[go: up one dir, main page]

MX2017008190A - Metodos y composiciones para la modificacion genetica dirigida a traves de direccionamiento multiple de una sola etapa. - Google Patents

Metodos y composiciones para la modificacion genetica dirigida a traves de direccionamiento multiple de una sola etapa.

Info

Publication number
MX2017008190A
MX2017008190A MX2017008190A MX2017008190A MX2017008190A MX 2017008190 A MX2017008190 A MX 2017008190A MX 2017008190 A MX2017008190 A MX 2017008190A MX 2017008190 A MX2017008190 A MX 2017008190A MX 2017008190 A MX2017008190 A MX 2017008190A
Authority
MX
Mexico
Prior art keywords
methods
compositions
single stage
genomic locus
genetic modification
Prior art date
Application number
MX2017008190A
Other languages
English (en)
Other versions
MX388784B (es
Inventor
Macdonald Lynn
Voronina Vera
Zambrowicz Brian
J Murphy Andrew
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 MX2017008190A publication Critical patent/MX2017008190A/es
Publication of MX388784B publication Critical patent/MX388784B/es

Links

Classifications

    • 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
    • A01K67/0276—Knock-out vertebrates
    • 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
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • 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
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • 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]

Landscapes

  • 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)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (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)

Abstract

Se proporcionan métodos y composiciones para realizar una o más modificaciones genéticas dirigidas en un locus genómico objetivo dentro de una célula y para producir animales no humanos que comprenden el locus genómico modificado. Los métodos emplean dos o más vectores dirigidos grandes que son capaces de recombinarse entre sí y con el locus genómico objetivo en una única etapa de direccionamiento genómico. Los métodos pueden emplearse, además, en combinación con un agente de nucleasa. Además, se proporcionan métodos y composiciones para mejorar la recombinación homóloga en un locus genómico objetivo en una célula. Los métodos emplean dos o más ácidos nucleicos que comprenden una o más secuencias de superposición. Los métodos pueden emplearse en combinación con un agente de nucleasa o sin agente de nucleasa.
MX2017008190A 2014-12-19 2015-12-18 Métodos y composiciones para la modificación genética dirigida a través de direccionamiento múltiple de una sola etapa. MX388784B (es)

Applications Claiming Priority (4)

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
PCT/US2015/066681 WO2016100819A1 (en) 2014-12-19 2015-12-18 Methods and compositions for targeted genetic modification through single-step multiple targeting

Publications (2)

Publication Number Publication Date
MX2017008190A true MX2017008190A (es) 2018-03-23
MX388784B MX388784B (es) 2025-03-20

Family

ID=55229831

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2017008190A MX388784B (es) 2014-12-19 2015-12-18 Métodos y composiciones para la modificación genética dirigida a través de direccionamiento múltiple de una sola etapa.

Country Status (15)

Country Link
US (2) US11326184B2 (es)
EP (2) EP3232774B1 (es)
JP (2) JP6840077B2 (es)
KR (1) KR102530821B1 (es)
CN (1) CN107208113A (es)
AU (1) AU2015364427B2 (es)
BR (1) BR112017013104A2 (es)
CA (1) CA2971213C (es)
ES (2) ES2760508T3 (es)
IL (1) IL252755B (es)
MX (1) MX388784B (es)
NZ (1) NZ732895A (es)
RU (1) RU2707137C2 (es)
SG (1) SG11201704646YA (es)
WO (1) WO2016100819A1 (es)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201406547YA (en) 2012-04-25 2014-11-27 Regeneron Pharma Nuclease-mediated targeting with large targeting vectors
HRP20181648T1 (hr) 2013-04-16 2019-01-25 Regeneron Pharmaceuticals, Inc. Ciljana modifikacija genoma štakora
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
MX385689B (es) 2014-06-06 2025-03-18 Regeneron Pharma Métodos y composiciones para modificar un locus dirigido.
ES2694629T3 (es) 2014-06-26 2018-12-26 Regeneron Pharmaceuticals, Inc. Métodos y composiciones para modificaciones genéticas objetivo y métodos de uso
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
CN107002042A (zh) 2014-10-15 2017-08-01 瑞泽恩制药公司 用于产生或维持多能细胞的方法和组合物
US11680268B2 (en) 2014-11-07 2023-06-20 Editas Medicine, Inc. Methods 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
MX388784B (es) * 2014-12-19 2025-03-20 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida a través de direccionamiento múltiple de una sola etapa.
RU2725737C2 (ru) 2015-05-29 2020-07-03 Регенерон Фармасьютикалс, Инк. Животные, отличные от человека, с нарушением в локусе c9orf72
CA2999500A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
IL260526B2 (en) 2016-01-13 2023-10-01 Regeneron Pharma Rodents having an engineered heavy chain diversity region
EP3433363A1 (en) 2016-03-25 2019-01-30 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
SG10202102997UA (en) 2016-09-30 2021-04-29 Regeneron Pharma Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus
HRP20250695T1 (hr) 2016-11-04 2025-08-01 Regeneron Pharmaceuticals, Inc. Ne-ljudske životinje koje imaju konstruirani lokus imunoglobulinskog lambda lakog lanca
CA3048501A1 (en) * 2017-01-10 2018-07-19 Christiana Care Health Services, Inc. Methods for in vitro site-directed mutagenesis using gene editing technologies
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
CA3068072A1 (en) * 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. Methods and compositions for assessing crispr/cas-mediated disruption or excision and crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo
HUE071852T2 (hu) 2017-12-05 2025-09-28 Regeneron Pharma Genetikailag módosított immunglobulin lambda könnyûlánccal rendelkezõ egerek és azok alkalmazása
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
AU2019403015B2 (en) 2018-12-20 2024-01-18 Regeneron Pharmaceuticals, Inc. Nuclease-mediated repeat expansion
RU2771374C1 (ru) * 2019-04-04 2022-05-04 Редженерон Фармасьютикалс, Инк. Способы для бесшовного внесения целевых модификаций в направленные векторы
AU2020289554A1 (en) 2019-06-05 2021-11-18 Regeneron Pharmaceuticals, Inc. Non-human animals having a limited lambda light chain repertoire expressed from the kappa locus and uses thereof
EP4051700B1 (en) 2020-12-23 2025-05-07 Regeneron Pharmaceuticals, Inc. Nucleic acids encoding anchor modified antibodies and uses thereof
CA3222922A1 (en) * 2021-09-24 2023-03-30 Jiwei Zhang Methods for large-size chromosomal transfer and modified chromosomes and organisims using same
WO2024091578A2 (en) * 2022-10-27 2024-05-02 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
CA2090473A1 (en) * 1990-08-29 1992-03-01 Robert M. Kay Homologous recombinatin 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
WO1999005266A2 (en) 1997-07-26 1999-02-04 Wisconsin Alumni Research Foundation Trans-species nuclear transfer
AU2391000A (en) 1998-12-31 2000-07-31 J. David Gladstone Institutes, The Transgenic rodents and rodent cell lines expressing hiv co-receptors
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
IL150069A0 (en) 1999-12-06 2002-12-01 Sangamo Biosciences Inc Methods of using randomized libraries of zinc finger proteins for the identification of gene function
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
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
US7105348B2 (en) * 2000-10-31 2006-09-12 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
AU2884102A (en) 2000-12-07 2002-06-18 Sangamo Biosciences Inc Regulation of angiogenesis with zinc finger proteins
WO2002057308A2 (en) 2001-01-22 2002-07-25 Sangamo Biosciences, Inc. Zinc finger polypeptides and their use
DK1353941T3 (da) 2001-01-22 2013-06-17 Sangamo Biosciences Inc Modificerede zinkfingerbindingsproteiner
AUPR451401A0 (en) 2001-04-20 2001-05-24 Monash University A method of nuclear transfer
BRPI0307383B1 (pt) 2002-01-23 2019-12-31 The Univ Of Utah Research Foundation método de recombinação genética direcionada em célula de planta hospedeira
WO2003078619A1 (en) 2002-03-15 2003-09-25 Cellectis Hybrid and single chain meganucleases and use thereof
AU2003218382B2 (en) 2002-03-21 2007-12-13 Sangamo Therapeutics, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7612250B2 (en) 2002-07-29 2009-11-03 Trustees Of Tufts College Nuclear transfer embryo formation method
EP1581610A4 (en) 2002-09-05 2009-05-27 California Inst Of Techn USE OF CHIMERIC NUCLEASES TO STIMULATE GEN-TARGETING
WO2004031346A2 (en) 2002-09-06 2004-04-15 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
EP2292274A1 (en) 2004-09-16 2011-03-09 Sangamo BioSciences, Inc. Compositions and methods for protein production
PL2767161T3 (pl) 2004-10-19 2018-09-28 Regeneron Pharmaceuticals, Inc. Sposób wytwarzania zwierzęcia homozygotycznego pod względem modyfikacji genetycznej
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
US7897372B2 (en) 2005-03-15 2011-03-01 Cellectis I-CreI meganuclease variants with modified specificity, method of preparation and uses thereof
WO2006097784A1 (en) 2005-03-15 2006-09-21 Cellectis I-crei 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
CA2638774C (en) 2006-03-31 2015-11-24 Medarex, Inc. 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
TWI470079B (zh) 2006-12-14 2015-01-21 Dow Agrosciences Llc 最佳化之非標準鋅指蛋白
US7771967B2 (en) 2006-12-22 2010-08-10 The J. David Gladstone Institutes Nucleic acid encoding apolipoprotein E-I3
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
KR102096731B1 (ko) 2007-06-01 2020-04-02 오픈 모노클로날 테크놀로지, 인코포레이티드 내생적 면역글로불린 유전자를 억제하고 트랜스제닉 인간 이디오타입 항체를 생산하기 위한 방법 및 조성물
GB0803109D0 (en) * 2008-02-20 2008-03-26 Gene Bridges Gmbh Method of nucleic acid recombination
EP3156494B8 (en) 2008-12-04 2018-09-19 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
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
WO2010107493A2 (en) 2009-03-20 2010-09-23 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
US20120276537A1 (en) 2009-10-28 2012-11-01 Kuehn Ralf Homologous recombination in the oocyte
AU2010319894B2 (en) 2009-10-29 2015-03-05 Regeneron Pharmaceuticals, Inc. Multifunctional alleles
US20120315670A1 (en) 2009-11-02 2012-12-13 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
US8586363B2 (en) 2009-12-10 2013-11-19 Regents Of The University Of Minnesota TAL effector-mediated DNA modification
WO2011078665A1 (en) 2009-12-21 2011-06-30 Keygene N.V. Improved techniques for transfecting protoplasts
US9255259B2 (en) 2010-02-09 2016-02-09 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
ES2727738T3 (es) 2010-06-11 2019-10-18 Regeneron Pharma Producción de animales hembra xy fértiles a partir de células es xy
CA2805442C (en) 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus
US9512444B2 (en) 2010-07-23 2016-12-06 Sigma-Aldrich Co. Llc Genome editing using targeting endonucleases and single-stranded nucleic acids
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
LT3424947T (lt) 2011-10-28 2021-03-10 Regeneron Pharmaceuticals, Inc. Genetiškai modifikuotos t ląstelių receptorių pelės
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
SG11201406547YA (en) 2012-04-25 2014-11-27 Regeneron Pharma Nuclease-mediated targeting with large targeting vectors
KR102116153B1 (ko) 2012-05-07 2020-05-27 상가모 테라퓨틱스, 인코포레이티드 도입유전자의 뉴클레아제-매개 표적화된 통합을 위한 방법 및 조성물
US20160046961A1 (en) 2012-05-25 2016-02-18 Emmanuelle Charpentier Methods and Compositions for RNA-Directed Target DNA Modification and For RNA-Directed Modulation of Transcription
EP2912175B1 (en) 2012-10-23 2018-08-22 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
CN108913676B (zh) 2012-12-06 2023-11-03 西格马-奥尔德里奇有限责任公司 基于crispr的基因组修饰和调控
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
DK2898075T3 (en) 2012-12-12 2016-06-27 Broad Inst Inc CONSTRUCTION AND OPTIMIZATION OF IMPROVED SYSTEMS, PROCEDURES AND ENZYME COMPOSITIONS FOR SEQUENCE MANIPULATION
CN104994729B (zh) * 2012-12-14 2019-01-11 Omt公司 编码具有人独特型的啮齿动物抗体的多核苷酸以及包含所述多核苷酸的动物
SG10201912991WA (en) 2012-12-17 2020-03-30 Harvard College Rna-guided human genome engineering
CN105025701B (zh) 2012-12-27 2018-09-25 凯津公司 去除植物中遗传连锁的方法
EP3561050B1 (en) 2013-02-20 2021-12-08 Regeneron Pharmaceuticals, Inc. Genetic modification of rats
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
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
JP6346266B2 (ja) 2013-03-21 2018-06-20 サンガモ セラピューティクス， インコーポレイテッド 操作されたジンクフィンガータンパク質ヌクレアーゼを使用するｔ細胞受容体遺伝子の標的化された破壊
HRP20181648T1 (hr) 2013-04-16 2019-01-25 Regeneron Pharmaceuticals, Inc. Ciljana modifikacija genoma štakora
ES3013744T3 (en) 2013-09-18 2025-04-15 Kymab Ltd Methods, cells and organisms
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
CN106029886B (zh) 2013-12-19 2021-02-05 阿迈瑞斯公司 基因组整合的方法
KR101823661B1 (ko) 2014-04-24 2018-01-30 기초과학연구원 마이크로 상동 기반의 유전자 녹아웃을 위한 뉴클레아제 표적 서열을 확인하는 방법
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
MX385689B (es) 2014-06-06 2025-03-18 Regeneron Pharma Métodos y composiciones para modificar un locus dirigido.
ES2694629T3 (es) 2014-06-26 2018-12-26 Regeneron Pharmaceuticals, Inc. Métodos y composiciones para modificaciones genéticas objetivo y métodos de uso
KR20170083534A (ko) * 2014-09-19 2017-07-18 리제너론 파마슈티칼스 인코포레이티드 키메라 항원 수용체
WO2016061073A1 (en) 2014-10-14 2016-04-21 Memorial Sloan-Kettering Cancer Center Composition and method for in vivo engineering of chromosomal rearrangements
CN107002042A (zh) 2014-10-15 2017-08-01 瑞泽恩制药公司 用于产生或维持多能细胞的方法和组合物
US10362771B2 (en) 2014-11-20 2019-07-30 Kyoto University Method for knock-in of DNA into target region of mammalian genome, and cell
RU2734770C2 (ru) 2014-11-21 2020-10-23 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации с использованием парных гидовых рнк
WO2016089866A1 (en) 2014-12-01 2016-06-09 President And Fellows Of Harvard College Rna-guided systems for in vivo gene editing
MX388784B (es) 2014-12-19 2025-03-20 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida a través de direccionamiento múltiple de una sola etapa.
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems

Also Published As

Publication number Publication date
EP3232774B1 (en) 2019-10-09
CA2971213C (en) 2023-09-26
CN107208113A (zh) 2017-09-26
RU2017124909A3 (es) 2019-06-05
AU2015364427A1 (en) 2017-07-06
JP2017538428A (ja) 2017-12-28
JP7095066B2 (ja) 2022-07-04
MX388784B (es) 2025-03-20
KR102530821B1 (ko) 2023-05-10
JP2021045174A (ja) 2021-03-25
EP3653048A1 (en) 2020-05-20
JP6840077B2 (ja) 2021-03-10
AU2015364427B2 (en) 2021-05-27
IL252755B (en) 2021-10-31
IL252755A0 (en) 2017-08-31
ES2947714T3 (es) 2023-08-17
EP3232774A1 (en) 2017-10-25
BR112017013104A2 (pt) 2018-05-15
US11326184B2 (en) 2022-05-10
RU2707137C2 (ru) 2019-11-22
WO2016100819A1 (en) 2016-06-23
ES2947714T9 (es) 2024-03-14
US20220235381A1 (en) 2022-07-28
NZ732895A (en) 2022-05-27
KR20170093246A (ko) 2017-08-14
CA2971213A1 (en) 2016-06-23
SG11201704646YA (en) 2017-07-28
ES2760508T3 (es) 2020-05-14
RU2017124909A (ru) 2019-01-21
EP3653048B9 (en) 2023-10-04
EP3653048B1 (en) 2023-06-07
EP3653048C0 (en) 2023-06-07
US20160177339A1 (en) 2016-06-23

Similar Documents

Publication Publication Date Title
MX2017008190A (es) Metodos y composiciones para la modificacion genetica dirigida a traves de direccionamiento multiple de una sola etapa.
CY1125385T1 (el) Συστημα πολλαπλων φορεων και χρησεις αυτου
CY1122940T1 (el) Μη ανθρωπινα ζωα που εχουν ενα εξανθρωπισμενο γονιδιο προγραμματισμενου κυτταρικου θανατου 1
CY1122897T1 (el) Μεθοδοι και συνθεσεις για την τροποποιηση ενος στοχευομενου τοπου
CY1120572T1 (el) Στοχευση μεσω νουκλεασης με μεγαλους φορεις στοχευσης
MX388127B (es) Metodos y composiciones para la modificacion dirigida de un genoma.
BR112018005937A2 (pt) conjugados de oligonucleotídeo de afinidade e usos destes
CY1122850T1 (el) Υπερδιακλαδισμενος πολυ (βητα-αμινοεστερας) για γονιδιακη θεραπεια
CO2017000357A2 (es) Ácidos nucleicos antisentido
PE20150336A1 (es) Metodos y composiciones para la modificacion de adn objetivo dirigida por arn y para la modulacion de la transcripcion dirigida por arn
MX2018007519A (es) Alteracion dirigida del receptor celular del complejo mayor de histocompatibilidad (mhc).
MX2017007127A (es) Composiciones que comprenden polipeptidos que tienen actividad xilanasa y polipeptidos que tienen actividad arabinofuranosidasa.
EP3562492A4 (en) GENETICALLY MODIFIED NATURAL KILLER CELLS
MX2017011679A (es) Polipeptidos de transposasa y sus usos.
CO2017007335A2 (es) Supresión del gen de la huntingtina inducida por la arni
CA3010891A1 (en) Recombinogenic nucleic acid strands in situ
DOP2017000094A (es) Composiciones y métodos para inhibir la expresión del gen hao1 (hidroxiácido-oxidasa 1 (glicolato-oxidasa))
AR101845A1 (es) Anticuerpos anti-her2 e inmunoconjugados
MX2018000307A (es) Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
AR100216A1 (es) Edición de genes multiplexados
BR112019000693A2 (pt) composições de muc1- car e métodos para uso
BR112018071221A2 (pt) composições e métodos para programar células terapêuticas usando nanocarreadores de ácido nucleico alvejados
EA201790505A1 (ru) Соединение, нацеленое на ил-23а и фно-альфа, и его применение
BR112019005748A2 (pt) afinidade-conjugados de oligonucleotídeo e usos destes
PH12016502024B1 (en) Tonoplast proton/sugar antiporter proteins and the use thereof to increase the saccharose concentration in a saccharose storage organ of plants