ES2784754T3 - Métodos y composiciones para modificar un locus objetivo - Google Patents

Métodos y composiciones para modificar un locus objetivo Download PDF

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Publication number
ES2784754T3
ES2784754T3 ES15729724T ES15729724T ES2784754T3 ES 2784754 T3 ES2784754 T3 ES 2784754T3 ES 15729724 T ES15729724 T ES 15729724T ES 15729724 T ES15729724 T ES 15729724T ES 2784754 T3 ES2784754 T3 ES 2784754T3
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cell
polynucleotide
target
nuclease
nucleic acid
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English (en)
Spanish (es)
Inventor
Wojtek Auerbach
David Frendewey
Gustavo Droguett
Anthony Gagliardi
Junko Kuno
David M Valenzuela
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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    • 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
    • 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
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • 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
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • 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
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • 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
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0387—Animal model for diseases of the immune system
    • 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
    • C12N2015/8527—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety

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  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Environmental Sciences (AREA)
  • Mycology (AREA)
  • Veterinary Medicine (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
ES15729724T 2014-06-06 2015-06-05 Métodos y composiciones para modificar un locus objetivo Active ES2784754T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462008832P 2014-06-06 2014-06-06
US201462017916P 2014-06-27 2014-06-27
PCT/US2015/034503 WO2015188109A1 (en) 2014-06-06 2015-06-05 Methods and compositions for modifying a targeted locus

Publications (1)

Publication Number Publication Date
ES2784754T3 true ES2784754T3 (es) 2020-09-30

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ES15729724T Active ES2784754T3 (es) 2014-06-06 2015-06-05 Métodos y composiciones para modificar un locus objetivo
ES20155939T Active ES3019688T3 (en) 2014-06-06 2015-06-05 Methods and compositions for modifying a targeted locus

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ES20155939T Active ES3019688T3 (en) 2014-06-06 2015-06-05 Methods and compositions for modifying a targeted locus

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US (4) US10106820B2 (cg-RX-API-DMAC7.html)
EP (2) EP3708671B1 (cg-RX-API-DMAC7.html)
JP (2) JP6688231B2 (cg-RX-API-DMAC7.html)
KR (1) KR102374379B1 (cg-RX-API-DMAC7.html)
CN (2) CN106795521B (cg-RX-API-DMAC7.html)
AU (1) AU2015269187B2 (cg-RX-API-DMAC7.html)
BR (1) BR112016028564A2 (cg-RX-API-DMAC7.html)
CA (1) CA2950173C (cg-RX-API-DMAC7.html)
CY (1) CY1122897T1 (cg-RX-API-DMAC7.html)
DK (1) DK3152312T3 (cg-RX-API-DMAC7.html)
ES (2) ES2784754T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20200529T1 (cg-RX-API-DMAC7.html)
HU (1) HUE049776T2 (cg-RX-API-DMAC7.html)
IL (1) IL249042B (cg-RX-API-DMAC7.html)
LT (1) LT3152312T (cg-RX-API-DMAC7.html)
MX (1) MX385689B (cg-RX-API-DMAC7.html)
NZ (1) NZ727481A (cg-RX-API-DMAC7.html)
PL (1) PL3152312T3 (cg-RX-API-DMAC7.html)
PT (1) PT3152312T (cg-RX-API-DMAC7.html)
RS (1) RS60359B1 (cg-RX-API-DMAC7.html)
RU (1) RU2704283C9 (cg-RX-API-DMAC7.html)
SG (2) SG10201913804WA (cg-RX-API-DMAC7.html)
SI (1) SI3152312T1 (cg-RX-API-DMAC7.html)
SM (1) SMT202000264T1 (cg-RX-API-DMAC7.html)
WO (1) WO2015188109A1 (cg-RX-API-DMAC7.html)

Families Citing this family (79)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
PT2847335T (pt) 2012-04-25 2018-09-28 Regeneron Pharma Alvejamento mediado por nuclease com vetores de alvejamento grandes
EP3561050B1 (en) 2013-02-20 2021-12-08 Regeneron Pharmaceuticals, Inc. Genetic modification of rats
HUE040575T2 (hu) 2013-04-16 2019-03-28 Regeneron Pharma A patkány genom célzott módosítása
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
SMT201800653T1 (it) 2013-12-11 2019-01-11 Regeneron Pharma Metodi e composizioni per la modificazione indirizzata a bersaglio di un genoma
US9546384B2 (en) 2013-12-11 2017-01-17 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a mouse genome
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
LT3152312T (lt) 2014-06-06 2020-04-27 Regeneron Pharmaceuticals, Inc. Tikslinio lokuso modifikavimo būdai ir kompozicijos
EP3461885B1 (en) 2014-06-26 2021-10-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
JP2017536811A (ja) 2014-10-15 2017-12-14 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 多能性細胞の樹立又は維持のための方法及び組成物
HUE044907T2 (hu) 2014-11-21 2019-11-28 Regeneron Pharma Módszerek és készítmények célzott genetikai módosításhoz, párosított mutató RNS-ek használatával
NZ732895A (en) 2014-12-19 2022-05-27 Regeneron Pharma Methods and compositions for targeted genetic modification through single-step multiple targeting
ES2920525T3 (es) 2015-10-05 2022-08-05 Prec Biosciences Inc Células genéticamente modificadas que comprenden un gen de región constante alfa del receptor de linfocitos T humano modificado
CA3001008A1 (en) 2015-10-05 2017-04-13 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
US20190241910A1 (en) * 2016-03-11 2019-08-08 Bluebird Bio, Inc. Genome edited immune effector cells
IL299000A (en) * 2016-04-04 2023-02-01 Eth Zuerich A mammalian cell line for protein production and a breeding library
AU2017250769B2 (en) 2016-04-15 2022-07-14 Memorial Sloan Kettering Cancer Center Transgenic T cell and chimeric antigen receptor T cell compositions and related methods
AU2017268458B2 (en) 2016-05-20 2022-07-21 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide RNAS
EP3462853B1 (en) 2016-06-03 2023-03-01 Regeneron Pharmaceuticals, Inc. Rodents expressing exogenous terminal deoxynucleotidyltransferase
CN107513538A (zh) * 2016-06-17 2017-12-26 北京大学 基因敲除方法
US11359234B2 (en) 2016-07-01 2022-06-14 Microsoft Technology Licensing, Llc Barcoding sequences for identification of gene expression
US20180004537A1 (en) 2016-07-01 2018-01-04 Microsoft Technology Licensing, Llc Molecular State Machines
US10669558B2 (en) 2016-07-01 2020-06-02 Microsoft Technology Licensing, Llc Storage through iterative DNA editing
KR102827276B1 (ko) 2016-08-03 2025-07-01 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN110214180A (zh) 2016-10-14 2019-09-06 哈佛大学的校长及成员们 核碱基编辑器的aav递送
PL3766343T3 (pl) 2016-11-04 2022-09-26 Regeneron Pharmaceuticals, Inc. Zwierzęta inne niż człowiek posiadające zmodyfikowany sposobami inżynierii locus łańcucha lekkiego lambda immunoglobuliny
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
CN110662421B (zh) 2017-01-19 2023-03-24 欧莫诺艾比公司 来自具有多个重链免疫球蛋白基因座的转基因啮齿类动物的人抗体
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
WO2018151514A1 (ko) * 2017-02-16 2018-08-23 고려대학교 산학협력단 골재생 효능이 우수한 골질환 예방 또는 치료용 조성물
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
JP2020510038A (ja) 2017-03-09 2020-04-02 プレジデント アンド フェローズ オブ ハーバード カレッジ がんワクチン
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
EP3596206A1 (en) * 2017-03-16 2020-01-22 Pfizer Inc Tyrosine prototrophy
GB2575930A (en) 2017-03-23 2020-01-29 Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
CA3068465A1 (en) 2017-06-30 2019-01-03 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
CN111094573A (zh) * 2017-07-12 2020-05-01 梅约医学教育与研究基金会 有效靶向敲入或基因置换的材料和方法
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
IL272688B2 (en) * 2017-08-21 2025-03-01 Univ Tokushima Target sequence-specific modification technology, which uses nucleotide target recognition
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
WO2019051237A1 (en) * 2017-09-08 2019-03-14 Life Technologies Corporation ENHANCED RECOMBINANT RECOMBINATION METHODS AND COMPOSITIONS THEREOF
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US20210017544A1 (en) * 2017-12-22 2021-01-21 Novozymes A/S Counter-Selection by Inhibition of Conditionally Essential Genes
CN107904208B (zh) * 2017-12-25 2019-11-01 云舟生物科技（广州）有限公司 细胞表型研究用的细胞克隆及其筛选方法和应用
KR102839528B1 (ko) * 2018-01-23 2025-07-29 기초과학연구원 연장된 단일 가이드 rna 및 그 용도
PE20212076A1 (es) 2018-02-01 2021-10-26 Homology Medicines Inc Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas
AU2019242586B2 (en) 2018-03-26 2025-09-11 Regeneron Pharmaceuticals, Inc. Humanized rodents for testing therapeutic agents
CA3095795A1 (en) 2018-04-12 2019-10-17 Precision Biosciences, Inc. Optimized engineered nucleases having specificity for the human t cell receptor alpha constant region gene
WO2019213592A1 (en) * 2018-05-04 2019-11-07 Locus Biosciences, Inc. Methods and compositions for killing a target bacterium
WO2019222545A1 (en) 2018-05-16 2019-11-21 Synthego Corporation Methods and systems for guide rna design and use
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
CN117904208A (zh) * 2018-12-30 2024-04-19 豪夫迈·罗氏有限公司 基于可检测标签与靶蛋白的CRISPR/Cas控制的整合而选择细胞的方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
CN113795588B (zh) * 2019-04-04 2025-02-25 瑞泽恩制药公司 用于在靶向性载体中无瘢痕引入靶向修饰的方法
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN114286861B (zh) * 2019-05-13 2024-12-06 Lgc基因组学有限责任公司 捕获和分析靶基因组区域
US20220273715A1 (en) * 2019-07-25 2022-09-01 Precision Biosciences, Inc. Compositions and methods for sequential stacking of nucleic acid sequences into a genomic locus
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021119594A1 (en) * 2019-12-13 2021-06-17 The Regents Of The University Of California Reducing antibiotic resistance in bacteria using pro-active genetics
IL271656A (en) 2019-12-22 2021-06-30 Yeda Res & Dev System and methods for identifying cells that have undergone genome editing
AU2021267940A1 (en) 2020-05-08 2022-12-08 President And Fellows Of Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
WO2022104344A2 (en) * 2020-11-10 2022-05-19 The Board Of Trustees Of The Leland Stanford Junior University Knock-in of large dna for long-term high genomic expression
CN114763559B (zh) * 2021-01-15 2024-07-19 中国农业大学 一种不依赖于同源重组的靶向性基因捕获系统及其应用
US20250027116A1 (en) * 2021-09-24 2025-01-23 Immunocan Biotech Co. Ltd. Methods for large-size chromosomal transfer and modified chromosomes and organisims using same
WO2024231883A1 (en) * 2023-05-10 2024-11-14 Takeda Pharmaceutical Company Limited Re-editable templates, cells, compositions and methods of making

Family Cites Families (103)

* 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
PT1538206E (pt) * 1997-09-16 2010-04-12 Centocor Ortho Biotech Inc Método para a síntese química e construção completa de genes e genomas
WO2000039316A1 (en) 1998-12-31 2000-07-06 The J. David Gladstone Institutes 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
US6503717B2 (en) 1999-12-06 2003-01-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US20030104526A1 (en) 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
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
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US6586251B2 (en) 2000-10-31 2003-07-01 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
WO2002046412A2 (en) 2000-12-07 2002-06-13 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
ES2409080T3 (es) 2001-01-22 2013-06-24 Sangamo Biosciences Inc. Proteínas de unión con dedos de zinc modificadas
WO2002057308A2 (en) 2001-01-22 2002-07-25 Sangamo Biosciences, Inc. Zinc finger polypeptides and their use
AUPR451401A0 (en) 2001-04-20 2001-05-24 Monash University A method of nuclear transfer
WO2003010333A2 (en) 2001-07-24 2003-02-06 Affinium Pharmaceuticals Inc. Methods for gene disruption and uses thereof
CN100575485C (zh) 2002-01-23 2009-12-30 犹他大学研究基金会 使用锌指核酸酶的定向染色体诱变
EP1485475B2 (en) 2002-03-15 2017-09-20 Cellectis Hybrid and single chain meganucleases and use thereof
EP2368982A3 (en) 2002-03-21 2011-10-12 Sangamo BioSciences, 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
JP2006502748A (ja) 2002-09-05 2006-01-26 カリフォルニア インスティテュート オブ テクノロジー 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法
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
WO2004063356A2 (en) 2003-01-13 2004-07-29 Rao Mahendra S 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
EP1789095A2 (en) 2004-09-16 2007-05-30 Sangamo Biosciences Inc. Compositions and methods for protein production
PL1802193T3 (pl) 2004-10-19 2014-09-30 Regeneron Pharma Sposób wytwarzania myszy homozygotycznej 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
WO2006097784A1 (en) 2005-03-15 2006-09-21 Cellectis I-crei meganuclease variants with modified specificity, method of preparation and uses thereof
WO2006097854A1 (en) 2005-03-15 2006-09-21 Cellectis Heterodimeric meganucleases and use thereof
WO2007005053A1 (en) 2005-06-30 2007-01-11 Codon Devices, Inc. Hierarchical assembly methods for genome engineering
GB0615327D0 (en) 2006-03-30 2006-09-13 Univ Edinburgh Culture medium containing kinase inhibitors and uses thereof
EP2003960B1 (en) 2006-03-31 2015-06-10 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
PT2415873E (pt) 2006-12-14 2015-03-31 Sangamo Biosciences Inc Proteínas com dedos de zinco não canónicas optimizadas
US7771967B2 (en) 2006-12-22 2010-08-10 The J. David Gladstone Institutes Nucleic acid encoding apolipoprotein E-I3
EP2137310B1 (en) 2007-04-26 2010-11-24 Sangamo BioSciences, Inc. Targeted integration into the ppp1r12c locus
SI2602323T1 (en) 2007-06-01 2018-05-31 Open Monoclonal Technology, Inc. Compositions and procedures for inhibiting endogenous immunoglobin gene and the formation of transgenic human idiopathic antibodies
US20110119779A1 (en) 2007-12-10 2011-05-19 Aliva Biopharmaceuticals, Inc. Methods for sequential replacement of targeted region by homologous recombination
CA2745031C (en) 2008-12-04 2018-08-14 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
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
CA2755192C (en) 2009-03-20 2018-09-11 Sangamo Biosciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
WO2010127111A1 (en) 2009-04-30 2010-11-04 The Trustees Of Comlumbia University In The City Of New York In vivo assembly of dna via homologous recombination
US8772008B2 (en) 2009-05-18 2014-07-08 Sangamo Biosciences, Inc. Methods and compositions for increasing nuclease activity
EP2517556B2 (en) * 2009-07-08 2023-03-22 Kymab Limited Site-Specific Recombination Method, Rodents & Rodent Cells capable of Expressing Chimaeric Antibodies or Chains
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
JP5908405B2 (ja) 2009-10-29 2016-04-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 多機能性対立遺伝子
US20120315670A1 (en) 2009-11-02 2012-12-13 Gen9, Inc. Compositions and Methods for the Regulation of Multiple Genes of Interest in a Cell
EP2508595B1 (en) 2009-12-01 2016-11-23 National Cancer Center Method for constructing chimeric rat using rat embryonic stem cells
HUE041436T2 (hu) 2009-12-10 2019-05-28 Univ Minnesota Tal-effektor-közvetített DNS-módosítás
AU2010335107B2 (en) 2009-12-21 2014-07-03 Keygene N.V. Improved techniques for transfecting protoplasts
KR101951489B1 (ko) 2010-01-22 2019-04-22 다우 아그로사이언시즈 엘엘씨 유전적으로 변형된 유기체에서 도입유전자의 절제
JP2013518602A (ja) 2010-02-09 2013-05-23 サンガモ バイオサイエンシーズ， インコーポレイテッド 部分的に一本鎖のドナー分子による標的化ゲノム改変
GB201009732D0 (en) 2010-06-10 2010-07-21 Gene Bridges Gmbh Direct cloning
CN102933074B (zh) 2010-06-11 2015-04-08 瑞泽恩制药公司 由xy es细胞制备能育的xy雌性动物
SG186238A1 (en) * 2010-06-17 2013-01-30 Kymab Ltd Animal models and therapeutic molecules
CA2805442C (en) 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus
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
IN2014CN03890A (cg-RX-API-DMAC7.html) 2011-10-28 2015-10-16 Regeneron Pharma
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
PT2847335T (pt) 2012-04-25 2018-09-28 Regeneron Pharma Alvejamento mediado por nuclease com vetores de alvejamento grandes
AU2013259647B2 (en) 2012-05-07 2018-11-08 Corteva Agriscience Llc Methods and compositions for nuclease-mediated targeted integration of transgenes
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
CN110643600A (zh) 2012-10-23 2020-01-03 基因工具股份有限公司 用于切割靶dna的系统及其用途
LT3138910T (lt) 2012-12-06 2017-11-10 Sigma-Aldrich Co. Llc Crispr pagrįstas genomo modifikavimas ir reguliavimas
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
ES2786193T3 (es) 2012-12-12 2020-10-09 Broad Inst Inc Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
SG10201704932UA (en) 2012-12-17 2017-07-28 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
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
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
EP2975942B1 (en) * 2013-03-21 2018-08-08 Sangamo Therapeutics, Inc. Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases
CN115261411A (zh) 2013-04-04 2022-11-01 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
HUE040575T2 (hu) 2013-04-16 2019-03-28 Regeneron Pharma A patkány genom célzott módosítása
CA2910489A1 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
AU2014273082B2 (en) 2013-05-29 2018-11-08 Cellectis A method for producing precise DNA cleavage using Cas9 nickase activity
JP2016523084A (ja) 2013-06-19 2016-08-08 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーＳｉｇｍａ−Ａｌｄｒｉｃｈ Ｃｏ．， ＬＬＣ 標的組込み
DE202014010413U1 (de) 2013-09-18 2015-12-08 Kymab Limited Zellen und Organismen
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9546384B2 (en) 2013-12-11 2017-01-17 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a mouse genome
US20160312233A1 (en) 2013-12-13 2016-10-27 Cellectis New method of selection of algal-transformed cells using nuclease
US10233456B2 (en) 2014-01-30 2019-03-19 The Board Of Trustees Of The University Of Arkansas Method, vectors, cells, seeds and kits for stacking genes into a single genomic site
WO2015163733A1 (en) 2014-04-24 2015-10-29 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
LT3152312T (lt) 2014-06-06 2020-04-27 Regeneron Pharmaceuticals, Inc. Tikslinio lokuso modifikavimo būdai ir kompozicijos
EP3461885B1 (en) 2014-06-26 2021-10-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
JP2017536811A (ja) 2014-10-15 2017-12-14 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 多能性細胞の樹立又は維持のための方法及び組成物
HUE044907T2 (hu) 2014-11-21 2019-11-28 Regeneron Pharma Módszerek és készítmények célzott genetikai módosításhoz, párosított mutató RNS-ek használatával
NZ732895A (en) 2014-12-19 2022-05-27 Regeneron Pharma Methods and compositions for targeted genetic modification through single-step multiple targeting
GB201504223D0 (en) 2015-03-12 2015-04-29 Genome Res Ltd Biallelic genetic modification
WO2019046350A1 (en) 2017-08-30 2019-03-07 President And Fellows Of Harvard College ITERATIVE GENOMIC ASSEMBLY

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