[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

SG11201703747RA - METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs - Google Patents

METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs

Info

Publication number
SG11201703747RA
SG11201703747RA SG11201703747RA SG11201703747RA SG11201703747RA SG 11201703747R A SG11201703747R A SG 11201703747RA SG 11201703747R A SG11201703747R A SG 11201703747RA SG 11201703747R A SG11201703747R A SG 11201703747RA SG 11201703747R A SG11201703747R A SG 11201703747RA
Authority
SG
Singapore
Prior art keywords
compositions
methods
genetic modification
guide rnas
targeted genetic
Prior art date
Application number
SG11201703747RA
Inventor
Andrew J Murphy
David Frendewey
Ka-Man Venus Lai
Wojtek Auerbach
Gustavo Droguett
Anthony Gagliardi
David M Valenzuela
Vera Voronina
Lynn Macdonald
George D Yancopoulos
Original Assignee
Regeneron Pharma
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 Regeneron Pharma filed Critical Regeneron Pharma
Publication of SG11201703747RA publication Critical patent/SG11201703747RA/en

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
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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 RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6827—Hybridisation assays for detection of mutation or polymorphism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6888—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for detection or identification of organisms
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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 [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2537/00—Reactions characterised by the reaction format or use of a specific feature
    • C12Q2537/10—Reactions characterised by the reaction format or use of a specific feature the purpose or use of
    • C12Q2537/16—Assays for determining copy number or wherein the copy number is of special importance

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Environmental Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Husbandry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
SG11201703747RA 2014-11-21 2015-11-20 METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs SG11201703747RA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201462083005P 2014-11-21 2014-11-21
US201562182314P 2015-06-19 2015-06-19
US201562211421P 2015-08-28 2015-08-28
PCT/US2015/062023 WO2016081923A2 (en) 2014-11-21 2015-11-20 METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs

Publications (1)

Publication Number Publication Date
SG11201703747RA true SG11201703747RA (en) 2017-06-29

Family

ID=54771219

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10201913829YA SG10201913829YA (en) 2014-11-21 2015-11-20 METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs
SG11201703747RA SG11201703747RA (en) 2014-11-21 2015-11-20 METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SG10201913829YA SG10201913829YA (en) 2014-11-21 2015-11-20 METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs

Country Status (25)

Country Link
US (4) US10457960B2 (en)
EP (2) EP3221457B1 (en)
JP (2) JP6727199B2 (en)
KR (3) KR102415093B1 (en)
CN (2) CN107208078B (en)
AU (2) AU2015349692B2 (en)
BR (1) BR112017010547A2 (en)
CA (2) CA3176380A1 (en)
CY (1) CY1121738T1 (en)
DK (1) DK3221457T3 (en)
ES (1) ES2731437T3 (en)
HR (1) HRP20190949T1 (en)
HU (1) HUE044907T2 (en)
IL (3) IL301900A (en)
LT (1) LT3221457T (en)
MX (2) MX2017006670A (en)
NZ (1) NZ731962A (en)
PL (1) PL3221457T3 (en)
PT (1) PT3221457T (en)
RS (1) RS58893B1 (en)
RU (2) RU2734770C2 (en)
SG (2) SG10201913829YA (en)
SI (1) SI3221457T1 (en)
SM (1) SMT201900354T1 (en)
WO (1) WO2016081923A2 (en)

Families Citing this family (121)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
US20100069614A1 (en) 2008-06-27 2010-03-18 Merus B.V. Antibody producing non-human mammals
WO2004106375A1 (en) 2003-05-30 2004-12-09 Merus Biopharmaceuticals B.V. I.O. Fab library for the preparation of anti vegf and anti rabies virus fabs
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
WO2014130706A1 (en) 2013-02-20 2014-08-28 Regeneron Pharmaceuticals, Inc. Genetic modification of rats
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
DK3456831T3 (en) 2013-04-16 2021-09-06 Regeneron Pharma TARGETED MODIFICATION OF RAT GENOMES
US20150044192A1 (en) 2013-08-09 2015-02-12 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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
JP2016536021A (en) 2013-11-07 2016-11-24 エディタス・メディシン，インコーポレイテッド CRISPR-related methods and compositions with governing gRNA
MX2016007654A (en) 2013-12-11 2017-08-15 Regeneron Pharma Methods and compositions for the targeted modification of a genome.
US20150166985A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting von willebrand factor point mutations
SI3152312T1 (en) 2014-06-06 2020-07-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for modifying a targeted locus
AU2015279642B2 (en) 2014-06-26 2021-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
EP4079847A1 (en) 2014-07-30 2022-10-26 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
AU2015330699B2 (en) 2014-10-10 2021-12-02 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
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
EP3221457B1 (en) 2014-11-21 2019-03-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
SG11201704646YA (en) 2014-12-19 2017-07-28 Regeneron Pharma Methods and compositions for targeted genetic modification through single-step multiple targeting
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
AU2016261358B2 (en) 2015-05-11 2021-09-16 Editas Medicine, Inc. Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells
CN107920498A (en) 2015-05-29 2018-04-17 瑞泽恩制药公司 The non-human animal destroyed with C9ORF72 locus
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
EP3322804B1 (en) 2015-07-15 2021-09-01 Rutgers, The State University of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
US11667911B2 (en) 2015-09-24 2023-06-06 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/CAS-mediated genome editing
US11970710B2 (en) 2015-10-13 2024-04-30 Duke University Genome engineering with Type I CRISPR systems in eukaryotic cells
EP3365357B1 (en) 2015-10-23 2024-02-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing
EP3370513A1 (en) * 2015-11-06 2018-09-12 The Jackson Laboratory Large genomic dna knock-in and uses thereof
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
KR20230006929A (en) 2016-01-13 2023-01-11 리제너론 파마슈티칼스 인코포레이티드 Rodents having an engineered heavy chain diversity region
EP3413908B1 (en) * 2016-02-11 2024-06-19 The Regents of The University of California Methods and compositions for modifying a mutant dystrophin gene in a cell's genome
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
SG11201809552SA (en) * 2016-05-20 2018-11-29 Regeneron Pharma Methods for breaking immunological tolerance using multiple guide rnas
US20210010022A1 (en) * 2016-05-27 2021-01-14 Cambridge Enterprise Limited Novel nucleic acid construct
EP3472311A4 (en) * 2016-06-17 2020-03-04 Montana State University BIDIRECTIONAL TARGETING FOR GENOMEDITATION
ES2982085T3 (en) 2016-06-20 2024-10-14 Octapharma Ag Means and methods for modifying multiple alleles
EP3476865B1 (en) * 2016-06-28 2023-09-13 Biocytogen Pharmaceuticals (Beijing) Co., Ltd. Method for constructing pd-1 gene-modified humanized animal model and use thereof
JP7033583B2 (en) * 2016-07-13 2022-03-10 バーテックス ファーマシューティカルズ インコーポレイテッド Methods, compositions and kits for increasing genome editing efficiency
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
WO2017037304A2 (en) * 2016-07-28 2017-03-09 Dsm Ip Assets B.V. An assembly system for a eukaryotic cell
CN109803530A (en) 2016-07-29 2019-05-24 瑞泽恩制药公司 Mouse comprising causing the mutation of the truncated old information model expression of C-
IL308426A (en) 2016-08-03 2024-01-01 Harvard 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
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3518667B1 (en) 2016-09-30 2024-10-30 Regeneron Pharmaceuticals, Inc. Rodents having a hexanucleotide repeat expansion in a c9orf72 locus
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
RU2757665C2 (en) 2016-11-04 2021-10-20 Регенерон Фармасьютикалс, Инк. Animals other than humans with constructed locus of light lambda chain of immunoglobulin
CN106520831B (en) * 2016-11-18 2020-05-26 青岛市畜牧兽医研究所 Method for modifying mammalian genome
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
CN110520163A (en) 2017-01-05 2019-11-29 新泽西鲁特格斯州立大学 Independently of the target gene editing platform and application thereof of DNA double chain fracture
EP4095263A1 (en) 2017-01-06 2022-11-30 Editas Medicine, Inc. Methods of assessing nuclease cleavage
ES2969213T3 (en) 2017-02-15 2024-05-17 2Seventy Bio Inc Donor repair templates for multiplex genome editing
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592777A1 (en) 2017-03-10 2020-01-15 President and Fellows of Harvard College Cytosine to guanine base editor
EP3601562A1 (en) 2017-03-23 2020-02-05 President and Fellows of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2018213081A1 (en) * 2017-05-16 2018-11-22 Generos Biopharma Ltd. Compositions and methods for treating alzheimer's disease
CN110997908A (en) 2017-06-09 2020-04-10 爱迪塔斯医药公司 Engineered CAS9 nucleases
EP3644722B1 (en) 2017-06-27 2024-12-11 Regeneron Pharmaceuticals, Inc. Rodents comprising a humanized asgr1 locus
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
CN110891420B (en) 2017-07-31 2022-06-03 瑞泽恩制药公司 CAS transgenic mouse embryonic stem cells and mice and their applications
CN111182790A (en) 2017-07-31 2020-05-19 瑞泽恩制药公司 CRISPR reporter non-human animals and uses thereof
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR102544051B1 (en) 2017-09-29 2023-06-16 리제너론 파마슈티칼스 인코포레이티드 Non-human animals comprising humanized TTR loci and methods of use
WO2019072241A1 (en) 2017-10-13 2019-04-18 Beijing Biocytogen Co., Ltd Genetically modified non-human animal with human or chimeric pd-1
EP3697906A1 (en) 2017-10-16 2020-08-26 The Broad Institute, Inc. Uses of adenosine base editors
US20190141966A1 (en) 2017-11-10 2019-05-16 Regeneron Pharmaceuticals, Inc. Non-Human Animals Comprising SLC30A8 Mutation And Methods Of Use
AU2018375796B2 (en) 2017-11-30 2024-11-21 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized TRKB locus
WO2019113065A1 (en) 2017-12-05 2019-06-13 Regeneron Pharmaceuticals, Inc. Non-human animals having an engineered immunoglobulin lambda light chain and uses thereof
AU2019209293B2 (en) 2018-01-17 2023-07-27 Vertex Pharmaceuticals Incorporated DNA-PK inhibitors
BR112020014140A2 (en) 2018-01-17 2020-12-01 Vertex Pharmaceuticals Incorporated dna-pk inhibitors
US11643670B2 (en) 2018-01-29 2023-05-09 Massachusetts Institute Of Technology Methods of enhancing chromosomal homologous recombination
CN116349651A (en) 2018-03-19 2023-06-30 瑞泽恩制药公司 Transcription modulation of animals using CRISPR/CAS systems
WO2019189573A1 (en) 2018-03-29 2019-10-03 学校法人自治医科大学 Genome editing method, composition, cell, cell preparation, and method for producing cell preparation
US20240075164A1 (en) * 2018-04-30 2024-03-07 The Trustees Of The University Of Pennsylvania In utero crispr-mediated therapeutic editing of genes
US20210230637A1 (en) * 2018-05-08 2021-07-29 Osaka University Method for producing homozygous cells
CN112654710A (en) 2018-05-16 2021-04-13 辛瑟高公司 Methods and systems for directing RNA design and use
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
LT3629720T (en) 2018-06-14 2022-03-10 Regeneron Pharmaceuticals, Inc. ANIMALS OTHER THAN HUMANS ABLE TO PERFORM DH-DH REFORM OF THE IMMUNOGLOBULIN HEAVY CHAIN CODING SEQUENCES
EP3844272A1 (en) * 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
US11845958B2 (en) * 2018-09-05 2023-12-19 Wisconsin Alumni Research Foundation Genetically modified genes and cells, and methods of using same for silencing virus gene expression
WO2020050294A1 (en) * 2018-09-05 2020-03-12 学校法人慶應義塾 Homologous recombination efficiency increasing agent, and use thereof
CA3112612C (en) 2018-09-13 2024-02-27 Regeneron Pharmaceuticals, Inc. Complement factor h gene knockout rat as a model of c3 glomerulopathy
US20210395774A1 (en) * 2018-09-25 2021-12-23 Microbial Chemistry Research Foundation Novel virus vector and methods for producing and using same
CN113423831B (en) 2018-12-20 2023-03-10 瑞泽恩制药公司 Nuclease-mediated repeat amplification
EP3942042A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
ES2966625T3 (en) 2019-04-04 2024-04-23 Regeneron Pharma Rodents comprising a humanized coagulation factor 12 locus
CN113874510A (en) 2019-06-04 2021-12-31 瑞泽恩制药公司 Non-human animals comprising humanized TTR loci with beta slip mutations and methods of use
MA56117A (en) 2019-06-05 2022-04-13 Regeneron Pharma NON-HUMAN ANIMALS WITH A LIMITED LAMBDA LIGHT CHAIN REPERTORY EXPRESSED FROM THE KAPPA LOCUS AND THEIR USES
CA3137764A1 (en) 2019-06-07 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
EP4096396A1 (en) 2020-01-28 2022-12-07 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized pnpla3 locus and methods of use
WO2021158883A1 (en) 2020-02-07 2021-08-12 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized klkb1 locus and methods of use
WO2021188526A1 (en) * 2020-03-16 2021-09-23 Pairwise Plants Services, Inc. Natural guide architectures and methods of making and using the same
US20230102342A1 (en) 2020-03-23 2023-03-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
JP6867635B1 (en) * 2020-04-06 2021-04-28 株式会社Ｌｏｇｏｍｉｘ Genome modification method and genome modification kit
JP2023515710A (en) * 2020-04-27 2023-04-13 デューク ユニバーシティ A High-Throughput Screening Method to Find Optimal gRNA Pairs for CRISPR-Mediated Exon Deletion
CN116096873A (en) 2020-05-08 2023-05-09 布罗德研究所股份有限公司 Methods and compositions for simultaneously editing both strands of a target double-stranded nucleotide sequence
US20230232796A1 (en) 2020-06-26 2023-07-27 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ace2 locus
CN112481297A (en) * 2020-11-02 2021-03-12 深圳先进技术研究院 Method for making chromosome structure variation
CN112382339B (en) * 2020-11-17 2024-08-13 中国人民解放军军事科学院军事医学研究院 A method and device for identifying zygotic gene activation ZGA gene
TW202241934A (en) 2020-12-23 2022-11-01 美商再生元醫藥公司 Nucleic acids encoding anchor modified antibodies and uses thereof
US20220389461A1 (en) * 2021-05-26 2022-12-08 Inscripta, Inc. Crispr editing in diploid genomes
CN113832189B (en) * 2021-09-06 2022-10-21 中国人民解放军军事科学院军事医学研究院 gRNA for knocking out pig immunoglobulin heavy chain IGHG region and application thereof
JPWO2023054573A1 (en) * 2021-09-30 2023-04-06
CN118251491A (en) 2021-10-28 2024-06-25 瑞泽恩制药公司 CRISPR/Cas related methods and compositions for knocking out C5
KR20240117571A (en) 2021-12-08 2024-08-01 리제너론 파마슈티칼스 인코포레이티드 Mutant myocilin disease model and uses thereof
WO2023122506A1 (en) 2021-12-20 2023-06-29 Regeneron Pharmaceuticals, Inc. Non-human animals comprising humanized ace2 and tmprss loci
EP4475671A1 (en) 2022-02-07 2024-12-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
EP4476245A1 (en) 2022-02-11 2024-12-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for screening 4r tau targeting agents
WO2023250511A2 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
WO2024073679A1 (en) 2022-09-29 2024-04-04 Regeneron Pharmaceuticals, Inc. Correction of hepatosteatosis in humanized liver animals through restoration of il6/il6r/gp130 signaling in human hepatocytes

Family Cites Families (181)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4516228A (en) 1983-08-25 1985-05-07 Mobil Oil Corporation Acoustic well logging device for detecting compressional and shear waves
US5830729A (en) 1996-04-18 1998-11-03 Institut Pasteur I Sce I-induced gene replacement and gene conversion in embryonic stem cells
WO1999005266A2 (en) 1997-07-26 1999-02-04 Wisconsin Alumni Research Foundation Trans-species nuclear transfer
CA2354542A1 (en) 1998-12-31 2000-07-06 The J. David Gladstone Institutes Transgenic rodents and rodent cell lines expressing hiv co-receptors
JP2002535995A (en) 1999-02-03 2002-10-29 ザ チルドレンズ メディカル センター コーポレイション Gene repair involving induction of double-stranded DNA breaks at chromosomal target sites
ATE309536T1 (en) 1999-12-06 2005-11-15 Sangamo Biosciences Inc METHODS OF USING RANDOMIZED ZINC FINGER PROTEIN LIBRARIES TO IDENTIFY GENE FUNCTIONS
ATE387909T1 (en) 2000-05-31 2008-03-15 Novartis Vaccines & Diagnostic METHODS AND COMPOSITIONS FOR TREATING NEOPLASTIC DISEASES USING CHEMOTHERAPY AND RADIATION SENSITIZERS
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
US6586251B2 (en) * 2000-10-31 2003-07-01 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US7105348B2 (en) 2000-10-31 2006-09-12 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
AUPR451401A0 (en) 2001-04-20 2001-05-24 Monash University A method of nuclear transfer
CA2474486C (en) 2002-01-23 2013-05-14 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US7612250B2 (en) 2002-07-29 2009-11-03 Trustees Of Tufts College Nuclear transfer embryo formation method
US20030175968A1 (en) 2002-10-30 2003-09-18 Golic Kent G. Gene targeting method
CA2513226A1 (en) 2003-01-13 2004-07-29 Mahendra S. Rao Persistent expression of candidate molecule in proliferating stem and progenitor cells for delivery of therapeutic products
DK1684736T3 (en) 2003-12-01 2011-11-21 Kudos Pharm Ltd DNA damage repair inhibitors for cancer treatment
SI1802193T1 (en) 2004-10-19 2014-08-29 Regeneron Pharmaceuticals, Inc. Method for generating a mouse homozygous for a genetic modification
FR2879622B1 (en) 2004-12-17 2008-02-01 Agronomique Inst Nat Rech IN VITRO PROCESS FOR THE PRODUCTION OF OOCYTES OR EGGS WITH TARGETED GENOMIC MODIFICATION
GB0615327D0 (en) 2006-03-30 2006-09-13 Univ Edinburgh Culture medium containing kinase inhibitors and uses thereof
JP5514539B2 (en) 2006-03-31 2014-06-04 メダレックス・リミテッド・ライアビリティ・カンパニー Transgenic animals expressing chimeric antibodies for use in preparing human antibodies
CN101117633B (en) 2006-08-03 2011-07-20 上海交通大学附属儿童医院 Nucleus transplantation method
US7771967B2 (en) 2006-12-22 2010-08-10 The J. David Gladstone Institutes Nucleic acid encoding apolipoprotein E-I3
AU2008244473B2 (en) 2007-04-26 2013-06-20 Sangamo Therapeutics, Inc. Targeted integration into the PPP1R12C locus
ES2664218T3 (en) 2007-06-01 2018-04-18 Open Monoclonal Technology, Inc Compositions and methods of inhibiting endogenous immunoglobulin genes and producing transgenic human idiotypic antibodies
AU2009283194B2 (en) 2008-08-22 2014-10-16 Sangamo Therapeutics, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
JP5681114B2 (en) 2008-12-04 2015-03-04 サンガモ バイオサイエンシーズ， インコーポレイテッド Rat genome editing using zinc finger nuclease
BR112012003257A2 (en) * 2009-08-11 2016-11-22 Sangamo Biosciences Inc homozygous organisms for directed modification
US8354389B2 (en) 2009-08-14 2013-01-15 Regeneron Pharmaceuticals, Inc. miRNA-regulated differentiation-dependent self-deleting cassette
WO2011051390A1 (en) 2009-10-28 2011-05-05 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Homologous recombination in the oocyte
TR201903376T4 (en) 2009-10-29 2019-04-22 Regeneron Pharma Multifunctional alleles.
WO2011053957A2 (en) 2009-11-02 2011-05-05 Gen9, Inc. Compositions and methods for the regulation of multiple genes of interest in a cell
WO2011068103A1 (en) 2009-12-01 2011-06-09 独立行政法人国立がん研究センター Method for constructing chimeric rat using rat embryonic stem cells
WO2011078665A1 (en) 2009-12-21 2011-06-30 Keygene N.V. Improved techniques for transfecting protoplasts
UA113493C2 (en) 2010-01-22 2017-02-10 METHOD OF REMOVAL OF A DNA SECTION IN A PLANT
WO2011100058A1 (en) 2010-02-09 2011-08-18 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
KR101953237B1 (en) 2010-05-17 2019-02-28 상가모 테라퓨틱스, 인코포레이티드 Novel dna-binding proteins and uses thereof
GB201009732D0 (en) 2010-06-10 2010-07-21 Gene Bridges Gmbh Direct cloning
JP2013528062A (en) 2010-06-11 2013-07-08 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 Production of breedable XY female animals from XYES cells
JP6050230B2 (en) 2010-07-21 2016-12-21 サンガモ バイオサイエンシーズ， インコーポレイテッド Methods and compositions for modification of HLA loci
WO2012018726A1 (en) 2010-08-02 2012-02-09 Cellectis Sa Method for increasing double-strand break-induced gene targeting
WO2012129198A1 (en) 2011-03-23 2012-09-27 Transposagen Biopharmaceuticals, Inc. Genetically modified rat models for obesity and diabetes
WO2012168307A2 (en) 2011-06-07 2012-12-13 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Improved recombination efficiency by inhibition of nhej dna repair
ES2858978T3 (en) 2011-10-28 2021-09-30 Regeneron Pharma Mice with genetically modified T-cell receptors
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
SG10201702445TA (en) 2012-04-25 2017-04-27 Regeneron Pharma Nuclease-mediated targeting with large targeting vectors
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
SI3401400T1 (en) 2012-05-25 2019-10-30 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
JP6279562B2 (en) 2012-06-12 2018-02-14 ジェネンテック， インコーポレイテッド Methods and compositions for generating conditional knockout alleles
EP3808844A1 (en) 2012-07-25 2021-04-21 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
JP5952141B2 (en) * 2012-08-31 2016-07-13 京セラメディカル株式会社 Artificial cardiopulmonary pump drive
EP4357457B1 (en) 2012-10-23 2024-10-16 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
EP2928496B1 (en) 2012-12-06 2019-10-09 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
SG10201912327SA (en) 2012-12-12 2020-02-27 Broad Inst Inc Engineering and Optimization of Improved Systems, Methods and Enzyme Compositions for Sequence Manipulation
CN114634950A (en) 2012-12-12 2022-06-17 布罗德研究所有限公司 CRISPR-CAS component systems, methods, and compositions for sequence manipulation
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
SG10201912328UA (en) 2012-12-12 2020-02-27 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
PL2931898T3 (en) 2012-12-12 2016-09-30 Le Cong Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
KR20150095861A (en) 2012-12-17 2015-08-21 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Rna-guided human genome engineering
TR201900252T4 (en) * 2012-12-27 2019-02-21 Keygene Nv Method for Eliminating the Genetic Link in a Plant
WO2014127287A1 (en) 2013-02-14 2014-08-21 Massachusetts Institute Of Technology Method for in vivo tergated mutagenesis
WO2014130706A1 (en) 2013-02-20 2014-08-28 Regeneron Pharmaceuticals, Inc. Genetic modification of rats
AU2014218621B2 (en) 2013-02-25 2019-11-07 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
WO2014131833A1 (en) 2013-02-27 2014-09-04 Helmholtz Zentrum München Deutsches Forschungszentrum Für Gesundheit Und Umwelt (Gmbh) Gene editing in the oocyte by cas9 nucleases
US10612043B2 (en) * 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
BR112015022061B8 (en) 2013-03-14 2024-02-27 Caribou Biosciences Inc Genetically engineered single-stranded guide nucleic acid targeting nucleic acid, polynucleotide, method for cleaving a target nucleic acid and for ligating a target nucleic acid, composition and kit
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
EP2975942B1 (en) 2013-03-21 2018-08-08 Sangamo Therapeutics, Inc. Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases
WO2014165825A2 (en) 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
DK3456831T3 (en) 2013-04-16 2021-09-06 Regeneron Pharma TARGETED MODIFICATION OF RAT GENOMES
EP2986709A4 (en) 2013-04-16 2017-03-15 University Of Washington Through Its Center For Commercialization Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering
WO2014172470A2 (en) 2013-04-16 2014-10-23 Whitehead Institute For Biomedical Research Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal
EP2796558A1 (en) 2013-04-23 2014-10-29 Rheinische Friedrich-Wilhelms-Universität Bonn Improved gene targeting and nucleic acid carrier molecule, in particular for use in plants
CA2910427C (en) 2013-05-10 2024-02-20 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
AU2014273082B2 (en) 2013-05-29 2018-11-08 Cellectis A method for producing precise DNA cleavage using Cas9 nickase activity
US20140359795A1 (en) 2013-05-31 2014-12-04 Recombinetics, Inc. Genetic techniques for making animals with sortable sperm
WO2014201015A2 (en) 2013-06-11 2014-12-18 The Regents Of The University Of California Methods and compositions for target dna modification
EP3011035B1 (en) 2013-06-17 2020-05-13 The Broad Institute, Inc. Assay for quantitative evaluation of target site cleavage by one or more crispr-cas guide sequences
EP3011029B1 (en) 2013-06-17 2019-12-11 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
RU2716421C2 (en) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Delivery, use and use in therapy of crispr-cas systems and compositions for targeted action on disorders and diseases using viral components
DK3011031T3 (en) 2013-06-17 2020-12-21 Broad Inst Inc PERFORMANCE AND APPLICATION OF CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITIONS FOR LIVER TARGET DIRECTION AND THERAPY
EP3620524A1 (en) 2013-06-17 2020-03-11 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
CN105492611A (en) 2013-06-17 2016-04-13 布罗德研究所有限公司 Optimized CRISPR-CAS double nickase systems, methods and compositions for sequence manipulation
SG11201510297QA (en) 2013-06-19 2016-01-28 Sigma Aldrich Co Llc Targeted integration
JP2016528890A (en) 2013-07-09 2016-09-23 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 Therapeutic use of genome editing using the CRISPR / Cas system
JP6482546B2 (en) 2013-07-19 2019-03-13 ラリクス・バイオサイエンス・リミテッド・ライアビリティ・カンパニーＬａｒｉｘ Ｂｉｏｓｃｉｅｎｃｅ， Ｌｌｃ Methods and compositions for generating double allelic knockouts
US11060083B2 (en) 2013-07-19 2021-07-13 Larix Bioscience Llc Methods and compositions for producing double allele knock outs
WO2015011210A1 (en) 2013-07-24 2015-01-29 Medic Activ Vertriebs Gmbh Replacement cartridge
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
US20150059010A1 (en) * 2013-08-22 2015-02-26 Pioneer Hi-Bred International Inc Genome modification using guide polynucleotide/cas endonuclease systems and methods of use
EA037850B1 (en) 2013-08-29 2021-05-27 Тэмпл Юниверсити Оф Зе Коммонвэлс Систем Оф Хайе Эдьюкейшн Methods and compositions for rna-guided treatment of hiv infection
EP3418379B1 (en) 2013-09-18 2020-12-09 Kymab Limited Methods, cells & organisms
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
CN105899665B (en) 2013-10-17 2019-10-22 桑格摩生物科学股份有限公司 The delivering method and composition being transformed for nuclease-mediated genome project
CN105683375B (en) 2013-11-06 2021-02-02 国立大学法人广岛大学 Vector for inserting nucleic acid
JP2016536021A (en) 2013-11-07 2016-11-24 エディタス・メディシン，インコーポレイテッド CRISPR-related methods and compositions with governing gRNA
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
CN106103699B (en) 2013-11-28 2019-11-26 地平线探索有限公司 Body cell monoploid Human cell line
MX2016007654A (en) 2013-12-11 2017-08-15 Regeneron Pharma Methods and compositions for the targeted modification of a genome.
BR112016013213A2 (en) 2013-12-12 2017-12-05 Massachusetts Inst Technology administration, use and therapeutic applications of crisper systems and compositions for targeting disorders and diseases using particle delivery components
DK3079725T3 (en) 2013-12-12 2020-01-20 Broad Inst Inc ADMINISTRATION, USE, AND THERAPEUTICAL APPLICATIONS OF CRISPR CAS SYSTEMS AND COMPOSITIONS FOR RETURNING
KR20160089530A (en) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
KR20160097327A (en) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
EP3080258A1 (en) 2013-12-12 2016-10-19 The Broad Institute, Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
PT3083958T (en) 2013-12-19 2019-06-27 Amyris Inc Methods for genomic integration
SG11201605550QA (en) 2014-01-08 2016-08-30 Harvard College Rna-guided gene drives
WO2015112790A2 (en) 2014-01-24 2015-07-30 Children's Medical Center Corporation High-throughput mouse model for optimizing antibody affinities
WO2015117041A1 (en) 2014-01-30 2015-08-06 Nair Ramesh B Gene modification-mediated methods and compositions for generating dominant traits in eukaryotic systems
WO2015116969A2 (en) 2014-01-30 2015-08-06 The Board Of Trustees Of The University Of Arkansas Method, vectors, cells, seeds and kits for stacking genes into a single genomic site
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
EP3858376A1 (en) 2014-03-12 2021-08-04 Precision Biosciences, Inc. Dystrophin gene exon deletion using engineered nucleases
HRP20240186T1 (en) 2014-03-14 2024-05-10 Cibus Us Llc Methods and compositions for increasing efficiency of targeted gene modification using oligonucleotide-mediated gene repair
US10349639B2 (en) 2014-03-26 2019-07-16 University Of Maryland, College Park Targeted genome editing in zygotes of domestic large animals
GB201406968D0 (en) 2014-04-17 2014-06-04 Green Biologics Ltd Deletion mutants
KR101823661B1 (en) 2014-04-24 2018-01-30 기초과학연구원 A method of selecting a nuclease target sequence for gene knockout based on microhomology
GB201407852D0 (en) 2014-05-02 2014-06-18 Iontas Ltd Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules
EP3142706A1 (en) 2014-05-16 2017-03-22 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
AU2015266767A1 (en) 2014-05-30 2016-12-08 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods to treat latent viral infections
SI3152312T1 (en) 2014-06-06 2020-07-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for modifying a targeted locus
SG10202002486QA (en) 2014-06-16 2020-04-29 Univ Johns Hopkins Compositions and methods for the expression of crispr guide rnas using the h1 promoter
AU2015279642B2 (en) 2014-06-26 2021-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
AU2015289644A1 (en) 2014-07-15 2017-02-02 Juno Therapeutics, Inc. Engineered cells for adoptive cell therapy
EP3193944B1 (en) 2014-07-17 2021-04-07 University of Pittsburgh - Of the Commonwealth System of Higher Education Methods of treating cells containing fusion genes
US9944933B2 (en) 2014-07-17 2018-04-17 Georgia Tech Research Corporation Aptamer-guided gene targeting
CA2959070C (en) 2014-08-27 2020-11-10 Caribou Biosciences, Inc. Methods for increasing cas9-mediated engineering efficiency
WO2016036754A1 (en) 2014-09-02 2016-03-10 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification
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
WO2016049024A2 (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 competition of multiple cancer mutations in vivo
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
CN107002098A (en) 2014-09-29 2017-08-01 杰克逊实验室 Genetic modification mammal is produced by electroporation high efficiency, high flux
AU2015324935B2 (en) 2014-10-01 2021-04-08 The General Hospital Corporation Methods for increasing efficiency of nuclease-induced homology-directed repair
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
WO2016061073A1 (en) 2014-10-14 2016-04-21 Memorial Sloan-Kettering Cancer Center Composition and method for in vivo engineering of chromosomal rearrangements
CA2963315A1 (en) 2014-10-15 2016-04-21 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
EP3207139A1 (en) 2014-10-17 2017-08-23 The Penn State Research Foundation Methods and compositions for multiplex rna guided genome editing and other rna technologies
WO2016065364A1 (en) 2014-10-24 2016-04-28 Life Technologies Corporation Compositions and methods for enhancing homologous recombination
US12180263B2 (en) 2014-11-06 2024-12-31 President And Fellows Of Harvard College Cells lacking B2M surface expression and methods for allogeneic administration of such cells
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
EP3221457B1 (en) 2014-11-21 2019-03-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
CA2968808A1 (en) 2014-11-27 2016-06-02 Danziger Innovations Ltd. Nucleic acid constructs for genome editing
US20170266320A1 (en) 2014-12-01 2017-09-21 President And Fellows Of Harvard College RNA-Guided Systems for In Vivo Gene Editing
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)
EP3230452A1 (en) 2014-12-12 2017-10-18 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016097751A1 (en) 2014-12-18 2016-06-23 The University Of Bath Method of cas9 mediated genome engineering
SG11201704646YA (en) 2014-12-19 2017-07-28 Regeneron Pharma Methods and compositions for targeted genetic modification through single-step multiple targeting
JPWO2016104716A1 (en) 2014-12-26 2017-10-05 国立研究開発法人理化学研究所 Gene knockout method
US20180155708A1 (en) 2015-01-08 2018-06-07 President And Fellows Of Harvard College Split Cas9 Proteins
US10280451B2 (en) 2015-01-09 2019-05-07 Bio-Rad Laboratories, Inc. Detection of genome editing
CN107429263A (en) 2015-01-15 2017-12-01 斯坦福大学托管董事会 The method of controlling gene group editor
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
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
GB2535532B (en) 2015-02-23 2021-05-12 Knorr Bremse Systeme Fuer Nutzfahrzeuge Gmbh Brake valve arrangement
CA2977447A1 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
JP6871169B2 (en) 2015-03-02 2021-05-12 シナイ ヘルス システム Homologous recombination factor
GB201504223D0 (en) 2015-03-12 2015-04-29 Genome Res Ltd Biallelic genetic modification
WO2016154579A2 (en) 2015-03-26 2016-09-29 Editas Medicine, Inc. Crispr/cas-mediated gene conversion
CA2984237A1 (en) 2015-03-27 2016-10-06 President And Fellows Of Harvard College Modified t cells and methods of making and using the same
EP3277816B1 (en) 2015-04-01 2020-06-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
US10155938B2 (en) 2015-04-14 2018-12-18 City Of Hope Coexpression of CAS9 and TREX2 for targeted mutagenesis
WO2016168890A1 (en) 2015-04-22 2016-10-27 Genea Ip Holdings Pty Ltd Generation of muscle-lineage cells from stem cells
CA2983364A1 (en) 2015-04-27 2016-11-03 Genethon Compositions and methods for the treatment of nucleotide repeat expansion disorders
DK3289080T3 (en) 2015-04-30 2021-11-08 Univ Columbia GENERATION THERAPY FOR AUTOSOMAL DOMINANT DISEASES
PL3298134T3 (en) 2015-05-16 2023-09-18 Genzyme Corporation Gene editing of deep intronic mutations
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CN116064664A (en) 2015-10-08 2023-05-05 哈佛学院董事及会员团体 multiple genome editing
CN108138155A (en) 2015-10-20 2018-06-08 先锋国际良种公司 Via the function and application method that cas system is instructed to restore non-functional gene outcome
EP3370513A1 (en) 2015-11-06 2018-09-12 The Jackson Laboratory Large genomic dna knock-in and uses thereof
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017173004A1 (en) 2016-03-30 2017-10-05 Mikuni Takayasu A method for in vivo precise genome editing
EP3443080B1 (en) 2016-04-13 2021-08-11 University of Massachusetts Repairing compound heterozygous recessive mutations by allele exchange
SG11201809552SA (en) 2016-05-20 2018-11-29 Regeneron Pharma Methods for breaking immunological tolerance using multiple guide rnas
US11643670B2 (en) 2018-01-29 2023-05-09 Massachusetts Institute Of Technology Methods of enhancing chromosomal homologous recombination

Also Published As

Publication number Publication date
CN107208078B (en) 2021-07-16
CA3176380A1 (en) 2016-05-26
JP2017535271A (en) 2017-11-30
PT3221457T (en) 2019-06-27
CA2968440A1 (en) 2016-05-26
KR102415093B1 (en) 2022-07-04
EP3221457A2 (en) 2017-09-27
US10457960B2 (en) 2019-10-29
EP3521437A1 (en) 2019-08-07
KR20170102234A (en) 2017-09-08
SG10201913829YA (en) 2020-03-30
MX2022000378A (en) 2022-02-10
RU2017121367A (en) 2018-12-21
EP3521437B1 (en) 2025-01-15
IL283585A (en) 2021-07-29
MX2017006670A (en) 2018-03-01
SMT201900354T1 (en) 2019-07-11
JP2020191880A (en) 2020-12-03
WO2016081923A3 (en) 2016-07-07
HRP20190949T1 (en) 2019-07-26
IL301900A (en) 2023-06-01
CN113444747A (en) 2021-09-28
NZ731962A (en) 2022-07-01
US20200002731A1 (en) 2020-01-02
JP7101211B2 (en) 2022-07-14
KR102683423B1 (en) 2024-07-10
RU2020134412A (en) 2020-11-11
ES2731437T3 (en) 2019-11-15
CY1121738T1 (en) 2020-07-31
US20230332185A1 (en) 2023-10-19
DK3221457T3 (en) 2019-06-03
KR20220093013A (en) 2022-07-04
CN113444747B (en) 2025-02-11
CN107208078A (en) 2017-09-26
AU2021290301B2 (en) 2024-02-29
AU2015349692A1 (en) 2017-06-08
US20160145646A1 (en) 2016-05-26
IL252181A0 (en) 2017-07-31
KR20230070319A (en) 2023-05-22
WO2016081923A2 (en) 2016-05-26
PL3221457T3 (en) 2019-09-30
RU2017121367A3 (en) 2019-11-05
RU2734770C2 (en) 2020-10-23
US11697828B2 (en) 2023-07-11
EP3221457B1 (en) 2019-03-20
AU2015349692B2 (en) 2021-10-28
BR112017010547A2 (en) 2018-02-27
IL252181B (en) 2021-06-30
SI3221457T1 (en) 2019-08-30
HUE044907T2 (en) 2019-11-28
US20200002730A1 (en) 2020-01-02
AU2021290301A1 (en) 2022-01-27
JP6727199B2 (en) 2020-07-22
RS58893B1 (en) 2019-08-30
IL283585B1 (en) 2023-05-01
IL283585B2 (en) 2023-09-01
KR102531016B1 (en) 2023-05-10
LT3221457T (en) 2019-06-10

Similar Documents

Publication Publication Date Title
IL283585A (en) Methods and compositions for targeted genetic modification using paired guide rnas
ZA202007633B (en) Modified guide rnas
HRP20181630T1 (en) Methods and compositions for targeted genetic modifications and methods of use
IL252755A0 (en) Methods and compositions for targeted genetic modification through single-step multiple targeting
IL252386B (en) Targeted rna editing
SG11201608605QA (en) Methods and means for enhancing rna production
LT3108009T (en) Methods and compositions for dna profiling
PT3200815T (en) Methods and compositions for treating cancer
HK1246206A1 (en) Compositions and methods for treating glaucoma
HK1244782A1 (en) Compositions and methods for treating diseases and conditions
ZA201705645B (en) Methods and compositions for dna profiling
GB201407949D0 (en) Compositions and methods for treating biofilms