * 2015-11-11 2017-05-18 Lonza Ltd Crispr-associated (cas) proteins with reduced immunogenicity
EP3377042A4 (en) 2015-11-16 2019-05-29 The Research Institute at Nationwide Children's Hospital MATERIALS AND METHOD FOR THE TREATMENT OF TITIN-BASED MYOPATHIES AND OTHER TITINOPATHIES
CA3005878A1 (en) 2015-11-19 2017-05-26 The Brigham And Women's Hospital, Inc. Lymphocyte antigen cd5-like (cd5l)-interleukin 12b (p40) heterodimers in immunity
WO2017106569A1 (en) * 2015-12-18 2017-06-22 The Regents Of The University Of California Modified site-directed modifying polypeptides and methods of use thereof
CN109072218B (zh) * 2015-12-18 2023-04-18 国立研究开发法人科学技术振兴机构 基因修饰非人生物、卵细胞、受精卵以及目的基因的修饰方法
MY192848A (en) * 2015-12-28 2022-09-12 Intellia Therapeutics Inc Compositions and methods for the treatment of hemoglobinopathies
CA3009709A1 (en) 2015-12-30 2017-07-06 Novartis Ag Immune effector cell therapies with enhanced efficacy
WO2017127807A1 (en) * 2016-01-22 2017-07-27 The Broad Institute Inc. Crystal structure of crispr cpf1
WO2017147196A1 (en) 2016-02-22 2017-08-31 Massachusetts Institute Of Technology Methods for identifying and modulating immune phenotypes
WO2017147278A1 (en) * 2016-02-25 2017-08-31 The Children's Medical Center Corporation Customized class switch of immunoglobulin genes in lymphoma and hybridoma by crispr/cas9 technology
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
US11427861B2 (en) 2016-03-17 2022-08-30 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
US11512311B2 (en) 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
WO2017180694A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Cas9 fusion molecules gene editing systems, and methods of use thereof
CA3022290A1 (en) 2016-04-25 2017-11-02 President And Fellows Of Harvard College Hybridization chain reaction methods for in situ molecular detection
AU2017268458B2 (en) 2016-05-20 2022-07-21 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide RNAS
RU2018144745A (ru) * 2016-06-01 2020-07-09 Эксижн Биотерапьютикс, Инк. Композиции и способы лечения, направленные на литические и лизогенные вирусы
GB201609811D0 (en) 2016-06-05 2016-07-20 Snipr Technologies Ltd Methods, cells, systems, arrays, RNA and kits
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
CN106086008B (zh) * 2016-06-10 2019-03-12 中国农业科学院植物保护研究所 烟粉虱MED隐种TRP基因的CRISPR/cas9系统及其应用
WO2017217768A1 (ko) * 2016-06-15 2017-12-21 주식회사 툴젠 온타겟 및 오프타겟의 다중 타겟 시스템을 이용하는, 표적 특이적 유전자 가위 스크리닝 방법 및 이의 용도
US11708588B2 (en) 2016-06-16 2023-07-25 Adam Brian Robertson Gene editing
EP3472321A2 (en) * 2016-06-17 2019-04-24 Genesis Technologies Limited Crispr-cas system, materials and methods
CN107513538A (zh) * 2016-06-17 2017-12-26 北京大学 基因敲除方法
WO2017219027A1 (en) 2016-06-17 2017-12-21 The Broad Institute Inc. Type vi crispr orthologs and systems
AU2017280353B2 (en) * 2016-06-24 2021-11-11 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
CN106047877B (zh) * 2016-06-24 2019-01-11 中山大学附属第一医院 一种靶向敲除FTO基因的sgRNA及CRISPR/Cas9慢病毒系统与应用
CN105907777A (zh) * 2016-06-28 2016-08-31 中国科学院海洋研究所 一种Cas9核酸内切酶分泌型表达载体及其构建方法和应用
US11359234B2 (en) 2016-07-01 2022-06-14 Microsoft Technology Licensing, Llc Barcoding sequences for identification of gene expression
US20180004537A1 (en) * 2016-07-01 2018-01-04 Microsoft Technology Licensing, Llc Molecular State Machines
US10669558B2 (en) 2016-07-01 2020-06-02 Microsoft Technology Licensing, Llc Storage through iterative DNA editing
CN109863143B (zh) 2016-07-13 2021-10-15 威泰克斯制药公司 提高基因组编辑效率的方法、组合物和试剂盒
CN109563508B (zh) * 2016-07-29 2023-07-07 马克思—普朗克科学促进协会公司 通过定点dna裂解和修复靶向原位蛋白质多样化
US11566263B2 (en) 2016-08-02 2023-01-31 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
KR102827276B1 (ko) 2016-08-03 2025-07-01 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
TWI874884B (zh) * 2016-08-10 2025-03-01 日商領先基因生技股份有限公司 檢測標的部位中的檢測對象核酸序列之存在或不存在之方法
EP3500967A1 (en) 2016-08-17 2019-06-26 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2018035364A1 (en) 2016-08-17 2018-02-22 The Broad Institute Inc. Product and methods useful for modulating and evaluating immune responses
US20200283743A1 (en) 2016-08-17 2020-09-10 The Broad Institute, Inc. Novel crispr enzymes and systems
US11352647B2 (en) 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
WO2018053053A1 (en) 2016-09-13 2018-03-22 The Broad Institute, Inc. Proximity-dependent biotinylation and uses thereof
WO2017216392A1 (en) * 2016-09-23 2017-12-21 Dsm Ip Assets B.V. A guide-rna expression system for a host cell
US20190225974A1 (en) 2016-09-23 2019-07-25 BASF Agricultural Solutions Seed US LLC Targeted genome optimization in plants
WO2018064208A1 (en) 2016-09-28 2018-04-05 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
CN110214180A (zh) 2016-10-14 2019-09-06 哈佛大学的校长及成员们 核碱基编辑器的aav递送
GB2604416B (en) 2016-10-18 2023-03-15 Univ Minnesota Tumor infiltating lymphocytes and methods of therapy
US20180245065A1 (en) 2016-11-01 2018-08-30 Novartis Ag Methods and compositions for enhancing gene editing
WO2018089386A1 (en) 2016-11-11 2018-05-17 The Broad Institute, Inc. Modulation of intestinal epithelial cell differentiation, maintenance and/or function through t cell action
WO2018107103A1 (en) 2016-12-09 2018-06-14 The Broad Institute, Inc. Crispr-systems for modifying a trait of interest in a plant
US12286727B2 (en) 2016-12-19 2025-04-29 Editas Medicine, Inc. Assessing nuclease cleavage
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
US12065666B2 (en) 2017-01-05 2024-08-20 Rutgers, The State University Of New Jersey Targeted gene editing platform independent of DNA double strand break and uses thereof
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
KR102515727B1 (ko) * 2017-01-11 2023-03-30 주식회사 툴젠 중첩된 가이드핵산을 이용한 표적 핵산에 특정 핵산 서열을 삽입하기 위한 조성물 및 방법
WO2018135838A2 (ko) * 2017-01-17 2018-07-26 기초과학연구원 Dna 단일가닥 절단에 의한 염기 교정 비표적 위치 확인 방법
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
JP2020510038A (ja) 2017-03-09 2020-04-02 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 がんワクチン
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CN110959039B (zh) 2017-03-15 2025-03-14 博德研究所 新型cas13b直向同源物crispr酶和系统
WO2018170515A1 (en) 2017-03-17 2018-09-20 The Broad Institute, Inc. Methods for identifying and modulating co-occurant cellular phenotypes
GB2575930A (en) 2017-03-23 2020-01-29 Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
WO2018175924A1 (en) 2017-03-24 2018-09-27 The Broad Institute, Inc. Methods and compositions for regulating innate lymphoid cell inflammatory responses
CN110892069B (zh) * 2017-03-30 2024-02-09 国立大学法人京都大学 基于基因组编辑的外显子跳跃诱导方法
US11963966B2 (en) 2017-03-31 2024-04-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating ovarian tumors
EP3606518A4 (en) 2017-04-01 2021-04-07 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR DETECTION AND MODULATION OF IMMUNOTHERAPY RESISTANCE GENE SIGNATURE IN CANCER
US20200071773A1 (en) 2017-04-12 2020-03-05 Massachusetts Eye And Ear Infirmary Tumor signature for metastasis, compositions of matter methods of use thereof
KR20200006054A (ko) 2017-04-12 2020-01-17 더 브로드 인스티튜트, 인코퍼레이티드 신규 타입 vi crispr 오르소로그 및 시스템
US12331319B2 (en) 2017-04-12 2025-06-17 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
US20210293783A1 (en) 2017-04-18 2021-09-23 The General Hospital Corporation Compositions for detecting secretion and methods of use
WO2018195402A1 (en) 2017-04-20 2018-10-25 Egenesis, Inc. Methods for generating genetically modified animals
US12208140B2 (en) 2017-04-21 2025-01-28 The Broad Institute, Inc. Targeted delivery to beta cells
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
WO2018204777A2 (en) 2017-05-05 2018-11-08 The Broad Institute, Inc. Methods for identification and modification of lncrna associated with target genotypes and phenotypes
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
EP3622092A4 (en) 2017-05-11 2021-06-23 The Broad Institute, Inc. METHODS AND COMPOSITIONS OF USE OF CD8 + TUMOR-INFILTRATING LYMPHOCYTE SUBTYPES AND GENE SIGNATURES THEREOF
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
US12297436B2 (en) 2017-05-18 2025-05-13 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
AU2018270088B2 (en) 2017-05-18 2024-05-16 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
WO2018218188A2 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Base editors with improved precision and specificity
CA3102054A1 (en) 2017-06-05 2018-12-13 Fred Hutchinson Cancer Research Center Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies
EP3635104A1 (en) 2017-06-09 2020-04-15 Editas Medicine, Inc. Engineered cas9 nucleases
EP3638218A4 (en) 2017-06-14 2021-06-09 The Broad Institute, Inc. COMPOSITIONS AND METHOD OF TARGETING COMPLEMENTING COMPONENT 3 FOR INHIBITION OF TUMOR GROWTH
CN111328290A (zh) 2017-06-26 2020-06-23 博德研究所 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法
WO2019005926A1 (en) * 2017-06-27 2019-01-03 Massachusetts Eye And Ear Infirmary VEGFR2 AAV-CRISPR / CAS9 GENOME EDITION FOR THE TREATMENT OF OCULAR DISEASES
JP2020530307A (ja) 2017-06-30 2020-10-22 インティマ・バイオサイエンス，インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
EP3645721A1 (en) 2017-06-30 2020-05-06 Novartis AG Methods for the treatment of disease with gene editing systems
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
WO2019014581A1 (en) 2017-07-14 2019-01-17 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODULATING THE ACTIVITY OF A CYTOTOXIC LYMPHOCYTE
WO2019018440A1 (en) 2017-07-17 2019-01-24 The Broad Institute, Inc. HUMAN COLON CELL ATLAS IN GOOD HEALTH AND WITH HEMORRHAGIC RECTO-COLITIS
US12239646B2 (en) 2017-07-19 2025-03-04 Dana-Farber Cancer Institute, Inc. Cancer diagnostic and treatment
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
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
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR102338449B1 (ko) 2017-09-21 2021-12-10 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물
KR20250093420A (ko) * 2017-09-29 2025-06-24 인텔리아 테라퓨틱스, 인크. 게놈 편집을 위한 폴리뉴클레오티드, 조성물 및 방법
EP3695408A4 (en) 2017-10-02 2021-12-15 The Broad Institute, Inc. PROCEDURES AND COMPOSITIONS FOR DETECTION AND MODULATION OF IMMUNOTHERAPY RESISTANT GENE SIGNATURE IN CANCER
WO2019071054A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
WO2019079362A1 (en) 2017-10-16 2019-04-25 Massachusetts Institute Of Technology HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS
MX2020004063A (es) 2017-10-20 2020-10-05 Hutchinson Fred Cancer Res Sistemas y metodos para producir celulas b modificadas geneticamente para expresar anticuerpos seleccionados.
WO2019135816A2 (en) 2017-10-23 2019-07-11 The Broad Institute, Inc. Novel nucleic acid modifiers
WO2019089803A1 (en) 2017-10-31 2019-05-09 The Broad Institute, Inc. Methods and compositions for studying cell evolution
US20240229096A1 (en) * 2017-10-31 2024-07-11 Hub Biotech Co., Ltd. Recombinant cell and method for producing endogenous polypeptide
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
US12171783B2 (en) 2017-11-13 2024-12-24 The Broad Institute, Inc. Methods and compositions for targeting developmental and oncogenic programs in H3K27M gliomas
WO2019094984A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
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
WO2019113506A1 (en) 2017-12-07 2019-06-13 The Broad Institute, Inc. Methods and compositions for multiplexing single cell and single nuclei sequencing
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US11994512B2 (en) 2018-01-04 2024-05-28 Massachusetts Institute Of Technology Single-cell genomic methods to generate ex vivo cell systems that recapitulate in vivo biology with improved fidelity
AU2019207409B2 (en) 2018-01-12 2023-02-23 Basf Se Gene underlying the number of spikelets per spike qtl in wheat on chromosome 7a
BR112020013626A2 (pt) 2018-01-17 2020-12-01 Vertex Pharmaceuticals Incorporated compostos de quinoxalinona, composições, métodos e kits para aumentar a eficiência de edição de genoma
AU2019209293B2 (en) 2018-01-17 2023-07-27 Vertex Pharmaceuticals Incorporated DNA-PK inhibitors
EA202091709A1 (ru) 2018-01-17 2020-11-10 Вертекс Фармасьютикалз Инкорпорейтед Ингибиторы днк-пк
TWI815856B (zh) 2018-01-31 2023-09-21 美國全美兒童醫院之研究學會 用於肢帶型肌肉失養症2c型之基因療法
SG11202008956XA (en) 2018-03-14 2020-10-29 Editas Medicine Inc Systems and methods for the treatment of hemoglobinopathies
US10760075B2 (en) 2018-04-30 2020-09-01 Snipr Biome Aps Treating and preventing microbial infections
WO2019204585A1 (en) 2018-04-19 2019-10-24 Massachusetts Institute Of Technology Single-stranded break detection in double-stranded dna
ES2922902T3 (es) 2018-04-24 2022-09-21 Kws Saat Se & Co Kgaa Plantas con digestibilidad mejorada y haplotipos marcadores
US11957695B2 (en) 2018-04-26 2024-04-16 The Broad Institute, Inc. Methods and compositions targeting glucocorticoid signaling for modulating immune responses
WO2019210268A2 (en) 2018-04-27 2019-10-31 The Broad Institute, Inc. Sequencing-based proteomics
WO2019210491A1 (zh) * 2018-05-03 2019-11-07 深圳市博奥康生物科技有限公司 CRISPR/Cas9靶向敲除人CVAP基因及其特异性gRNA
WO2019213660A2 (en) 2018-05-04 2019-11-07 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
WO2019232542A2 (en) 2018-06-01 2019-12-05 Massachusetts Institute Of Technology Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients
EP3578658A1 (en) * 2018-06-08 2019-12-11 Johann Wolfgang Goethe-Universität Frankfurt Method for generating a gene editing vector with fixed guide rna pairs
US12036240B2 (en) 2018-06-14 2024-07-16 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
BR112020026306A2 (pt) 2018-06-26 2021-03-30 The Broad Institute Inc. Métodos de amplificação, sistemas e diagnósticos baseados em sistema efeitor crispr
MX2020013461A (es) 2018-06-26 2021-04-28 Broad Inst Inc Composiciones, sistemas y métodos de amplificación basados en la doble nicasa crispr.
US20210147915A1 (en) 2018-06-26 2021-05-20 The Broad Institute, Inc. Crispr/cas and transposase based amplification compositions, systems and methods
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
CN112543810A (zh) 2018-06-29 2021-03-23 全国儿童医院研究所 治疗肢带型肌营养不良2a型的重组腺相关病毒产品和方法
TW202023605A (zh) * 2018-07-13 2020-07-01 美商綠陽生物科技及製藥公司 達成基因組編輯之高特異性的方法
EP3830256A2 (en) 2018-07-31 2021-06-09 The Broad Institute, Inc. Novel crispr enzymes and systems
CN108998406B (zh) * 2018-08-03 2022-05-10 福州大学 一种人类原代培养细胞基因组编辑、定点基因敲入方法
CN108949830B (zh) * 2018-08-03 2021-11-26 福州大学 一种在鱼类中实现基因组编辑、精确定点基因敲入的方法
CN113286884A (zh) 2018-08-07 2021-08-20 博德研究所 新型cas12b酶和系统
WO2020041387A1 (en) 2018-08-20 2020-02-27 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
WO2020051507A1 (en) 2018-09-06 2020-03-12 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
WO2020056170A1 (en) 2018-09-12 2020-03-19 Fred Hutchinson Cancer Research Center Reducing cd33 expression to selectively protect therapeutic cells
WO2020061229A2 (en) 2018-09-18 2020-03-26 Vnv Newco Inc. Arc-based capsids and uses thereof
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
US20220411783A1 (en) 2018-10-12 2022-12-29 The Broad Institute, Inc. Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues
US20210379057A1 (en) 2018-10-16 2021-12-09 Massachusetts Institute Of Technology Nutlin-3a for use in treating a mycobacterium tuberculosis infection
WO2020082046A2 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
JP7472121B2 (ja) * 2018-10-18 2024-04-22 インテリア セラピューティクス，インコーポレーテッド アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
CN109411022A (zh) * 2018-11-07 2019-03-01 宁夏农林科学院农业生物技术研究中心（宁夏农业生物技术重点实验室） 一种基于字符切片技术的含PAM结构gRNA靶向序列筛选方法及应用
US12402610B2 (en) 2018-11-09 2025-09-02 The Broad Institute, Inc. Methods and compositions for modulating innate lymphoid cell pathogenic effectors
US12165743B2 (en) 2018-11-09 2024-12-10 The Broad Institute, Inc. Compressed sensing for screening and tissue imaging
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
WO2020131862A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
CN109679953B (zh) * 2018-12-28 2022-08-16 广州赛业百沐生物科技有限公司 利用CRISPR-Cas9系统制得基因点突变动物模型胚胎的靶序列组、载体和方法
US11739156B2 (en) 2019-01-06 2023-08-29 The Broad Institute, Inc. Massachusetts Institute of Technology Methods and compositions for overcoming immunosuppression
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3921417A4 (en) 2019-02-04 2022-11-09 The General Hospital Corporation VARIANTS OF ADENINE DNA BASES WITH REDUCED OFF-TARGET RNA EDIT
BR112021016570A2 (pt) 2019-02-26 2021-11-03 Res Inst Nationwide Childrens Hospital Entrega de vetor de vírus adenoassociado de beta-sarcoglicano e tratamento de distrofia muscular
US20220154282A1 (en) 2019-03-12 2022-05-19 The Broad Institute, Inc. Detection means, compositions and methods for modulating synovial sarcoma cells
WO2020186237A1 (en) 2019-03-13 2020-09-17 The Broad Institute, Inc. Microglial progenitors for regeneration of functional microglia in the central nervous system and therapeutics uses thereof
WO2020186235A1 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate intestinal innate lymphoid cells
WO2020191069A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Modulation of type 2 immunity by targeting clec-2 signaling
US20220142948A1 (en) 2019-03-18 2022-05-12 The Broad Institute, Inc. Compositions and methods for modulating metabolic regulators of t cell pathogenicity
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US20220195514A1 (en) 2019-03-29 2022-06-23 The Broad Institute, Inc. Construct for continuous monitoring of live cells
US20220162649A1 (en) 2019-04-01 2022-05-26 The Broad Institute, Inc. Novel nucleic acid modifiers
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
UY38693A (es) 2019-05-13 2020-12-31 Kws Saat Se & Co Kgaa Tolerancia a la sequía en maíz
WO2020232271A1 (en) 2019-05-14 2020-11-19 The Broad Institute, Inc. Compositions and methods for targeting multinucleated cells
US20220235340A1 (en) 2019-05-20 2022-07-28 The Broad Institute, Inc. Novel crispr-cas systems and uses thereof
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
AR118995A1 (es) 2019-05-25 2021-11-17 Kws Saat Se & Co Kgaa Mejorador de la inducción de haploides
WO2020243371A1 (en) 2019-05-28 2020-12-03 Massachusetts Institute Of Technology Methods and compositions for modulating immune responses
WO2020243661A1 (en) 2019-05-31 2020-12-03 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
AU2020298572A1 (en) 2019-07-02 2021-11-18 Fred Hutchinson Cancer Center Recombinant Ad35 vectors and related gene therapy improvements
EP3772542A1 (en) 2019-08-07 2021-02-10 KWS SAAT SE & Co. KGaA Modifying genetic variation in crops by modulating the pachytene checkpoint protein 2
WO2021030627A1 (en) 2019-08-13 2021-02-18 The General Hospital Corporation Methods for predicting outcomes of checkpoint inhibition and treatment thereof
US12421557B2 (en) 2019-08-16 2025-09-23 The Broad Institute, Inc. Methods for predicting outcomes and treating colorectal cancer using a cell atlas
EP4374880A3 (en) 2019-08-21 2024-08-14 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
WO2021041922A1 (en) 2019-08-30 2021-03-04 The Broad Institute, Inc. Crispr-associated mu transposase systems
EP4031660A1 (en) 2019-09-20 2022-07-27 The Broad Institute, Inc. Novel type vi crispr enzymes and systems
US11981922B2 (en) 2019-10-03 2024-05-14 Dana-Farber Cancer Institute, Inc. Methods and compositions for the modulation of cell interactions and signaling in the tumor microenvironment
US12195725B2 (en) 2019-10-03 2025-01-14 Dana-Farber Cancer Institute, Inc. Compositions and methods for modulating and detecting tissue specific TH17 cell pathogenicity
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
AU2020366566A1 (en) 2019-10-17 2022-04-21 KWS SAAT SE & Co. KGaA Enhanced disease resistance of crops by downregulation of repressor genes
CN112725348B (zh) * 2019-10-28 2022-04-01 安徽省农业科学院水稻研究所 一种提高水稻单碱基编辑效率的基因、方法及应用
US11844800B2 (en) 2019-10-30 2023-12-19 Massachusetts Institute Of Technology Methods and compositions for predicting and preventing relapse of acute lymphoblastic leukemia
US12195723B2 (en) 2019-11-08 2025-01-14 The Broad Institute, Inc. Engineered antigen presenting cells and uses thereof
US20230340537A1 (en) 2020-01-17 2023-10-26 The Broad Institute, Inc. Small type ii-d cas proteins and methods of use thereof
US12165747B2 (en) 2020-01-23 2024-12-10 The Broad Institute, Inc. Molecular spatial mapping of metastatic tumor microenvironment
EP4093863A4 (en) 2020-01-24 2024-04-10 The General Hospital Corporation CRISPR-CAS ENZYMES WITH ENHANCED ON-TARGET ACTIVITY
EP4093864A4 (en) 2020-01-24 2024-04-10 The General Hospital Corporation Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants
WO2021202800A1 (en) * 2020-03-31 2021-10-07 Arbor Biotechnologies, Inc. Compositions comprising a cas12i2 variant polypeptide and uses thereof
AU2021267940A1 (en) 2020-05-08 2022-12-08 President And Fellows Of Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
EP4156913A1 (en) 2020-05-29 2023-04-05 KWS SAAT SE & Co. KGaA Plant haploid induction
CN116194138A (zh) 2020-08-07 2023-05-30 博德研究所 治疗性靶向癌症中经由xpr1:kidins220蛋白质复合物的磷酸盐调节异常
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
WO2022169235A1 (ko) * 2021-02-04 2022-08-11 연세대학교 산학협력단 교정 효율이 개선된 프라임에디팅용 조성물
EP4047095A1 (en) * 2021-02-22 2022-08-24 Eberhard Karls Universität Tübingen Medizinische Fakultät Method and composition for a targeted gene knockout
CN113403309B (zh) * 2021-05-24 2022-08-16 珠海舒桐医疗科技有限公司 非同源双链寡聚核苷酸片段在基因敲除系统中的应用
KR20240031238A (ko) 2021-06-01 2024-03-07 아버 바이오테크놀로지스, 인크. Crispr 뉴클레이스를 포함하는 유전자 편집 시스템 및 이의 용도
WO2023006933A1 (en) 2021-07-30 2023-02-02 KWS SAAT SE & Co. KGaA Plants with improved digestibility and marker haplotypes
EP4392557A1 (en) * 2021-08-23 2024-07-03 Max-Delbrück-Centrum für Molekulare Medizin in der Helmholtz-Gemeinschaft Method for cas9 nickase-mediated gene editing
US11884915B2 (en) 2021-09-10 2024-01-30 Agilent Technologies, Inc. Guide RNAs with chemical modification for prime editing
US12285497B2 (en) 2021-10-15 2025-04-29 Research Institute At Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
WO2023077148A1 (en) 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
CN118401658A (zh) 2021-11-26 2024-07-26 益杰立科(上海)生物科技有限公司 调节pcsk9的方法及其用途
CA3244180A1 (en) 2021-12-22 2023-06-29 Tome Biosciences, Inc. CO-ADMINISTRATION OF A GENE EDITOR CONSTRUCTION AND A PATTERN DONOR
CN119562762A (zh) 2022-04-04 2025-03-04 加利福尼亚大学董事会 遗传互补组合物和方法
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
GB202209518D0 (en) 2022-06-29 2022-08-10 Snipr Biome Aps Treating & preventing E coli infections
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
AU2023329149A1 (en) 2022-08-26 2025-02-20 KWS SAAT SE & Co. KGaA Use of paired genes in hybrid breeding
CN116218905B (zh) * 2022-12-06 2024-01-26 复旦大学附属华山医院 一种点突变大鼠癫痫模型的构建与应用
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
KR20250151430A (ko) 2023-02-15 2025-10-21 아버 바이오테크놀로지스, 인크. 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법
WO2024192141A1 (en) 2023-03-13 2024-09-19 Dana-Farber Cancer Institute, Inc. Treatment of cancers having a drug-resistant mesenchymal cell state
WO2024197185A1 (en) 2023-03-21 2024-09-26 The Broad Institute, Inc. Methods and compositions for dissecting organelle physiology
TW202449159A (zh) 2023-04-19 2024-12-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於產生文庫之組合物及方法
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
WO2024236547A1 (en) 2023-05-18 2024-11-21 Inceptor Bio, Llc Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof
US20240425868A1 (en) 2023-06-26 2024-12-26 Flagship Pioneering Innovations Vii, Llc Engineered plasmodia and related methods
WO2025046513A1 (en) 2023-08-29 2025-03-06 Inceptor Bio, Llc Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025059533A1 (en) 2023-09-13 2025-03-20 The Broad Institute, Inc. Crispr enzymes and systems
KR20250113347A (ko) 2024-01-17 2025-07-25 중앙대학교 산학협력단 CRISPR-Cas nickase 시스템 기반 정교한 유전체 편집 방법
WO2025171210A1 (en) 2024-02-09 2025-08-14 Arbor Biotechnologies, Inc. Compositions and methods for gene editing via homology-mediated end joining
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025224107A1 (en) 2024-04-22 2025-10-30 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
JP7787627B1 (ja) * 2025-08-08 2025-12-17 国立研究開発法人農業・食品産業技術総合研究機構 予測装置及び予測方法

Family Cites Families (171)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
JP2874751B2 (ja) 1986-04-09 1999-03-24 ジェンザイム・コーポレーション 希望する蛋白質をミルク中へ分泌する遺伝子移植動物
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US4873316A (en) 1987-06-23 1989-10-10 Biogen, Inc. Isolation of exogenous recombinant proteins from the milk of transgenic mammals
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5210015A (en) 1990-08-06 1993-05-11 Hoffman-La Roche Inc. Homogeneous assay system using the nuclease activity of a nucleic acid polymerase
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US7150982B2 (en) 1991-09-09 2006-12-19 Third Wave Technologies, Inc. RNA detection assays
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US7745416B2 (en) 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
AU698739B2 (en) 1995-06-06 1998-11-05 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5985662A (en) 1995-07-13 1999-11-16 Isis Pharmaceuticals Inc. Antisense inhibition of hepatitis B virus replication
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
NZ533288A (en) 1997-10-24 2005-10-28 Invitrogen Corp Recombinational cloning using nucleic acids having recombination sites
US6750059B1 (en) 1998-07-16 2004-06-15 Whatman, Inc. Archiving of vectors
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7868149B2 (en) 1999-07-20 2011-01-11 Monsanto Technology Llc Plant genome sequence and uses thereof
US6603061B1 (en) 1999-07-29 2003-08-05 Monsanto Company Agrobacterium-mediated plant transformation method
WO2002074968A1 (en) 2001-03-16 2002-09-26 Naoya Kobayashi Method for proliferating a liver cell, a liver cell obtained thereby, and use thereof
PT1385538E (pt) 2001-04-05 2013-05-07 Univ Johns Hopkins Vacinas quiméricas compreendendo o domínio luminal de lamp-1 ou lamp-2
EP1412371B1 (en) 2001-07-12 2016-02-24 University of Massachusetts IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING
WO2003014318A2 (en) 2001-08-08 2003-02-20 Genzyme Corporation Methods for treating diabetes and other blood sugar disorders
WO2003016338A1 (en) 2001-08-15 2003-02-27 Parker Hughes Institute Crystal structure of the btk kinase domain
US7776321B2 (en) 2001-09-26 2010-08-17 Mayo Foundation For Medical Education And Research Mutable vaccines
US20090100536A1 (en) 2001-12-04 2009-04-16 Monsanto Company Transgenic plants with enhanced agronomic traits
WO2003087993A2 (en) 2002-04-09 2003-10-23 Beattie Kenneth L Oligonucleotide probes for genosensor chips
US20070020627A1 (en) 2002-06-11 2007-01-25 The Scripps Research Institute Artificial transcription factors
EP1546397A4 (en) 2002-09-27 2007-10-31 Cold Spring Harbor Lab CELL BASED RNA INTERFERENCE AND ASSOCIATED METHODS AND COMPOSITIONS
WO2004046321A2 (en) 2002-11-15 2004-06-03 Trustees Of Boston University Cis/trans riboregulators
US20060178297A1 (en) 2003-01-28 2006-08-10 Troy Carol M Systems and methods for silencing expression of a gene in a cell and uses thereof
AU2004257191B2 (en) 2003-07-03 2009-05-07 The Regents Of The University Of California Genome mapping of functional DNA elements and cellular proteins
CA2534296C (en) 2003-08-08 2013-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
FR2862659B1 (fr) 2003-11-21 2006-02-10 Pasteur Institut Genome de legionella pneumophila souche paris- applications diagnostiques et epidemiologiques
WO2005070948A1 (en) 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
US20070244031A1 (en) 2004-01-27 2007-10-18 Quan Lu Methods and Compositions for Homozygous Gene Inactivation Using Collections of Pre-Defined Nucleotide Sequences Complementary Chromosomal Transcripts
US20050220796A1 (en) 2004-03-31 2005-10-06 Dynan William S Compositions and methods for modulating DNA repair
FR2872170B1 (fr) 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
AU2005274948B2 (en) 2004-07-16 2011-09-22 Genvec, Inc. Vaccines against aids comprising CMV/R-nucleic acid constructs
CN101228176A (zh) 2005-04-28 2008-07-23 贝尼泰克有限公司 用于同时递送与杂合表达模式相关的RNAi因子的多重RNAi表达盒
US7892224B2 (en) 2005-06-01 2011-02-22 Brainlab Ag Inverse catheter planning
WO2007014275A2 (en) 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
DE602006015484D1 (de) 2005-08-26 2010-08-26 Danisco Verwendung der crispr assoziierten gene (cas)
US8362229B2 (en) 2006-02-08 2013-01-29 Quark Pharmaceuticals, Inc. Tandem siRNAS
EP1994182B1 (en) 2006-03-15 2019-05-29 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
EA015676B1 (ru) 2006-05-11 2011-10-31 Элнилэм Фармасьютикалз, Инк. Композиции и способы ингибирования экспрессии гена pcsk9
US8748567B2 (en) 2006-05-22 2014-06-10 Children's Medical Center Corporation Method for delivery across the blood brain barrier
WO2008093152A1 (en) 2007-02-01 2008-08-07 Cellectis Obligate heterodimer meganucleases and uses thereof
ES2719789T3 (es) 2007-03-02 2019-07-16 Dupont Nutrition Biosci Aps Cultivos con resistencia mejorada a fagos
PE20090064A1 (es) 2007-03-26 2009-03-02 Novartis Ag Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
EP2225002A4 (en) 2007-12-31 2011-06-22 Nanocor Therapeutics Inc RNA INTERFERENCE FOR THE TREATMENT OF CARDIAC SUFFICIENCIES
WO2009105690A2 (en) 2008-02-21 2009-08-27 Targeted Genetics Corporation Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
US8546553B2 (en) 2008-07-25 2013-10-01 University Of Georgia Research Foundation, Inc. Prokaryotic RNAi-like system and methods of use
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
CA3006395C (en) 2008-11-07 2022-05-31 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
US20110016540A1 (en) 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
CA2745031C (en) 2008-12-04 2018-08-14 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
EP2373382B1 (en) 2008-12-10 2017-02-15 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
WO2010075424A2 (en) 2008-12-22 2010-07-01 The Regents Of University Of California Compositions and methods for downregulating prokaryotic genes
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
ES2539327T3 (es) * 2009-04-07 2015-06-29 Dow Agrosciences Llc Reparto mediado de nanopartículas de nucleasas específicas de secuencias
WO2011036510A1 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease variants cleaving the genome of the herpes simplex virus and uses thereof
JP2012529287A (ja) * 2009-06-11 2012-11-22 トゥールゲン インコーポレイション 部位−特異的ヌクレアーゼを用いた標的ゲノムの再配列
JP2013500018A (ja) 2009-07-24 2013-01-07 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニー ゲノム編集のための方法
EP2727600B1 (en) 2009-07-28 2019-03-27 Sangamo Therapeutics, Inc. Zinc finger fusion proteins for repressing a huntington gene
WO2011028929A2 (en) 2009-09-03 2011-03-10 The Regents Of The University Of California Nitrate-responsive promoter
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
CA2782014C (en) 2009-11-27 2021-08-31 Basf Plant Science Company Gmbh Optimized endonucleases and uses thereof
HUE041436T2 (hu) * 2009-12-10 2019-05-28 Univ Minnesota Tal-effektor-közvetített DNS-módosítás
SG181823A1 (en) 2009-12-23 2012-07-30 Max Planck Gesellschaft Influenza targets
JP2013518602A (ja) * 2010-02-09 2013-05-23 サンガモ バイオサイエンシーズ， インコーポレイテッド 部分的に一本鎖のドナー分子による標的化ゲノム改変
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
WO2011143124A2 (en) 2010-05-10 2011-11-17 The Regents Of The University Of California Endoribonuclease compositions and methods of use thereof
WO2011146121A1 (en) 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Novel dna-binding proteins and uses thereof
SG187075A1 (en) * 2010-07-23 2013-03-28 Sigma Aldrich Co Llc Genome editing using targeting endonucleases and single-stranded nucleic acids
EP2601611B1 (en) 2010-08-02 2020-12-09 Integrated Dna Technologies, Inc. Methods for predicting stability and melting temperatures of nucleic acid duplexes
US9549901B2 (en) 2010-09-03 2017-01-24 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
CN103270046B (zh) 2010-10-12 2016-05-11 费城儿童医院 治疗b型血友病的方法和组合物
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
ES2638342T3 (es) 2011-04-22 2017-10-19 The Regents Of The University Of California Viriones de virus adenoasociado con cápside variante y métodos para su uso
SG194089A1 (en) 2011-04-27 2013-11-29 Amyris Inc Methods for genomic modification
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
EP2758529B1 (en) 2011-09-21 2019-03-20 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
WO2013052681A1 (en) * 2011-10-06 2013-04-11 Sangamo Biosciences, Inc. Methods and compositions for regulating hiv infection
EP2591770B1 (en) 2011-11-14 2016-03-16 Silenseed Ltd Compositions for siRNA delivery and methods of manufacturing and using same
WO2013071440A1 (en) 2011-11-18 2013-05-23 UNIVERSITé LAVAL Methods and products for increasing frataxin levels and uses thereof
EP2782596A4 (en) 2011-11-22 2015-07-29 Philadelphia Children Hospital VIRUS VECTORS FOR HIGHLY EFFICIENT TRANSGENIC ADMINISTRATION
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
CN108285491B (zh) 2012-02-29 2021-08-10 桑格摩生物科学股份有限公司 治疗亨廷顿氏病的方法和组合物
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
AU2013204327B2 (en) 2012-04-20 2016-09-01 Aviagen Cell transfection method
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US9623071B2 (en) 2012-06-01 2017-04-18 Drexel University Modulation of hepatitis B virus CCCDNA transcription
JP6401704B2 (ja) 2012-10-10 2018-10-10 サンガモ セラピューティクス， インコーポレイテッド Ｔ細胞を修飾する化合物およびその使用
CN110643600A (zh) 2012-10-23 2020-01-03 基因工具股份有限公司 用于切割靶dna的系统及其用途
LT3138910T (lt) * 2012-12-06 2017-11-10 Sigma-Aldrich Co. Llc Crispr pagrįstas genomo modifikavimas ir reguliavimas
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2921557B1 (en) 2012-12-12 2016-07-13 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
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
EP3434776A1 (en) 2012-12-12 2019-01-30 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
PL2771468T3 (pl) 2012-12-12 2015-07-31 Broad Inst Inc Inżynieria systemów, metod i zoptymalizowanych kompozycji kierujących dla manipulacji sekwencjami
ES2786193T3 (es) 2012-12-12 2020-10-09 Broad Inst Inc Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
SG10201704932UA (en) 2012-12-17 2017-07-28 Harvard College Rna-guided human genome engineering
EP2946015B1 (en) 2013-01-16 2021-05-26 Emory University Cas9-nucleic acid complexes and uses related thereto
US11135273B2 (en) 2013-02-07 2021-10-05 The Rockefeller University Sequence specific antimicrobials
WO2014124226A1 (en) 2013-02-07 2014-08-14 The Rockefeller University Sequence specific antimicrobials
US9163837B2 (en) 2013-02-27 2015-10-20 Siemens Aktiengesellschaft Flow conditioner in a combustor of a gas turbine engine
NZ712727A (en) 2013-03-14 2017-05-26 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
KR102271292B1 (ko) 2013-03-15 2021-07-02 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도
ES2645931T3 (es) 2013-03-27 2017-12-11 Wilco Ag Método de inspección y/o de prueba en línea de dispositivos y aparato para realizar tal método
CA2908253C (en) 2013-04-04 2024-01-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
CA2910489A1 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
AU2014273082B2 (en) 2013-05-29 2018-11-08 Cellectis A method for producing precise DNA cleavage using Cas9 nickase activity
SG11201509962QA (en) * 2013-06-04 2016-01-28 Harvard College Rna-guideded transcriptional regulation
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
ES2987399T3 (es) 2013-06-05 2024-11-14 Univ Duke Edición génica guiada por ARN y regulación génica
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
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
EP3825406A1 (en) 2013-06-17 2021-05-26 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
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
CN103343120B (zh) 2013-07-04 2015-03-04 中国科学院遗传与发育生物学研究所 一种小麦基因组定点改造方法
PL3019619T3 (pl) 2013-07-11 2022-01-10 Modernatx, Inc. Kompozycje zawierające syntetyczne polinkleotydy kodujące białka powiązane z crispr i syntetyczne sgrna oraz sposoby ich stosowania
CN103388006B (zh) 2013-07-26 2015-10-28 华东师范大学 一种基因定点突变的构建方法
KR20160060659A (ko) 2013-08-29 2016-05-30 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염증의 rna-유도 치료를 위한 방법 및 조성물
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
WO2015048690A1 (en) 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
AU2014350051A1 (en) 2013-11-18 2016-07-07 Crispr Therapeutics Ag CRISPR-Cas system materials and methods
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
AU2014362248A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
CN106536729A (zh) 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
EP3080260B1 (en) 2013-12-12 2019-03-06 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
CN103668472B (zh) 2013-12-31 2014-12-24 北京大学 利用CRISPR/Cas9系统构建真核基因敲除文库的方法
US10354746B2 (en) 2014-01-27 2019-07-16 Georgia Tech Research Corporation Methods and systems for identifying CRISPR/Cas off-target sites
JP6323228B2 (ja) 2014-07-18 2018-05-16 富士電機株式会社 電力変換装置
US9932566B2 (en) 2014-08-07 2018-04-03 Agilent Technologies, Inc. CIS-blocked guide RNA
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
CN107532161A (zh) 2015-03-03 2018-01-02 通用医疗公司 具有改变的PAM特异性的工程化CRISPR‑Cas9核酸酶
EP3291679B1 (en) 2015-05-06 2021-12-15 SNIPR Technologies Limited Altering microbial populations & modifying microbiota
KR102840885B1 (ko) 2015-06-18 2025-07-30 더 브로드 인스티튜트, 인코퍼레이티드 표적외 효과를 감소시키는 crispr 효소 돌연변이
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
JP6186470B2 (ja) 2016-04-20 2017-08-23 パイオニア株式会社 音響装置、音量制御方法、音量制御プログラム及び記録媒体
CN107939288B (zh) 2017-11-14 2019-04-02 中国科学院地质与地球物理研究所 一种非旋转套的防转装置以及旋转导向装置

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