ES2947714T3 - Métodos y composiciones para la modificación genética dirigida a través de múltiples direccionamientos en un solo paso - Google Patents

Métodos y composiciones para la modificación genética dirigida a través de múltiples direccionamientos en un solo paso Download PDF

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ES2947714T3
ES2947714T3 ES19201384T ES19201384T ES2947714T3 ES 2947714 T3 ES2947714 T3 ES 2947714T3 ES 19201384 T ES19201384 T ES 19201384T ES 19201384 T ES19201384 T ES 19201384T ES 2947714 T3 ES2947714 T3 ES 2947714T3
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nucleic acid
cell
acid insert
ltvec
sequence
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ES2947714T9 (es
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Vera Voronina
Lynn Macdonald
Brian Zambrowicz
Andrew Murphy
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276Knock-out vertebrates
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278Knock-in vertebrates, e.g. humanised vertebrates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/072Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Environmental Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
ES19201384T 2014-12-19 2015-12-18 Métodos y composiciones para la modificación genética dirigida a través de múltiples direccionamientos en un solo paso Active ES2947714T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462094104P 2014-12-19 2014-12-19
US201562167408P 2015-05-28 2015-05-28
US201562205524P 2015-08-14 2015-08-14

Publications (2)

Publication Number Publication Date
ES2947714T3 true ES2947714T3 (es) 2023-08-17
ES2947714T9 ES2947714T9 (es) 2024-03-14

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Family Applications (2)

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ES19201384T Active ES2947714T3 (es) 2014-12-19 2015-12-18 Métodos y composiciones para la modificación genética dirigida a través de múltiples direccionamientos en un solo paso
ES15828604T Active ES2760508T3 (es) 2014-12-19 2015-12-18 Métodos y composiciones para la modificación genética dirigida a través de la transformación múltiple en una sola etapa

Family Applications After (1)

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ES15828604T Active ES2760508T3 (es) 2014-12-19 2015-12-18 Métodos y composiciones para la modificación genética dirigida a través de la transformación múltiple en una sola etapa

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US (2) US11326184B2 (enExample)
EP (2) EP3653048B9 (enExample)
JP (2) JP6840077B2 (enExample)
KR (1) KR102530821B1 (enExample)
CN (1) CN107208113A (enExample)
AU (1) AU2015364427B2 (enExample)
BR (1) BR112017013104A2 (enExample)
CA (1) CA2971213C (enExample)
ES (2) ES2947714T3 (enExample)
IL (1) IL252755B (enExample)
MX (1) MX388784B (enExample)
NZ (1) NZ732895A (enExample)
RU (1) RU2707137C2 (enExample)
SG (1) SG11201704646YA (enExample)
WO (1) WO2016100819A1 (enExample)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104364380B (zh) 2012-04-25 2018-10-09 瑞泽恩制药公司 核酸酶介导的使用大靶向载体的靶向
MX369747B (es) 2013-04-16 2019-11-20 Regeneron Pharma Modificación dirigida del genoma de rata.
RU2685914C1 (ru) 2013-12-11 2019-04-23 Регенерон Фармасьютикалс, Инк. Способы и композиции для направленной модификации генома
HUE049776T2 (hu) 2014-06-06 2020-10-28 Regeneron Pharma Módszerek és készítmények egy célzott lókusz módosítására
WO2015200805A2 (en) 2014-06-26 2015-12-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
US20180250424A1 (en) 2014-10-10 2018-09-06 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
PT3207124T (pt) 2014-10-15 2019-08-27 Regeneron Pharma Métodos e composições para a produção ou conservação de células pluripotentes
EP3215617B1 (en) 2014-11-07 2024-05-08 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
RU2734770C2 (ru) 2014-11-21 2020-10-23 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации с использованием парных гидовых рнк
RU2707137C2 (ru) 2014-12-19 2019-11-22 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации посредством одноэтапного множественного нацеливания
BR112017025507A2 (pt) 2015-05-29 2018-08-07 Regeneron Pharmaceuticals, Inc. roedor, célula isolada de roedor ou tecido isolado de roedor, célula-tronco embrionária de roedor, embrião de roedor, e, métodos para preparação de um roedor e para identificação de um candidato terapêutico.
WO2017053879A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
CN108777951B (zh) 2016-01-13 2021-11-05 瑞泽恩制药公司 具有包括改造的多样性簇的免疫球蛋白重链可变区的非人动物及其用途
WO2017165826A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
US11236313B2 (en) 2016-04-13 2022-02-01 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
RU2760877C2 (ru) 2016-09-30 2021-12-01 Регенерон Фармасьютикалс, Инк. Животные, отличные от человека, характеризующиеся экспансией гексануклеотидных повторов в локусе c9orf72
CN109996441B (zh) 2016-11-04 2022-02-08 瑞泽恩制药公司 具有经工程化的免疫球蛋白λ轻链基因座的非人类动物
AU2018208463A1 (en) * 2017-01-10 2019-08-01 Christina Care Gene Editing Institute, Inc. Methods for in vitro site-directed mutagenesis using gene editing technologies
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
KR20200033259A (ko) * 2017-07-31 2020-03-27 리제너론 파마슈티칼스 인코포레이티드 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물
EP3720279B1 (en) 2017-12-05 2022-09-21 Regeneron Pharmaceuticals, Inc. Mice having an engineered immunoglobulin lambda light chain and uses thereof
IL318469A (en) 2018-06-14 2025-03-01 Regeneron Pharma Non-human animals capable of reorganizing transgenic DH-DH, and their uses
CN111321171A (zh) * 2018-12-14 2020-06-23 江苏集萃药康生物科技有限公司 一种应用CRISPR/Cas9介导ES打靶技术制备基因打靶动物模型的方法
JP7449291B2 (ja) 2018-12-20 2024-03-13 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 ヌクレアーゼ媒介リピート伸長
IL286917B (en) * 2019-04-04 2022-09-01 Regeneron Pharma Methods for scarless introduction of targeted modifications into targeting vectors
TW202110323A (zh) 2019-06-05 2021-03-16 美商再生元醫藥公司 具有表現自κ 基因座的有限λ 輕鏈組庫的非人類動物及其用途
CA3165366A1 (en) 2020-12-23 2022-06-30 Regeneron Pharmaceuticals, Inc. Nucleic acids encoding anchor modified antibodies and uses thereof
WO2023046038A1 (en) * 2021-09-24 2023-03-30 Immunocan Biotech Co. Ltd. Methods for large-size chromosomal transfer and modified chromosomes and organisims using same
EP4608982A2 (en) * 2022-10-27 2025-09-03 Seawolf Therapeutics, Inc. Nuclear targeted dna delivery and compositions for use in practicing the same

Family Cites Families (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU664976B2 (en) * 1990-08-29 1995-12-14 Gene Pharming Europe Bv Homologous recombination in mammalian cells
US5830729A (en) 1996-04-18 1998-11-03 Institut Pasteur I Sce I-induced gene replacement and gene conversion in embryonic stem cells
AU8587598A (en) 1997-07-26 1999-02-16 Wisconsin Alumni Research Foundation Trans-species nuclear transfer
US6372956B1 (en) 1998-12-31 2002-04-16 The J. David Gladstone Institutes Transgenic rats and rat cell lines expressing human CD4 and a human chemokine receptor
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US20030104526A1 (en) 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
ATE309536T1 (de) 1999-12-06 2005-11-15 Sangamo Biosciences Inc Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
US6586251B2 (en) 2000-10-31 2003-07-01 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US7105348B2 (en) * 2000-10-31 2006-09-12 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
AU2884102A (en) 2000-12-07 2002-06-18 Sangamo Biosciences Inc Regulation of angiogenesis with zinc finger proteins
AU2002225187A1 (en) 2001-01-22 2002-07-30 Sangamo Biosciences, Inc. Zinc finger polypeptides and their use
EP1353941B1 (en) 2001-01-22 2013-03-13 Sangamo BioSciences, Inc. Modified zinc finger binding proteins
AUPR451401A0 (en) 2001-04-20 2001-05-24 Monash University A method of nuclear transfer
EP1476547B1 (en) 2002-01-23 2006-12-06 The University of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20060078552A1 (en) 2002-03-15 2006-04-13 Sylvain Arnould Hybrid and single chain meganucleases and use thereof
ATE531796T1 (de) 2002-03-21 2011-11-15 Sangamo Biosciences Inc Verfahren und zusammensetzungen zur verwendung von zinkfinger-endonukleasen zur verbesserung der homologen rekombination
US7612250B2 (en) 2002-07-29 2009-11-03 Trustees Of Tufts College Nuclear transfer embryo formation method
AU2003298574B2 (en) 2002-09-05 2008-04-24 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
AU2003290518A1 (en) 2002-09-06 2004-04-23 Fred Hutchinson Cancer Research Center Methods and compositions concerning designed highly-specific nucleic acid binding proteins
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
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
EP1591521A1 (en) 2004-04-30 2005-11-02 Cellectis I-Dmo I derivatives with enhanced activity at 37 degrees C and use thereof
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
DK1802193T3 (da) 2004-10-19 2014-06-10 Regeneron Pharma Fremgangsmåde til frembringelse af en homozygot mus til genetisk modifikation
FR2879622B1 (fr) 2004-12-17 2008-02-01 Agronomique Inst Nat Rech Procede in vitro de production d'ovocytes ou d'oeufs presentant une modification genomique ciblee
WO2006097784A1 (en) 2005-03-15 2006-09-21 Cellectis I-crei meganuclease variants with modified specificity, method of preparation and uses thereof
EP2327773A1 (en) 2005-03-15 2011-06-01 Cellectis I-crel meganuclease variants with modified specificity, method of preparation and uses thereof
GB0615327D0 (en) 2006-03-30 2006-09-13 Univ Edinburgh Culture medium containing kinase inhibitors and uses thereof
AU2007235496B2 (en) 2006-03-31 2013-11-21 E. R. Squibb & Sons, L.L.C. Transgenic animals expressing chimeric antibodies for use in preparing human antibodies
CN101117633B (zh) 2006-08-03 2011-07-20 上海交通大学附属儿童医院 一种细胞核移植方法
JP5632610B2 (ja) 2006-12-14 2014-11-26 ダウ アグロサイエンシィズ エルエルシー 最適化された非正準ジンクフィンガータンパク質
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
EP2336329B8 (en) 2007-06-01 2012-11-21 Omt, Inc. Compositions and methods for inhibiting endogenous immunoglobulin genes and producing transgenis human idiotype antibodies
GB0803109D0 (en) * 2008-02-20 2008-03-26 Gene Bridges Gmbh Method of nucleic acid recombination
ES2627552T3 (es) 2008-12-04 2017-07-28 Sigma Aldrich Company Edición de genoma en ratas usando nucleasas con dedos de cinc
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
EP2408921B1 (en) 2009-03-20 2017-04-19 Sangamo BioSciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
US8772008B2 (en) 2009-05-18 2014-07-08 Sangamo Biosciences, Inc. Methods and compositions for increasing nuclease activity
US20120178647A1 (en) 2009-08-03 2012-07-12 The General Hospital Corporation Engineering of zinc finger arrays by context-dependent assembly
WO2011051390A1 (en) 2009-10-28 2011-05-05 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Homologous recombination in the oocyte
TR201903376T4 (tr) 2009-10-29 2019-04-22 Regeneron Pharma Çok fonksiyonlu alleller.
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 (ja) 2009-12-01 2011-06-09 独立行政法人国立がん研究センター ラット胚性幹細胞を用いたキメララットの作製法
CN106834320B (zh) 2009-12-10 2021-05-25 明尼苏达大学董事会 Tal效应子介导的dna修饰
ES2583060T3 (es) 2009-12-21 2016-09-16 Keygene N.V. Técnicas mejoradas para la transfección de protoplastos
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
GB201009732D0 (en) 2010-06-10 2010-07-21 Gene Bridges Gmbh Direct cloning
CA2800534C (en) 2010-06-11 2019-01-22 Regeneron Pharmaceuticals, Inc. Production of fertile xy female animals from xy es cells
JP6050230B2 (ja) 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 Hla遺伝子座の修飾のための方法及び組成物
KR101971741B1 (ko) 2010-07-23 2019-08-14 시그마-알드리치 컴퍼니., 엘엘씨 표적화 엔도뉴클레아제 및 단일-가닥 핵산을 사용하는 게놈 편집
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
EP2771357B1 (en) 2011-10-28 2018-07-18 Regeneron Pharmaceuticals, Inc. Genetically modified t cell receptor mice
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
CN104364380B (zh) 2012-04-25 2018-10-09 瑞泽恩制药公司 核酸酶介导的使用大靶向载体的靶向
WO2013169802A1 (en) 2012-05-07 2013-11-14 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
RS59199B1 (sr) 2012-05-25 2019-10-31 Univ California Metode i jedinjenja za rnk-upravljanu ciljanu dnk modifikaciju i za rnk- upravljanu modulaciju transkripta
JP6517143B2 (ja) 2012-10-23 2019-05-22 ツールゲン インコーポレイテッド 標的dnaに特異的なガイドrnaおよびcasタンパク質コード核酸またはcasタンパク質を含む、標的dnaを切断するための組成物、ならびにその使用
KR102479178B1 (ko) 2012-12-06 2022-12-19 시그마-알드리치 컴퍼니., 엘엘씨 Crispr-기초된 유전체 변형과 조절
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP2898075B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP2931030B2 (en) * 2012-12-14 2024-01-17 OmniAb, Inc. Polynucleotides encoding rodent antibodies with human idiotypes and animals comprising same
JP6700788B2 (ja) 2012-12-17 2020-05-27 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 Rna誘導性ヒトゲノム改変
EP3491915B1 (en) 2012-12-27 2023-05-31 Keygene N.V. Method for inducing a targeted translocation in a plant.
EP3561050B1 (en) 2013-02-20 2021-12-08 Regeneron Pharmaceuticals, Inc. Genetic modification of rats
EP2922393B2 (en) 2013-02-27 2022-12-28 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Gene editing in the oocyte by cas9 nucleases
WO2014143381A1 (en) 2013-03-09 2014-09-18 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple crispr/cas selections of recombineering events
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US9937207B2 (en) 2013-03-21 2018-04-10 Sangamo Therapeutics, Inc. Targeted disruption of T cell receptor genes using talens
MX369747B (es) * 2013-04-16 2019-11-20 Regeneron Pharma Modificación dirigida del genoma de rata.
WO2015040402A1 (en) 2013-09-18 2015-03-26 Kymab Limited Methods. cells & organisms
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
RU2685914C1 (ru) 2013-12-11 2019-04-23 Регенерон Фармасьютикалс, Инк. Способы и композиции для направленной модификации генома
PT3083958T (pt) 2013-12-19 2019-06-27 Amyris Inc Métodos de integração genómica
US20170076039A1 (en) 2014-04-24 2017-03-16 Institute For Basic Science 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
HUE049776T2 (hu) 2014-06-06 2020-10-28 Regeneron Pharma Módszerek és készítmények egy célzott lókusz módosítására
WO2015200805A2 (en) 2014-06-26 2015-12-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
AU2015317370A1 (en) * 2014-09-19 2017-03-23 Regeneron Pharmaceuticals, Inc. Chimeric antigen receptors
WO2016061073A1 (en) 2014-10-14 2016-04-21 Memorial Sloan-Kettering Cancer Center Composition and method for in vivo engineering of chromosomal rearrangements
PT3207124T (pt) 2014-10-15 2019-08-27 Regeneron Pharma Métodos e composições para a produção ou conservação de células pluripotentes
JP6772067B2 (ja) 2014-11-20 2020-10-21 国立大学法人京都大学 哺乳動物の標的ゲノム領域にdnaをノックインする方法及び細胞
RU2734770C2 (ru) 2014-11-21 2020-10-23 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации с использованием парных гидовых рнк
US20170266320A1 (en) 2014-12-01 2017-09-21 President And Fellows Of Harvard College RNA-Guided Systems for In Vivo Gene Editing
RU2707137C2 (ru) 2014-12-19 2019-11-22 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации посредством одноэтапного множественного нацеливания
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems

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