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PL3152312T3 - Sposoby i kompozycje do modyfikowania docelowego locus - Google Patents

Sposoby i kompozycje do modyfikowania docelowego locus

Info

Publication number
PL3152312T3
PL3152312T3 PL15729724T PL15729724T PL3152312T3 PL 3152312 T3 PL3152312 T3 PL 3152312T3 PL 15729724 T PL15729724 T PL 15729724T PL 15729724 T PL15729724 T PL 15729724T PL 3152312 T3 PL3152312 T3 PL 3152312T3
Authority
PL
Poland
Prior art keywords
modifying
compositions
methods
targeted locus
locus
Prior art date
Application number
PL15729724T
Other languages
English (en)
Inventor
Wojtek Auerbach
David Frendewey
Gustavo Droguett
Anthony Gagliardi
Junko Kuno
David M. Valenzuela
Original Assignee
Regeneron Pharmaceuticals, Inc.
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 Pharmaceuticals, Inc. filed Critical Regeneron Pharmaceuticals, Inc.
Publication of PL3152312T3 publication Critical patent/PL3152312T3/pl

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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
    • 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
    • C12N15/63—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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • 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)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Environmental Sciences (AREA)
  • Mycology (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (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)
PL15729724T 2014-06-06 2015-06-05 Sposoby i kompozycje do modyfikowania docelowego locus PL3152312T3 (pl)

Applications Claiming Priority (4)

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
EP15729724.3A EP3152312B1 (en) 2014-06-06 2015-06-05 Methods and compositions for modifying a targeted locus

Publications (1)

Publication Number Publication Date
PL3152312T3 true PL3152312T3 (pl) 2020-08-10

Family

ID=53404960

Family Applications (1)

Application Number Title Priority Date Filing Date
PL15729724T PL3152312T3 (pl) 2014-06-06 2015-06-05 Sposoby i kompozycje do modyfikowania docelowego locus

Country Status (24)

Country Link
US (4) US10106820B2 (pl)
EP (2) EP3708671A1 (pl)
JP (2) JP6688231B2 (pl)
KR (1) KR102374379B1 (pl)
CN (2) CN106795521B (pl)
AU (1) AU2015269187B2 (pl)
BR (1) BR112016028564A2 (pl)
CA (1) CA2950173C (pl)
CY (1) CY1122897T1 (pl)
DK (1) DK3152312T3 (pl)
ES (1) ES2784754T3 (pl)
HR (1) HRP20200529T1 (pl)
HU (1) HUE049776T2 (pl)
IL (1) IL249042B (pl)
LT (1) LT3152312T (pl)
MX (1) MX2016016133A (pl)
NZ (1) NZ727481A (pl)
PL (1) PL3152312T3 (pl)
PT (1) PT3152312T (pl)
RS (1) RS60359B1 (pl)
RU (1) RU2704283C9 (pl)
SG (2) SG10201913804WA (pl)
SI (1) SI3152312T1 (pl)
WO (1) WO2015188109A1 (pl)

Families Citing this family (70)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
BR112014026294B1 (pt) 2012-04-25 2021-11-23 Regeneron Pharmaceuticals, Inc Método para modificar um lócus genômico alvo em uma célula tronco embrionária (es) de camundongo
KR102209979B1 (ko) 2013-02-20 2021-02-01 리제너론 파마슈티칼스 인코포레이티드 랫트의 유전자 변형
SI2986729T1 (sl) 2013-04-16 2019-02-28 Regeneron Pharmaceuticals, Inc. Ciljana sprememba genoma podgane
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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
RS58159B1 (sr) 2013-12-11 2019-03-29 Regeneron Pharma Postupci i kompozicije za ciljanu modifikaciju genoma
CA2933433C (en) 2013-12-11 2020-11-17 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
SG10201913804WA (en) 2014-06-06 2020-03-30 Regeneron Pharma Methods and compositions for modifying a targeted locus
KR102386101B1 (ko) 2014-06-26 2022-04-14 리제너론 파마슈티칼스 인코포레이티드 표적화된 유전자 변형을 위한 방법 및 그 조성물, 및 사용 방법
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
SG11201702309RA (en) 2014-10-15 2017-04-27 Regeneron Pharma Methods and compositions for generating or maintaining pluripotent cells
US10457960B2 (en) 2014-11-21 2019-10-29 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide RNAs
ES2760508T3 (es) 2014-12-19 2020-05-14 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida a través de la transformación múltiple en una sola etapa
MX2018004146A (es) 2015-10-05 2018-11-09 Prec Biosciences Inc Células modificadas genéticamente que comprenden un gen mano modificado de la región constante alfa del receptor de células t.
ES2768984T3 (es) 2015-10-05 2020-06-24 Prec Biosciences Inc Meganucleasas diseñadas con secuencias de reconocimiento encontradas en el gen de la región constante alfa del receptor de células T humanas
CA3002827A1 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
KR20220047898A (ko) * 2016-03-11 2022-04-19 2세븐티 바이오, 인코포레이티드 게놈 편집 면역 효과기 세포
WO2017180989A2 (en) 2016-04-15 2017-10-19 Memorial Sloan Kettering Cancer Center Transgenic t cell and chimeric antigen receptor t cell compositions and related methods
KR102356542B1 (ko) * 2016-05-20 2022-01-28 리제너론 파마슈티칼스 인코포레이티드 다중 가이드 RNAs를 이용한 면역학적 내성 파괴 방법
SI3462853T1 (sl) 2016-06-03 2023-05-31 Regeneron Pharmaceuticals, Inc. Glodavci, ki izražajo eksogeno terminalno deoksinukleotidiltransferazo
CN107513538A (zh) * 2016-06-17 2017-12-26 北京大学 基因敲除方法
ES2817973T3 (es) 2016-07-01 2021-04-08 Microsoft Technology Licensing Llc Almacenamiento a través de la edición iterativa del ADN
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
KR20230095129A (ko) 2016-08-03 2023-06-28 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
CN114369157A (zh) 2016-11-04 2022-04-19 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
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
KR20190104400A (ko) 2017-01-19 2019-09-09 오픈 모노클로날 테크놀로지, 인코포레이티드 다중 중쇄 면역글로불린 유전자좌를 갖는 트랜스제닉 설치류 기원의 인간 항체
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
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
JP7177076B2 (ja) * 2017-03-16 2022-11-22 ファイザー・インク チロシン原栄養性
US11268082B2 (en) 2017-03-23 2022-03-08 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
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
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
EP3652320A4 (en) * 2017-07-12 2021-04-14 Mayo Foundation for Medical Education and Research MATERIALS AND PROCEDURES FOR EFFICIENT TARGETED KNOCK-IN OR GENERAL REPLACEMENT
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
US12012596B2 (en) * 2017-08-21 2024-06-18 Tokushima University Target sequence specific alteration technology using nucleotide target recognition
EP3676376A2 (en) 2017-08-30 2020-07-08 President and Fellows of Harvard College High efficiency base editors comprising gam
JP2020534812A (ja) * 2017-09-08 2020-12-03 ライフ テクノロジーズ コーポレイション 改良された相同組換えおよびその組成物のための方法
EP3697906A1 (en) 2017-10-16 2020-08-26 The Broad Institute, Inc. Uses of adenosine base editors
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
US20210032621A1 (en) * 2018-01-23 2021-02-04 Institute For Basic Science Extended single guide rna and use thereof
CN111902164A (zh) * 2018-02-01 2020-11-06 同源药物公司 用于恢复pah基因功能的腺相关病毒组合物及其使用方法
CA3093850A1 (en) 2018-03-26 2019-10-03 Regeneron Pharmaceuticals, Inc. Humanized rodents for testing therapeutic agents
KR102617818B1 (ko) 2018-04-12 2023-12-27 프리시젼 바이오사이언시스 인코포레이티드 인간 t 세포 수용체 알파 불변 영역 유전자에 대한 특이성을 갖는 최적화된 조작된 뉴클레아제
CA3099316A1 (en) * 2018-05-04 2019-11-07 Locus Biosciences, Inc. Methods and compositions for killing a target bacterium
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
JP7233545B2 (ja) * 2018-12-30 2023-03-06 エフ． ホフマン－ラ ロシュ アーゲー 標的タンパク質への検出可能なタグのＣＲＩＳＰＲ／Ｃａｓ制御組み込みに基づく細胞の選択方法
WO2020191239A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020206134A1 (en) * 2019-04-04 2020-10-08 Regeneron Pharmaceuticals, Inc. Methods for scarless introduction of targeted modifications into targeting vectors
US20220220542A1 (en) * 2019-05-13 2022-07-14 Rapid Genomics Llc Capture and analysis of target genomic regions
WO2021016608A1 (en) * 2019-07-25 2021-01-28 Precision Biosciences, Inc. Compositions and methods for sequential stacking of nucleic acid sequences into a genomic locus
US20230046246A1 (en) * 2019-12-13 2023-02-16 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
CN116096873A (zh) 2020-05-08 2023-05-09 布罗德研究所股份有限公司 同时编辑靶标双链核苷酸序列的两条链的方法和组合物
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
US20240018493A1 (en) * 2020-11-10 2024-01-18 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
JP2024533683A (ja) * 2021-09-24 2024-09-12 イムノカン バイオテック カンパニー リミテッド 大型染色体の導入方法と改変染色体およびそれを用いた生物

Family Cites Families (102)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1992003917A1 (en) 1990-08-29 1992-03-19 Genpharm International 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
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
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
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
JP2004537260A (ja) 2000-12-07 2004-12-16 サンガモ バイオサイエンシーズ， インコーポレイテッド ジンクフィンガータンパク質による血管新生の調節
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
US20030082591A1 (en) 2001-07-24 2003-05-01 Donald Awrey Methods for gene disruption and uses thereof
BR0307383A (pt) 2002-01-23 2005-04-26 Univ Utah Res Found Mutagênese cromossomica alvo utilizando nucleases de ramificações de zinco
ATE372379T1 (de) 2002-03-15 2007-09-15 Cellectis Hybride and einzelkettige meganukleasen und deren anwendungen
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
US9447434B2 (en) 2002-09-05 2016-09-20 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
AU2004204509A1 (en) 2003-01-13 2004-07-29 Mario R. Capecchi Persistent expression of candidate molecule in proliferating stem and progenitor cells for delivery of therapeutic products
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
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
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
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
US20070004041A1 (en) 2005-06-30 2007-01-04 Codon Devices, Inc. Heirarchical 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
EP3070169B1 (en) 2006-12-14 2018-05-09 Dow AgroSciences LLC Optimized non-canonical zinc finger proteins
US7771967B2 (en) 2006-12-22 2010-08-10 The J. David Gladstone Institutes Nucleic acid encoding apolipoprotein E-I3
CA2684378C (en) 2007-04-26 2016-11-29 Sangamo Biosciences, Inc. Targeted integration into the ppp1r12c locus
DK2336329T3 (da) 2007-06-01 2013-01-07 Omt Inc Sammensætninger og fremgangsmåder til hæmning af endogene immunglobulingener og til produktion af transgene, humane, idiotypiske antistoffer
WO2009076464A2 (en) 2007-12-10 2009-06-18 Aliva Biopharmaceuticals, Inc. Methods for sequential replacement of targeted region by homologous recombination
EP3156494B8 (en) 2008-12-04 2018-09-19 Sangamo Therapeutics, 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
EP2408921B1 (en) 2009-03-20 2017-04-19 Sangamo BioSciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
EP2425018A4 (en) 2009-04-30 2013-06-05 Univ Columbia IN VIVO CONSTRUCTION OF DNA BY HOMOLOGOUS RECOMBINATION
US8772008B2 (en) 2009-05-18 2014-07-08 Sangamo Biosciences, Inc. Methods and compositions for increasing nuclease activity
PL2517556T3 (pl) * 2009-07-08 2016-03-31 Kymab Ltd Sposób miejscowo specyficznej rekombinacji, gryzonie i komórki gryzonia zdolne do ekspresji chimerycznych przeciwciał lub łańcuchów
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
DK3147362T3 (en) 2009-10-29 2019-04-08 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
JP5807862B2 (ja) 2009-12-01 2015-11-10 国立研究開発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
PT2510096E (pt) 2009-12-10 2015-02-04 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector tal
EP2813572B1 (en) 2009-12-21 2016-04-27 Keygene N.V. Improved techniques for transfecting protoplasts
US9695432B2 (en) 2010-01-22 2017-07-04 Dow Agrosciences Llc Excision of transgenes in genetically modified organisms
WO2011100058A1 (en) 2010-02-09 2011-08-18 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
PL2579711T3 (pl) 2010-06-11 2019-09-30 Regeneron Pharmaceuticals, Inc. Wytwarzanie płodnych zwierzęcych samic xy z komórek es xy
EP3366125A1 (en) * 2010-06-17 2018-08-29 Kymab Limited Animal models and therapeutic molecules
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
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
EP3424947B1 (en) * 2011-10-28 2020-11-25 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
BR112014026294B1 (pt) * 2012-04-25 2021-11-23 Regeneron Pharmaceuticals, Inc Método para modificar um lócus genômico alvo em uma célula tronco embrionária (es) de camundongo
US10174331B2 (en) 2012-05-07 2019-01-08 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
DK3241902T3 (en) 2012-05-25 2018-05-07 Univ California METHODS AND COMPOSITIONS FOR RNA DIRECTIVE TARGET DNA MODIFICATION AND FOR RNA DIRECTIVE MODULATION OF TRANSCRIPTION
SG11201503059XA (en) 2012-10-23 2015-06-29 Toolgen Inc 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
DK3360964T3 (da) 2012-12-06 2019-10-28 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
DK2898075T3 (en) 2012-12-12 2016-06-27 Broad Inst Inc CONSTRUCTION AND OPTIMIZATION OF IMPROVED SYSTEMS, PROCEDURES AND ENZYME COMPOSITIONS FOR SEQUENCE MANIPULATION
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
AU2013363194B2 (en) 2012-12-17 2019-05-16 President And Fellows Of Harvard College RNA-guided human genome engineering
WO2014104878A1 (en) 2012-12-27 2014-07-03 Keygene N.V. Method for removing genetic linkage in a plant
KR102209979B1 (ko) 2013-02-20 2021-02-01 리제너론 파마슈티칼스 인코포레이티드 랫트의 유전자 변형
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
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
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US9234213B2 (en) * 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
AU2014235968B2 (en) * 2013-03-21 2018-05-24 Ospedale San Raffaele Srl Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
CN105518146B (zh) 2013-04-04 2022-07-15 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
SI2986729T1 (sl) 2013-04-16 2019-02-28 Regeneron Pharmaceuticals, Inc. Ciljana sprememba genoma podgane
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
DK3004349T3 (en) 2013-05-29 2018-06-06 Cellectis Sa A method for producing precise DNA cleavage using CAS9 nickase activity
SG11201510297QA (en) 2013-06-19 2016-01-28 Sigma Aldrich Co Llc Targeted integration
ES2681622T3 (es) 2013-09-18 2018-09-14 Kymab Limited Métodos, células y organismos
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
CA2933433C (en) 2013-12-11 2020-11-17 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
AU2014363479A1 (en) 2013-12-13 2016-06-23 Cellectis New method of selection of algal-transformed cells using nuclease
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
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
SG10201913804WA (en) 2014-06-06 2020-03-30 Regeneron Pharma Methods and compositions for modifying a targeted locus
KR102386101B1 (ko) 2014-06-26 2022-04-14 리제너론 파마슈티칼스 인코포레이티드 표적화된 유전자 변형을 위한 방법 및 그 조성물, 및 사용 방법
SG11201702309RA (en) 2014-10-15 2017-04-27 Regeneron Pharma Methods and compositions for generating or maintaining pluripotent cells
US10457960B2 (en) 2014-11-21 2019-10-29 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide RNAs
ES2760508T3 (es) 2014-12-19 2020-05-14 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida a través de la transformación múltiple en una sola etapa
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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