DK3011029T3 - Administration, modificering og optimering af tandem-guidesystemer, fremgangsmåder og sammensætninger til sekvensmanipulering - Google Patents

Administration, modificering og optimering af tandem-guidesystemer, fremgangsmåder og sammensætninger til sekvensmanipulering Download PDF

Info

Publication number
DK3011029T3
DK3011029T3 DK14734708.2T DK14734708T DK3011029T3 DK 3011029 T3 DK3011029 T3 DK 3011029T3 DK 14734708 T DK14734708 T DK 14734708T DK 3011029 T3 DK3011029 T3 DK 3011029T3
Authority
DK
Denmark
Prior art keywords
optimization
administration
procedures
compositions
modification
Prior art date
Application number
DK14734708.2T
Other languages
Danish (da)
English (en)
Inventor
Feng Zhang
Chie-Yu Lin
Fei Ran
Original Assignee
Broad Inst Inc
Massachusetts Inst Technology
Harvard College
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Broad Inst Inc, Massachusetts Inst Technology, Harvard College filed Critical Broad Inst Inc
Application granted granted Critical
Publication of DK3011029T3 publication Critical patent/DK3011029T3/da

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
DK14734708.2T 2013-06-17 2014-06-10 Administration, modificering og optimering af tandem-guidesystemer, fremgangsmåder og sammensætninger til sekvensmanipulering DK3011029T3 (da)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201361836123P 2013-06-17 2013-06-17
US201361847537P 2013-07-17 2013-07-17
US201361862468P 2013-08-05 2013-08-05
US201361862355P 2013-08-05 2013-08-05
US201361871301P 2013-08-28 2013-08-28
US201361915407P 2013-12-12 2013-12-12
US201461979942P 2014-04-15 2014-04-15
PCT/US2014/041800 WO2014204724A1 (en) 2013-06-17 2014-06-10 Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation

Publications (1)

Publication Number Publication Date
DK3011029T3 true DK3011029T3 (da) 2020-03-16

Family

ID=51059658

Family Applications (1)

Application Number Title Priority Date Filing Date
DK14734708.2T DK3011029T3 (da) 2013-06-17 2014-06-10 Administration, modificering og optimering af tandem-guidesystemer, fremgangsmåder og sammensætninger til sekvensmanipulering

Country Status (10)

Country Link
US (2) US11008588B2 (enExample)
EP (2) EP3011029B1 (enExample)
JP (2) JP6625971B2 (enExample)
KR (1) KR20160044457A (enExample)
CN (1) CN106062197A (enExample)
AU (1) AU2014281026B2 (enExample)
CA (1) CA2915834A1 (enExample)
DK (1) DK3011029T3 (enExample)
ES (1) ES2777217T3 (enExample)
WO (1) WO2014204724A1 (enExample)

Families Citing this family (299)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
PL3494997T3 (pl) * 2012-07-25 2020-04-30 The Broad Institute, Inc. Indukowalne białka wiążące dna i narzędzia perturbacji genomu oraz ich zastosowania
KR101656236B1 (ko) 2012-10-23 2016-09-12 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
ES2701749T3 (es) 2012-12-12 2019-02-25 Broad Inst Inc Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos
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
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
AU2013359199C1 (en) 2012-12-12 2021-06-17 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
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
AU2014228981B2 (en) 2013-03-15 2019-11-28 The General Hospital Corporation Using truncated guide RNAs (tru-gRNAs) to increase specificity for RNA-guided genome editing
SI2986729T1 (sl) 2013-04-16 2019-02-28 Regeneron Pharmaceuticals, Inc. Ciljana sprememba genoma podgane
RU2716421C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка, применение и применения в терапии систем crispr-cas и композиций для целенаправленного воздействия на нарушения и заболевания с использованием вирусных компонентов
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
SG11201510284XA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
AU2014281026B2 (en) 2013-06-17 2020-05-28 Massachusetts Institute Of Technology Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
EP3988649B1 (en) 2013-09-18 2024-11-27 Kymab Limited Methods, cells and organisms
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
US10584358B2 (en) 2013-10-30 2020-03-10 North Carolina State University Compositions and methods related to a type-II CRISPR-Cas system in Lactobacillus buchneri
EP4372090A3 (en) 2013-11-07 2024-08-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
CN110951779B (zh) 2013-12-11 2024-04-16 瑞泽恩制药公司 用于靶向修饰基因组的方法和组合物
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3080261B1 (en) 2013-12-12 2019-05-22 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
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
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
KR20250068794A (ko) * 2013-12-12 2025-05-16 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용
AU2014362245A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US10787654B2 (en) 2014-01-24 2020-09-29 North Carolina State University Methods and compositions for sequence guiding Cas9 targeting
EP3514246B1 (en) 2014-02-27 2021-11-17 The Broad Institute, Inc. T cell balance gene expression and methods of use thereof
EP3129490A4 (en) 2014-04-08 2017-10-04 North Carolina State University Methods and compositions for rna-directed repression of transcription using crispr-associated genes
EP4079847A1 (en) 2014-07-30 2022-10-26 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
DK3180426T3 (da) * 2014-08-17 2020-03-30 Broad Inst Inc Genomredigering ved anvendelse af cas9-nickaser
EP3633032A3 (en) 2014-08-28 2020-07-29 North Carolina State University Novel cas9 proteins and guiding features for dna targeting and genome editing
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
CA2963315A1 (en) 2014-10-15 2016-04-21 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
WO2016069591A2 (en) 2014-10-27 2016-05-06 The Broad Institute Inc. Compositions, methods and use of synthetic lethal screening
US10725041B2 (en) * 2014-11-04 2020-07-28 Versiti Blood Research Institute Foundation, Inc. Method to bioengineer designer platelets using gene editing and stem cell methodologies
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
BR112017010547A2 (pt) 2014-11-21 2018-02-27 Regeneron Pharma métodos para produção de uma modificação bialélica, modificação de um genoma dentro de uma célula, produção de uma geração f0 de um animal não humano e identificação de uma inserção de um inserto de ácido nucleico.
US11168369B2 (en) 2014-11-25 2021-11-09 The Brigham And Women's Hospital, Inc. Method of identifying and treating a person having a predisposition to or afflicted with a cardiometabolic disease
KR102763527B1 (ko) 2014-12-03 2025-02-05 애질런트 테크놀로지스, 인크. 화학적 변형을 갖는 가이드 rna
WO2016094679A1 (en) 2014-12-10 2016-06-16 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3985115A1 (en) 2014-12-12 2022-04-20 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)
WO2016094872A1 (en) 2014-12-12 2016-06-16 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
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology 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
US11339399B2 (en) 2014-12-31 2022-05-24 Viridos, Inc. Compositions and methods for high efficiency in vivo genome editing
US9650617B2 (en) * 2015-01-28 2017-05-16 Pioneer Hi-Bred International. Inc. CRISPR hybrid DNA/RNA polynucleotides and methods of use
WO2016130697A1 (en) * 2015-02-11 2016-08-18 Memorial Sloan Kettering Cancer Center Methods and kits for generating vectors that co-express multiple target molecules
WO2016138488A2 (en) 2015-02-26 2016-09-01 The Broad Institute Inc. T cell balance gene expression, compositions of matters and methods of use thereof
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
KR102648489B1 (ko) 2015-04-06 2024-03-15 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
GB201506509D0 (en) 2015-04-16 2015-06-03 Univ Wageningen Nuclease-mediated genome editing
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
WO2016186772A2 (en) * 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
KR102451796B1 (ko) 2015-05-29 2022-10-06 노쓰 캐롤라이나 스테이트 유니버시티 크리스퍼 핵산을 사용하여 박테리아, 고세균, 조류 및 효모를 스크리닝하는 방법
EP3919622A1 (en) 2015-06-01 2021-12-08 Temple University - Of The Commonwealth System of Higher Education Methods and compositions for rna-guided treatment of hiv infection
JP7396783B2 (ja) 2015-06-09 2023-12-12 エディタス・メディシン、インコーポレイテッド 移植を改善するためのｃｒｉｓｐｒ／ｃａｓ関連方法および組成物
DK3307872T3 (da) 2015-06-15 2023-10-23 Univ North Carolina State Fremgangsmåder og sammensætninger til effektiv indgivelse af nukleinsyrer og rna-baserede antimikrober
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
MX392008B (es) 2015-06-18 2025-03-21 Broad Inst Inc Mutaciones de la enzima crispr que reducen los efectos fuera del blanco
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
EP3436575A1 (en) * 2015-06-18 2019-02-06 The Broad Institute Inc. Novel 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
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
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
CA2990699A1 (en) 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
WO2017023801A1 (en) 2015-07-31 2017-02-09 Regents Of The University Of Minnesota Intracellular genomic transplant and methods of therapy
WO2017023974A1 (en) * 2015-08-03 2017-02-09 President And Fellows Of Harvard College Cas9 genome editing and transcriptional regulation
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
AU2016308339A1 (en) 2015-08-18 2018-04-12 Baylor College Of Medicine Methods and compositions for altering function and structure of chromatin loops and/or domains
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
ES2959815T3 (es) * 2015-09-21 2024-02-28 Arcturus Therapeutics Inc Edición génica selectiva de alelo y usos de la misma
AU2016326711B2 (en) 2015-09-24 2022-11-03 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/Cas-mediated genome editing
US11286480B2 (en) 2015-09-28 2022-03-29 North Carolina State University Methods and compositions for sequence specific antimicrobials
US12241053B2 (en) 2015-10-09 2025-03-04 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
US12234454B2 (en) 2015-10-22 2025-02-25 The Broad Institute, Inc. Crispr enzymes and systems
IL294014B2 (en) 2015-10-23 2024-07-01 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
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
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
WO2017075265A1 (en) 2015-10-28 2017-05-04 The Broad Institute, Inc. Multiplex analysis of single cell constituents
EP3368689B1 (en) 2015-10-28 2020-06-17 The Broad Institute, Inc. Composition for modulating immune responses by use of immune cell gene signature
WO2017075294A1 (en) 2015-10-28 2017-05-04 The Board Institute Inc. Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction
WO2017087708A1 (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
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2017112620A1 (en) 2015-12-22 2017-06-29 North Carolina State University Methods and compositions for delivery of crispr based antimicrobials
AU2016382512A1 (en) 2015-12-30 2018-07-12 Novartis Ag Immune effector cell therapies with enhanced efficacy
KR102811061B1 (ko) * 2016-01-26 2025-05-21 세다르스-신나이 메디칼 센터 생체내 이중 재조합효소-매개된 카세트 교환 (dRMCE)을 위한 시스템과 방법 및 이들의 질환 모형
US20190144942A1 (en) 2016-02-22 2019-05-16 Massachusetts Institute Of Technology Methods for identifying and modulating immune phenotypes
US11427861B2 (en) 2016-03-17 2022-08-30 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017180694A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Cas9 fusion molecules gene editing systems, and methods of use thereof
EP3445856A1 (en) * 2016-04-19 2019-02-27 The Broad Institute Inc. Novel crispr enzymes and systems
US11286478B2 (en) 2016-04-19 2022-03-29 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
KR102670601B1 (ko) 2016-04-19 2024-05-29 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
IL262888B1 (en) 2016-05-20 2025-10-01 Regeneron Pharma Methods for breaking immunological tolerance using multiple guide RNAs
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
AU2017283713B2 (en) 2016-06-17 2021-04-08 Massachusetts Institute Of Technology Type VI CRISPR orthologs and systems
AU2017295720B2 (en) 2016-07-13 2021-07-22 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
US11608570B2 (en) * 2016-07-29 2023-03-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Targeted in situ protein diversification by site directed DNA cleavage and repair
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018035364A1 (en) 2016-08-17 2018-02-22 The Broad Institute Inc. Product and methods useful for modulating and evaluating immune responses
CN110114461A (zh) 2016-08-17 2019-08-09 博德研究所 新型crispr酶和系统
US12431216B2 (en) 2016-08-17 2025-09-30 Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
KR102594051B1 (ko) * 2016-08-20 2023-10-26 아벨리노 랩 유에스에이, 인크. 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US20190262399A1 (en) 2016-09-07 2019-08-29 The Broad Institute, Inc. Compositions and methods for evaluating and modulating immune responses
US12188059B2 (en) 2016-09-13 2025-01-07 The Broad Institute, Inc. Proximity-dependent biotinylation and uses thereof
EP3516056B1 (en) * 2016-09-23 2024-11-27 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
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
EP4338799A3 (en) 2016-10-18 2024-06-05 Regents of the University of Minnesota Tumor infiltrating lymphocytes and methods of therapy
EP3535396A1 (en) 2016-11-01 2019-09-11 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
US12331086B2 (en) * 2016-11-14 2025-06-17 Toolgen Incorporated Artificially engineered SC function control system
US12404514B2 (en) 2016-12-09 2025-09-02 The Broad Institute, Inc. CRISPR-systems for modifying a trait of interest in a plant
WO2018119010A1 (en) 2016-12-19 2018-06-28 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
EP4095263A1 (en) 2017-01-06 2022-11-30 Editas Medicine, Inc. Methods of assessing nuclease cleavage
WO2018129486A2 (en) * 2017-01-06 2018-07-12 President And Fellows Of Harvard College Composition and methods for enhanced knock-in reporter gene expression
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
CN110312797A (zh) * 2017-02-10 2019-10-08 齐默尔根公司 组装和编辑用于多个宿主的多个dna构建体的模块化通用质粒设计策略
GB201702847D0 (en) * 2017-02-22 2017-04-05 Cancer Res Tech Ltd Cell labelling, tracking and retrieval
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
KR102454284B1 (ko) 2017-03-15 2022-10-12 더 브로드 인스티튜트, 인코퍼레이티드 신규 cas13b 오르소로그 crispr 효소 및 시스템
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
KR102842382B1 (ko) 2017-03-24 2025-08-04 큐어백 에스이 Crispr-연관 단백질을 암호화하는 핵산 및 이의 용도
WO2018175924A1 (en) 2017-03-24 2018-09-27 The Broad Institute, Inc. Methods and compositions for regulating innate lymphoid cell inflammatory responses
WO2018183908A1 (en) 2017-03-31 2018-10-04 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
CA3059757A1 (en) 2017-04-12 2018-10-18 The Broad Institute, Inc. Novel type vi crispr orthologs and systems
EP3610266A4 (en) 2017-04-12 2021-04-21 Massachusetts Eye and Ear Infirmary TUMOR SIGNATURE FOR METASTASIS, COMPOSITIONS OF MATERIAL AND THEIR METHODS OF USE
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
EP3612629A1 (en) 2017-04-18 2020-02-26 The Broad Institute, Inc. Compositions for detecting secretion and methods of use
WO2018195486A1 (en) 2017-04-21 2018-10-25 The Broad Institute, Inc. Targeted delivery to beta cells
WO2018201086A1 (en) 2017-04-28 2018-11-01 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
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
WO2018213708A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US12297436B2 (en) 2017-05-18 2025-05-13 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US20200263186A1 (en) 2017-05-26 2020-08-20 North Carolina State University Altered guide rnas for modulating cas9 activity and methods of use
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
AU2018279829B2 (en) 2017-06-09 2024-01-04 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
KR102761791B1 (ko) 2017-06-26 2025-02-05 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법
EP3645021A4 (en) 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
EP3645721A1 (en) 2017-06-30 2020-05-06 Novartis AG Methods for the treatment of disease with gene editing systems
WO2019014390A1 (en) * 2017-07-12 2019-01-17 Refuge Biotechnologies, Inc. METHODS AND SYSTEMS FOR CONDITIONAL REGULATION OF GENE EXPRESSION
WO2019014581A1 (en) 2017-07-14 2019-01-17 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODULATING THE ACTIVITY OF A CYTOTOXIC LYMPHOCYTE
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
US12105089B2 (en) 2017-07-17 2024-10-01 The Broad Institute, Inc. Cell atlas of the healthy and ulcerative colitis human colon
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)
CN111278848B (zh) 2017-08-04 2023-06-27 北京大学 特异性识别甲基化修饰dna碱基的tale rvd及其应用
WO2019028686A1 (zh) 2017-08-08 2019-02-14 北京大学 基因敲除方法
US11286468B2 (en) 2017-08-23 2022-03-29 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
JP2020535802A (ja) 2017-09-21 2020-12-10 ザ・ブロード・インスティテュート・インコーポレイテッド 標的化核酸編集のための系、方法、及び組成物
US12043870B2 (en) 2017-10-02 2024-07-23 The Broad Institute, Inc. Methods and compositions for detecting and modulating an immunotherapy resistance gene signature in cancer
EP3692152B1 (en) 2017-10-04 2025-09-03 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
US11680296B2 (en) 2017-10-16 2023-06-20 Massachusetts Institute Of Technology Mycobacterium tuberculosis host-pathogen interaction
AU2018351072B9 (en) 2017-10-20 2024-09-05 Fred Hutchinson Cancer Center Systems and methods to produce B cells genetically modified to express selected antibodies
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
US11547614B2 (en) 2017-10-31 2023-01-10 The Broad Institute, Inc. Methods and compositions for studying cell evolution
WO2019087113A1 (en) 2017-11-01 2019-05-09 Novartis Ag Synthetic rnas and methods of use
WO2019094955A1 (en) 2017-11-13 2019-05-16 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
JP7522656B2 (ja) 2017-11-21 2024-07-25 クリスパー セラピューティクス アーゲー 常染色体優性網膜色素変性の処置のための材料および方法
WO2019113506A1 (en) 2017-12-07 2019-06-13 The Broad Institute, Inc. Methods and compositions for multiplexing single cell and single nuclei sequencing
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes 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
IL276080B2 (en) 2018-01-17 2023-10-01 Vertex Pharma Dna-pk inhibitor compounds, compositions comprising same and uses thereof
AU2019209292B2 (en) 2018-01-17 2023-10-05 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
US12121524B2 (en) 2018-01-17 2024-10-22 Vertex Pharmaceuticals Incorporated DNA-PK inhibitors
US12258571B2 (en) 2018-03-16 2025-03-25 Cedars-Sinai Medical Center Methods and compositions for inducible expression of neurotrophic factors
CN108424934A (zh) * 2018-04-16 2018-08-21 和元生物技术（上海）股份有限公司 一种慢病毒cag-cmv双启动子改造载体构建及应用
US12338486B2 (en) 2018-04-19 2025-06-24 Massachusetts Institute Of Technology Single-stranded break detection in double-stranded DNA
DK3560330T3 (da) 2018-04-24 2022-07-11 Kws Saat Se & Co Kgaa Planter med forbedret fordøjelighed og markørhaplotyper
US11957695B2 (en) 2018-04-26 2024-04-16 The Broad Institute, Inc. Methods and compositions targeting glucocorticoid signaling for modulating immune responses
US12281301B2 (en) 2018-04-27 2025-04-22 The Broad Institute, Inc. Sequencing-based proteomics
US20210386829A1 (en) 2018-05-04 2021-12-16 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
CN108846258B (zh) * 2018-06-08 2021-05-18 中国人民解放军军事科学院军事医学研究院 一种自动检测分节段rna病毒重配的方法
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
WO2019237399A1 (zh) * 2018-06-16 2019-12-19 深圳市博奥康生物科技有限公司 CRISPR-Cas9特异性敲除人C2orf40基因的方法及其特异的sgRNA
SG11202012786RA (en) 2018-06-26 2021-01-28 Massachusetts Inst Technology Crispr effector system based amplification methods, systems, and diagnostics
MX2020013778A (es) 2018-06-26 2021-05-27 Broad Inst Inc Composiciones, sistemas y metodos de amplificacion basados en crispr/cas y transposasa.
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
SG11202102068TA (en) 2018-07-31 2021-03-30 Broad Inst Inc Novel crispr enzymes and systems
WO2020033601A1 (en) 2018-08-07 2020-02-13 The Broad Institute, Inc. Novel cas12b enzymes and systems
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
US20210324357A1 (en) 2018-08-20 2021-10-21 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
WO2020051507A1 (en) 2018-09-06 2020-03-12 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
US12398402B2 (en) 2018-09-12 2025-08-26 Fred Hutchinson Cancer Center Reducing CD33 expression to selectively protect therapeutic cells
JP7344300B2 (ja) 2018-09-18 2023-09-13 ブイエヌブイ ニューコ インク． Ａｒｃベースのカプシドおよびその使用
US12203123B2 (en) 2018-10-01 2025-01-21 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for screening for variant cells
US12264330B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for killing target cells
US12264313B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for genome modification and alteration of expression
US10711267B2 (en) 2018-10-01 2020-07-14 North Carolina State University Recombinant type I CRISPR-Cas system
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
WO2020081730A2 (en) 2018-10-16 2020-04-23 Massachusetts Institute Of Technology Methods and compositions for modulating microenvironment
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020097360A1 (en) * 2018-11-07 2020-05-14 The Regents Of The University Of Colorado, A Body Corporate Methods and compositions for genome-wide analysis and use of genome cutting and repair
US12165743B2 (en) 2018-11-09 2024-12-10 The Broad Institute, Inc. Compressed sensing for screening and tissue imaging
CA3124110A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
US20220062394A1 (en) 2018-12-17 2022-03-03 The Broad Institute, Inc. Methods for identifying neoantigens
CN111349654B (zh) * 2018-12-20 2023-01-24 北京大学 使用加标签的向导rna构建体进行高效基因筛选的组合物和方法
WO2020125762A1 (en) * 2018-12-20 2020-06-25 Peking University Compositions and methods for highly efficient genetic screening using barcoded guide rna constructs
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
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
US20220162649A1 (en) 2019-04-01 2022-05-26 The Broad Institute, Inc. Novel nucleic acid modifiers
CN111812066B (zh) * 2019-04-10 2023-04-28 华东理工大学 基于CRISPR/Cas12a系统的生物传感器、试剂盒及其在小分子检测中的用途
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20220243287A1 (en) 2019-05-13 2022-08-04 KWS SAAT SE & Co. KGaA Drought tolerance in corn
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
US20220243178A1 (en) 2019-05-31 2022-08-04 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
CN112168808B (zh) * 2019-07-02 2023-01-24 深圳市第二人民医院 一种基于crispr的细胞核靶向药物递送系统
SG11202111943UA (en) 2019-07-02 2021-11-29 Hutchinson Fred Cancer Res Recombinant ad35 vectors and related gene therapy improvements
JPWO2021020550A1 (enExample) * 2019-08-01 2021-02-04
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
US20220298501A1 (en) 2019-08-30 2022-09-22 The Broad Institute, Inc. Crispr-associated mu transposase systems
CN115175996A (zh) 2019-09-20 2022-10-11 博德研究所 新颖vi型crispr酶和系统
WO2021061832A1 (en) * 2019-09-23 2021-04-01 Regents Of The University Of Minnesota Genetically-edited immune cells and methods of therapy
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
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
US20230357788A1 (en) 2019-10-17 2023-11-09 KWS SAAT SE & Co. KGaA Enhanced disease resistance of crops by downregulation of repressor genes
CN115176001A (zh) * 2019-12-11 2022-10-11 因特利亚治疗公司 用于基因编辑的修饰的引导rna
WO2021151085A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Crispr-cas enzymes with enhanced on-target activity
WO2021151073A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants
EP3872190A1 (en) 2020-02-26 2021-09-01 Antibodies-Online GmbH A method of using cut&run or cut&tag to validate crispr-cas targeting
GB2614813B (en) 2020-05-08 2025-05-07 Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
JP2023527446A (ja) 2020-05-29 2023-06-28 カー・ヴェー・エス ザート エス・エー ウント コー． カー・ゲー・アー・アー 植物単数体の誘導
EP4165198A4 (en) * 2020-06-12 2024-10-23 Flagship Pioneering Innovations V, Inc. TANDEM ANELLOVIRUS CONSTRUCTS
CN111979266A (zh) * 2020-08-28 2020-11-24 江苏集萃药康生物科技有限公司 一种pcsk9人源化小鼠模型的构建方法及其应用
EP4232925A1 (en) * 2020-10-21 2023-08-30 Verint Americas Inc. System and method of automated determination of use of sensitive information and corrective action for improper use
EP4001429A1 (en) 2020-11-16 2022-05-25 Antibodies-Online GmbH Analysis of crispr-cas binding and cleavage sites followed by high-throughput sequencing (abc-seq)
CN114606196A (zh) * 2020-12-04 2022-06-10 南京大学 一种进行siRNA表达和体内递送的细胞疗法
CN112899306B (zh) * 2020-12-21 2023-06-30 南京启真基因工程有限公司 Crispr系统及其在构建gabrg2基因突变的克隆猪核供体细胞中的应用
WO2022170059A1 (en) 2021-02-05 2022-08-11 Christiana Care Health Services, Inc. Methods of and compositions for reducing gene expression and/or activity
US20230052518A1 (en) 2021-07-12 2023-02-16 Labsimply, Inc. Nuclease cascade assay
CA3221517A1 (en) 2021-07-30 2023-02-02 Monika KLOIBER-MAITZ Plants with improved digestibility and marker haplotypes
WO2023019164A2 (en) * 2021-08-11 2023-02-16 The Board Of Trustees Of The Leland Stanford Junior University High-throughput precision genome editing in human cells
KR20240055811A (ko) 2021-09-10 2024-04-29 애질런트 테크놀로지스, 인크. 프라임 편집을 위한 화학적 변형을 갖는 가이드 rna
KR20240099393A (ko) 2021-11-01 2024-06-28 톰 바이오사이언시스, 인코포레이티드 유전자 편집 기구와 핵산 카고의 동시 전달을 위한 단일 작제물 플랫폼
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
WO2023114052A1 (en) 2021-12-13 2023-06-22 Labsimply, Inc. Tuning cascade assay kinetics via molecular design
US20230279375A1 (en) 2021-12-13 2023-09-07 Labsimply, Inc. Signal boost cascade assay
IL313765A (en) 2021-12-22 2024-08-01 Tome Biosciences Inc Joint provision of a gene editor structure and a donor template
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
WO2024020352A1 (en) * 2022-07-18 2024-01-25 Vertex Pharmaceuticals Incorporated Tandem guide rnas (tg-rnas) and their use in genome editing
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
EP4577660A1 (en) 2022-08-26 2025-07-02 KWS SAAT SE & Co. KGaA Use of paired genes in hybrid breeding
US12091689B2 (en) * 2022-09-30 2024-09-17 Vedabio, Inc. Delivery of therapeutics in vivo via a CRISPR-based cascade system
WO2024081156A1 (en) 2022-10-14 2024-04-18 Vedabio, Inc. Detection of nucleic acid and non-nucleic acid target molecules
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024253707A2 (en) 2023-01-07 2024-12-12 Vedabio, Inc. Engineered nucleic acid-guided nucleases
US12060602B2 (en) 2023-01-10 2024-08-13 Vedabio, Inc. Sample splitting for multiplexed detection of nucleic acids without amplification
WO2024220741A1 (en) 2023-04-19 2024-10-24 Flagship Pioneering Innovations Vii, Llc Compositions and methods for the production of libraries
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
TW202509220A (zh) 2023-06-26 2025-03-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 經工程化之瘧原蟲及相關方法
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
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

Family Cites Families (165)

* 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
DE122007000007I1 (de) 1986-04-09 2007-05-16 Genzyme Corp Genetisch transformierte Tiere, die ein gewünschtes Protein in Milch absondern
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
JPH10510433A (ja) 1995-06-06 1998-10-13 アイシス・ファーマシューティカルス・インコーポレーテッド 高いキラル純度のホスホロチオエート結合を有するオリゴヌクレオチド
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
NZ520579A (en) 1997-10-24 2004-08-27 Invitrogen Corp Recombinational cloning using nucleic acids having recombination sites and methods for synthesizing double stranded nucleic acids
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
US7033744B2 (en) 2001-03-16 2006-04-25 Naoya Kobayashi Method for proliferating a liver cell, a liver cell obtained thereby, and use thereof
DK1385538T3 (da) 2001-04-05 2013-04-29 Univ Johns Hopkins Kimære vacciner omfattende det lumenale domæne fra LAMP-1 eller LAMP-2
CA2921821A1 (en) 2001-07-12 2003-01-23 University Of Massachusetts In vivo production of small interfering rnas that mediate gene silencing
AU2002327430A1 (en) 2001-08-08 2003-02-24 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
AU2003224897A1 (en) 2002-04-09 2003-10-27 Kenneth L. Beattie Oligonucleotide probes for genosensor chips
US20070020627A1 (en) 2002-06-11 2007-01-25 The Scripps Research Institute Artificial transcription factors
WO2004029219A2 (en) 2002-09-27 2004-04-08 Cold Spring Harbor Laboratory Cell-based rna interference and related 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
EP1644534A4 (en) 2003-07-03 2006-08-09 Univ California GENOMKARTING OF FUNCTIONAL DNA ELEMENTS AND CELLULAR PROTEINS
EP2927318B1 (en) * 2003-08-08 2020-05-20 Sangamo Therapeutics, Inc. Methods and compositions for targeted cleavage and recombination
WO2005035718A2 (en) 2003-10-03 2005-04-21 Welgen, Inc. Biderectional promoters for small rna expression
FR2862659B1 (fr) 2003-11-21 2006-02-10 Pasteur Institut Genome de legionella pneumophila souche paris- applications diagnostiques et epidemiologiques
US8053232B2 (en) 2004-01-23 2011-11-08 Virxsys Corporation 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
CA2573702C (en) 2004-07-16 2013-10-15 The Government Of The United States Of America As Represented By The Sec Retary Of The Department Of Health And Human Services Vaccine constructs and combination of vaccines designed to improve the breadth of the immune response to diverse strains and clades of hiv
AU2006239169A1 (en) 2005-04-28 2006-11-02 Benitec, Limited. Multiple-RNAi expression cassettes for simultaneous delivery of RNAi agents related to heterozygotic expression patterns
US7892224B2 (en) 2005-06-01 2011-02-22 Brainlab Ag Inverse catheter planning
SG10201508995QA (en) 2005-07-26 2015-11-27 Sangamo Biosciences Inc Targeted integration and expression of exogenous nucleic acid sequences
DK3284833T3 (da) 2005-08-26 2022-02-07 Dupont Nutrition Biosci Aps Anvendelse af crispr-associerede gener (cas)
EP1989307B1 (en) * 2006-02-08 2012-08-08 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
EP1994182B1 (en) 2006-03-15 2019-05-29 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
EP2584047B1 (en) 2006-05-11 2014-11-19 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
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
WO2008108989A2 (en) 2007-03-02 2008-09-12 Danisco A/S Cultures with improved phage resistance
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
CA2711179A1 (en) 2007-12-31 2009-07-16 Nanocor Therapeutics, Inc. Rna interference for the treatment of heart failure
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
WO2010011961A2 (en) 2008-07-25 2010-01-28 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
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
MX2011004859A (es) 2008-11-07 2011-08-03 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
US20110016540A1 (en) 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
AU2009322964B2 (en) 2008-12-04 2014-10-09 Sangamo Therapeutics, 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
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
WO2011036510A1 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease variants cleaving the genome of the herpes simplex virus and uses thereof
WO2010143917A2 (en) 2009-06-11 2010-12-16 Toolgen Incorporation Targeted genomic rearrangements using site-specific nucleases
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
US9234016B2 (en) 2009-07-28 2016-01-12 Sangamo Biosciences, Inc. Engineered zinc finger proteins for treating trinucleotide repeat disorders
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
US8927807B2 (en) 2009-09-03 2015-01-06 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
DE112010004582T5 (de) 2009-11-27 2012-11-29 Basf Plant Science Company Gmbh Optimierte Endonukleasen und Anwendungen davon
JP2013515697A (ja) 2009-12-23 2013-05-09 マックスプランク−ゲセルシャフト・ツール・フェーデルング・デル・ヴィッセンシャフテン・エー・ファウ インフルエンザ標的
CA2788850C (en) 2010-02-09 2019-06-25 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
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
CA2798703A1 (en) 2010-05-10 2011-11-17 The Regents Of The University Of California Endoribonuclease compositions and methods of use thereof
KR20180121665A (ko) * 2010-07-23 2018-11-07 시그마-알드리치 컴퍼니., 엘엘씨 표적화 엔도뉴클레아제 및 단일-가닥 핵산을 사용하는 게놈 편집
WO2012018697A1 (en) 2010-08-02 2012-02-09 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
JP6018069B2 (ja) 2010-10-12 2016-11-02 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ 血友病ｂを治療する方法及び組成物
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
MX349138B (es) 2011-04-22 2017-07-13 Univ California Variones de virus adeno-asociados con capside variante y sus metodos de uso.
PT2702160T (pt) 2011-04-27 2020-07-30 Amyris Inc Métodos para modificação genómica
US20140113376A1 (en) 2011-06-01 2014-04-24 Rotem Sorek Compositions and methods for downregulating prokaryotic genes
CA3186126A1 (en) 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
AU2012318562A1 (en) 2011-10-06 2014-04-10 Sangamo Therapeutics, 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
US9708414B2 (en) 2011-11-18 2017-07-18 UNIVERSITé LAVAL Methods and products for increasing frataxin levels and uses thereof
AU2012340567B2 (en) 2011-11-22 2017-11-23 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
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
EP2820159B1 (en) 2012-02-29 2019-10-23 Sangamo Therapeutics, Inc. Methods and compositions for treating huntington's disease
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
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
HK1208823A1 (en) 2012-06-01 2016-03-18 德雷塞尔大学 Modulation of hepatitis b virus cccdna transcription
WO2014059173A2 (en) 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
KR101656236B1 (ko) 2012-10-23 2016-09-12 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
ES2713243T3 (es) 2012-12-06 2019-05-20 Sigma Aldrich Co Llc Modificación y regulación del genoma basada en 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
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
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
PL2931898T3 (pl) 2012-12-12 2016-09-30 Le Cong Projektowanie i optymalizacja systemów, sposoby i kompozycje do manipulacji sekwencją z domenami funkcjonalnymi
ES2701749T3 (es) 2012-12-12 2019-02-25 Broad Inst Inc Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos
IL307735A (en) 2012-12-12 2023-12-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
DK2921557T3 (en) 2012-12-12 2016-11-07 Broad Inst Inc Design of systems, methods and optimized sequence manipulation guide compositions
AU2013359199C1 (en) 2012-12-12 2021-06-17 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
EP4481048A3 (en) 2012-12-17 2025-02-26 President and Fellows of Harvard College Rna-guided human genome engineering
AU2014207618A1 (en) 2013-01-16 2015-08-06 Emory University Cas9-nucleic acid complexes and uses related thereto
US10660943B2 (en) 2013-02-07 2020-05-26 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
ES2901396T3 (es) 2013-03-14 2022-03-22 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico
AU2014228981B2 (en) 2013-03-15 2019-11-28 The General Hospital Corporation Using truncated guide RNAs (tru-gRNAs) to increase specificity for RNA-guided genome editing
PL2979143T3 (pl) 2013-03-27 2018-02-28 Wilco Ag Sposób badania w linii i/lub testowania w linii urządzeń i aparatura do przeprowadzania tego sposobu
WO2014165349A1 (en) 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
JP7065564B2 (ja) 2013-05-29 2022-05-12 セレクティス Ｃａｓ９ニッカーゼ活性を用いて正確なｄｎａ切断をもたらすための方法
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
KR102884236B1 (ko) 2013-06-04 2025-11-12 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Rna-가이드된 전사 조절
ES2991293T3 (es) 2013-06-05 2024-12-03 Univ Duke Edición génica guiada por ARN y regulación génica
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
SG11201510284XA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
RU2716421C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка, применение и применения в терапии систем crispr-cas и композиций для целенаправленного воздействия на нарушения и заболевания с использованием вирусных компонентов
CA2915845A1 (en) 2013-06-17 2014-12-24 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
AU2014281026B2 (en) 2013-06-17 2020-05-28 Massachusetts Institute Of Technology Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
SMT202100691T1 (it) 2013-07-11 2022-01-10 Modernatx Inc Composizioni 5 comprendenti polinucleotidi sintetici che codificano proteine correlate a crispr e sgrna sintetici e metodi d'uso
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
US9925248B2 (en) 2013-08-29 2018-03-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
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
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
EP4372090A3 (en) 2013-11-07 2024-08-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
EP3071695A2 (en) 2013-11-18 2016-09-28 Crispr Therapeutics AG Crispr-cas system materials and methods
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
KR20160097327A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 유전자 산물, 구조 정보 및 유도성 모듈형 ｃａｓ 효소의 발현의 변경을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 방법
AU2014362245A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
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
WO2015113063A1 (en) 2014-01-27 2015-07-30 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
DK3180426T3 (da) 2014-08-17 2020-03-30 Broad Inst Inc Genomredigering ved anvendelse af cas9-nickaser
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
MX392008B (es) 2015-06-18 2025-03-21 Broad Inst Inc Mutaciones de la enzima crispr que reducen los efectos fuera del blanco
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

Also Published As

Publication number Publication date
WO2014204724A8 (en) 2015-12-23
ES2777217T3 (es) 2020-08-04
AU2014281026B2 (en) 2020-05-28
WO2014204724A1 (en) 2014-12-24
KR20160044457A (ko) 2016-04-25
JP2020054354A (ja) 2020-04-09
AU2014281026A1 (en) 2016-01-28
CN106062197A (zh) 2016-10-26
US20160186213A1 (en) 2016-06-30
US11008588B2 (en) 2021-05-18
JP2016521993A (ja) 2016-07-28
WO2014204724A9 (en) 2016-03-24
US20210292794A1 (en) 2021-09-23
AU2014281026A9 (en) 2016-06-16
EP3011029B1 (en) 2019-12-11
EP3674411A1 (en) 2020-07-01
HK1223646A1 (en) 2017-08-04
EP3011029A1 (en) 2016-04-27
JP6625971B2 (ja) 2019-12-25
CA2915834A1 (en) 2014-12-24

Similar Documents

Publication Publication Date Title
DK3011029T3 (da) Administration, modificering og optimering af tandem-guidesystemer, fremgangsmåder og sammensætninger til sekvensmanipulering
DK3327127T3 (da) Fremføring, modificering og optimering af systemer, fremgangsmåder og sammensætninger til sekvensmanipulation og terapeutiske anvendelser
DK3064585T3 (da) Konstruering og optimering af forbedrede systemer, fremgangsmåder og enzymsammensætninger til sekvensmanipulation
DK3656557T3 (da) System og arbejdsstation til design, fabrikation og fremstilling af biomaterielle konstruktioner
DK3252160T3 (da) Systemer, fremgangsmåder og sammensætninger med crispr-cas-bestanddele til sekvensmanipulation
DK2970456T3 (da) Fremgangsmåder og sammensætninger til levering af mrna-kodede antistoffer
DK3611262T3 (da) Fremgangsmåder til sekvensering af immunrepertoire
DK2970872T3 (da) Mikroorganismer og fremgangsmåder til fremstilling af sialylerede og N-acetylglucosamin-holdige oligosaccharider
DK3139986T3 (da) System til administration af terapeutisk gas
DK2996556T3 (da) System til billedstyret procedure
DK3291679T3 (da) Ændring af mikrobielle populationer og modificering af mikrobiota
DK3556869T3 (da) Sammensætninger og fremgangsmåder til sekventering af polynukleotider
DK2943192T3 (da) Fremgangsmåder til behandling af pancreascancer, hvilke fremgangsmåder omfatter paclitaxel og gemcitabin
DK2964315T3 (da) System til behandling af neuromotorisk dysfunktion
DK2954051T3 (da) Modificeret kapsid til genoverførsel til behandling af nethinden
DK3008168T3 (da) Sc-celler og sammensætninger og fremgangsmåde til dannelse deraf
EP3397250C0 (en) LONG-TERM TRANSDERMAL DELIVERY SYSTEMS
DK2958900T3 (da) 2-aminopyrimidinderivater til behandling af virusinfektioner
DK3019503T3 (da) Polycykliske-carbamoylpyridon-forbindelser og anvendelse deraf til behandling af hiv-infektioner
PL2996514T3 (pl) Układ przesuwny
DK3404106T3 (da) Ekspressionssystem til selektiv genterapi
DK3300610T3 (da) Fremgangsmåde til fraktionering af opløselige ærter
DK2994533T3 (da) Fremgangsmåder og sammensætninger til behandling af cancer
DK3080281T3 (da) System til administration af mikrobiske inokulanter og relaterede materialer og fremgangsmåder
HUE042285T2 (hu) Bemenet lezáró rendszer