PT3152312T - Métodos e composições para modificar um locus alvo - Google Patents

Métodos e composições para modificar um locus alvo

Info

Publication number
PT3152312T
PT3152312T PT157297243T PT15729724T PT3152312T PT 3152312 T PT3152312 T PT 3152312T PT 157297243 T PT157297243 T PT 157297243T PT 15729724 T PT15729724 T PT 15729724T PT 3152312 T PT3152312 T PT 3152312T
Authority
PT
Portugal
Prior art keywords
modifying
compositions
methods
targeted locus
locus
Prior art date
Application number
PT157297243T
Other languages
English (en)
Portuguese (pt)
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 PT3152312T publication Critical patent/PT3152312T/pt

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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

Landscapes

  • 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)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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)
  • 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)
PT157297243T 2014-06-06 2015-06-05 Métodos e composições para modificar um locus alvo PT3152312T (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462008832P 2014-06-06 2014-06-06
US201462017916P 2014-06-27 2014-06-27

Publications (1)

Publication Number Publication Date
PT3152312T true PT3152312T (pt) 2020-04-23

Family

ID=53404960

Family Applications (1)

Application Number Title Priority Date Filing Date
PT157297243T PT3152312T (pt) 2014-06-06 2015-06-05 Métodos e composições para modificar um locus alvo

Country Status (25)

Country Link
US (4) US10106820B2 (enExample)
EP (2) EP3708671B1 (enExample)
JP (2) JP6688231B2 (enExample)
KR (1) KR102374379B1 (enExample)
CN (2) CN106795521B (enExample)
AU (1) AU2015269187B2 (enExample)
BR (1) BR112016028564A2 (enExample)
CA (1) CA2950173C (enExample)
CY (1) CY1122897T1 (enExample)
DK (1) DK3152312T3 (enExample)
ES (2) ES2784754T3 (enExample)
HR (1) HRP20200529T1 (enExample)
HU (1) HUE049776T2 (enExample)
IL (1) IL249042B (enExample)
LT (1) LT3152312T (enExample)
MX (1) MX385689B (enExample)
NZ (1) NZ727481A (enExample)
PL (1) PL3152312T3 (enExample)
PT (1) PT3152312T (enExample)
RS (1) RS60359B1 (enExample)
RU (1) RU2704283C9 (enExample)
SG (2) SG11201609634YA (enExample)
SI (1) SI3152312T1 (enExample)
SM (1) SMT202000264T1 (enExample)
WO (1) WO2015188109A1 (enExample)

Families Citing this family (82)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
ES2683071T3 (es) 2012-04-25 2018-09-24 Regeneron Pharmaceuticals, Inc. Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento
MX2015010841A (es) 2013-02-20 2016-05-09 Regeneron Pharma Modificacion genetica de ratas.
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
EP3460063B1 (en) 2013-12-11 2024-03-13 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
DK3152312T3 (da) * 2014-06-06 2020-04-06 Regeneron Pharma Fremgangsmåder og sammensætninger til modifikation af et mållocus
DK3161128T3 (en) 2014-06-26 2018-11-05 Regeneron Pharma METHODS AND COMPOSITIONS FOR TARGETED GENTICAL MODIFICATIONS AND PROCEDURES FOR USE THEREOF
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
BR112017007770A2 (pt) 2014-10-15 2018-01-16 Regeneron Pharma cultura in vitro, população de hipscs, método para modificar um locus-alvo genômico, e, hipsc.
EP3221457B1 (en) 2014-11-21 2019-03-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
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.
ES2803728T3 (es) 2015-10-05 2021-01-29 Prec Biosciences Inc Células genéticamente modificadas que comprenden un gen de región constante alfa del receptor de linfocitos T humano modificado
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
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
US20190241910A1 (en) * 2016-03-11 2019-08-08 Bluebird Bio, Inc. Genome edited immune effector cells
JP2019513415A (ja) * 2016-04-04 2019-05-30 イーティーエッチ チューリッヒ タンパク質産生及びライブラリー生成のための哺乳類細胞株
KR102614675B1 (ko) 2016-04-15 2023-12-19 메모리얼 슬로안 케터링 캔서 센터 유전자이식 t 세포 및 키메라 항원 수용체 t 세포 조성물 및 관련 방법
SG10202102885UA (en) * 2016-05-20 2021-05-28 Regeneron Pharma Methods for breaking immunological tolerance using multiple guide rnas
CN109640644B (zh) 2016-06-03 2021-10-26 瑞泽恩制药公司 表达外源末端脱氧核苷酸转移酶的非人动物
CN107513538A (zh) * 2016-06-17 2017-12-26 北京大学 基因敲除方法
CN109477130B (zh) 2016-07-01 2022-08-30 微软技术许可有限责任公司 通过迭代dna编辑的存储
US20180004537A1 (en) 2016-07-01 2018-01-04 Microsoft Technology Licensing, Llc Molecular State Machines
US11359234B2 (en) 2016-07-01 2022-06-14 Microsoft Technology Licensing, Llc Barcoding sequences for identification of gene expression
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 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
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
EP4567119A3 (en) 2016-11-04 2025-09-24 Regeneron Pharmaceuticals, Inc. Non-human animals having an engineered immunoglobulin lambda light chain locus
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
CA3050715A1 (en) 2017-01-19 2018-07-26 Open Monoclonal Technology, Inc. Human antibodies from transgenic rodents with multiple heavy chain immunoglobulin loci
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
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
WO2018167621A1 (en) * 2017-03-16 2018-09-20 Pfizer Inc. Tyrosine prototrophy
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include 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
EP4775669A2 (en) 2017-06-30 2026-07-15 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
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
WO2019039417A1 (ja) * 2017-08-21 2019-02-28 国立大学法人徳島大学 ヌクレオチド標的認識を利用した標的配列特異的改変技術
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CN111315889A (zh) 2017-09-08 2020-06-19 生命技术公司 提高同源重组的方法和其组合物
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes 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
US12391941B2 (en) 2018-01-23 2025-08-19 Institute For Basic Science Extended single guide RNA and use thereof
AU2019215152A1 (en) * 2018-02-01 2020-08-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof
IL277355B2 (en) 2018-03-26 2025-01-01 Regeneron Pharma 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
KR20210018273A (ko) * 2018-05-04 2021-02-17 로커스 바이오사이언스, 인크. 표적 박테리아를 사멸시키기 위한 방법 및 조성물
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
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
EP3902915B1 (en) * 2018-12-30 2023-08-23 F. Hoffmann-La Roche AG Method for the selection of cells based on crispr/cas-controlled integration of a detectable tag to a target protein
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
AU2020253531C1 (en) * 2019-04-04 2022-06-16 Regeneron Pharmaceuticals, Inc. Methods for scarless introduction of targeted modifications into targeting vectors
WO2020214842A1 (en) 2019-04-17 2020-10-22 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
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu 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
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
EP4262373A1 (en) 2020-12-16 2023-10-25 Regeneron Pharmaceuticals, Inc. Mice expressing humanized fc alpha receptors
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
WO2023046038A1 (en) * 2021-09-24 2023-03-30 Immunocan Biotech Co. Ltd. Methods for large-size chromosomal transfer and modified chromosomes and organisims using same
CN121079425A (zh) * 2023-05-10 2025-12-05 武田药品工业株式会社 可再编辑的模板、细胞、组合物和制备方法
WO2026106602A1 (en) * 2024-11-14 2026-05-21 Blueallele Corporation Materials and methods for redosing subjects receiving gene insertion therapeutics

Family Cites Families (104)

* 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
ES2243007T3 (es) * 1997-09-16 2005-11-16 Egea Biosciences Llc Sintesis quimica completa y sintesis de genes de genomas.
CA2354542A1 (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
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
US6586251B2 (en) 2000-10-31 2003-07-01 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US7105348B2 (en) 2000-10-31 2006-09-12 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
WO2002046412A2 (en) 2000-12-07 2002-06-13 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
US7273923B2 (en) 2001-01-22 2007-09-25 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
US7947469B2 (en) 2001-01-22 2011-05-24 Gendaq, Ltd. Modulation of HIV infection
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
ATE347593T1 (de) 2002-01-23 2006-12-15 Univ Utah Res Found Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen
US8206965B2 (en) 2002-03-15 2012-06-26 Cellectis S.A. Hybrid and single chain meganucleases and use thereof
EP1504092B2 (en) 2002-03-21 2014-06-25 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
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
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
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
KR20070060115A (ko) 2004-09-16 2007-06-12 상가모 바이오사이언스 인코포레이티드 단백질 생산을 위한 조성물 및 방법
EP1802193B1 (en) 2004-10-19 2014-04-30 Regeneron Pharmaceuticals, Inc. Method for generating a mouse homozygous for a genetic modification
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
ATE466933T1 (de) 2005-03-15 2010-05-15 Cellectis I-crei-meganuklease-varianten mit modifizierter spezifität sowie verfahren zu ihrer herstellung und verwendung
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
JP5514539B2 (ja) 2006-03-31 2014-06-04 メダレックス・リミテッド・ライアビリティ・カンパニー ヒト抗体の調製に用いるためのキメラ抗体を発現するトランスジェニック動物
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
CA2669746C (en) 2006-12-14 2017-05-16 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
US8110379B2 (en) 2007-04-26 2012-02-07 Sangamo Biosciences, Inc. Targeted integration into the PPP1R12C locus
KR101703299B1 (ko) 2007-06-01 2017-02-06 오픈 모노클로날 테크놀로지, 인코포레이티드 내생적 면역글로불린 유전자를 억제하고 트랜스제닉 인간 이디오타입 항체를 생산하기 위한 방법 및 조성물
KR20100103810A (ko) 2007-12-10 2010-09-28 알리바 바이오파마수티컬스, 아이엔씨. 동종 재조합에 의한 표적화된 영역의 순차적인 치환 방법
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
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
DK3241435T3 (da) * 2009-07-08 2021-08-23 Kymab Ltd Dyremodeller og terapeutiske molekyler
US20120178647A1 (en) 2009-08-03 2012-07-12 The General Hospital Corporation Engineering of zinc finger arrays by context-dependent assembly
CA2779337A1 (en) 2009-10-28 2011-05-05 Helmholtz Zentrum Muenchen Deutsches Forschungszentrum Fuer Gesundheit U Nd Umwelt (Gmbh) Homologous recombination in the oocyte
CN102666854B (zh) 2009-10-29 2016-08-24 瑞泽恩制药公司 多功能等位基因
WO2011053957A2 (en) 2009-11-02 2011-05-05 Gen9, Inc. Compositions and methods for the regulation of multiple genes of interest in a cell
WO2011068103A1 (ja) 2009-12-01 2011-06-09 独立行政法人国立がん研究センター ラット胚性幹細胞を用いたキメララットの作製法
PT2816112T (pt) 2009-12-10 2018-11-20 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector tal
US20130023051A1 (en) 2009-12-21 2013-01-24 Keygene N.V. Techniques for transfecting protoplasts
CA2787674C (en) 2010-01-22 2022-09-06 Dow Agrosciences Llc Excision of transgenes in genetically modified organisms
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
JP2013528062A (ja) 2010-06-11 2013-07-08 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Xyes細胞からの繁殖可能なxy雌動物の生成
CN103228130B (zh) * 2010-06-17 2016-03-16 科马布有限公司 动物模型及治疗分子
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
RS61455B1 (sr) * 2011-10-28 2021-03-31 Regeneron Pharma Genetski modifikovani miševi t ćelijskog receptora
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
ES2683071T3 (es) 2012-04-25 2018-09-24 Regeneron Pharmaceuticals, Inc. Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
EP4397759A3 (en) 2012-10-23 2024-10-09 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
DK3363902T3 (da) 2012-12-06 2020-01-02 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
KR102955556B1 (ko) * 2012-12-12 2026-04-22 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
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
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
IL308158A (en) 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
EP3491915B1 (en) 2012-12-27 2023-05-31 Keygene N.V. Method for inducing a targeted translocation in a plant.
MX2015010841A (es) 2013-02-20 2016-05-09 Regeneron Pharma Modificacion genetica de ratas.
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
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
EP4286517A3 (en) 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
EP2997146A4 (en) 2013-05-15 2017-04-26 Sangamo BioSciences, 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
BR112015031639A2 (pt) 2013-06-19 2019-09-03 Sigma Aldrich Co Llc integração alvo
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
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
CA2932948A1 (en) 2013-12-13 2015-06-18 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
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
DK3152312T3 (da) 2014-06-06 2020-04-06 Regeneron Pharma Fremgangsmåder og sammensætninger til modifikation af et mållocus
DK3161128T3 (en) 2014-06-26 2018-11-05 Regeneron Pharma METHODS AND COMPOSITIONS FOR TARGETED GENTICAL MODIFICATIONS AND PROCEDURES FOR USE THEREOF
BR112017007770A2 (pt) 2014-10-15 2018-01-16 Regeneron Pharma cultura in vitro, população de hipscs, método para modificar um locus-alvo genômico, e, hipsc.
EP3221457B1 (en) 2014-11-21 2019-03-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
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.
GB201504223D0 (en) 2015-03-12 2015-04-29 Genome Res Ltd Biallelic genetic modification
US20200392538A1 (en) 2017-08-30 2020-12-17 President And Fellows Of Harvard College Iterative genome assembly

Also Published As

Publication number Publication date
RS60359B1 (sr) 2020-07-31
HUE049776T2 (hu) 2020-10-28
EP3152312B1 (en) 2020-02-12
RU2704283C9 (ru) 2020-02-07
US10294494B2 (en) 2019-05-21
SI3152312T1 (sl) 2020-07-31
US10106820B2 (en) 2018-10-23
US12060571B2 (en) 2024-08-13
EP3708671B1 (en) 2025-04-02
MX385689B (es) 2025-03-18
IL249042B (en) 2021-09-30
MX2016016133A (es) 2017-08-04
KR20170027743A (ko) 2017-03-10
JP7154248B2 (ja) 2022-10-17
CN106795521A (zh) 2017-05-31
US20240352489A1 (en) 2024-10-24
RU2016150168A (ru) 2018-07-18
CN113215196A (zh) 2021-08-06
US20170211099A1 (en) 2017-07-27
EP3708671C0 (en) 2025-04-02
SG11201609634YA (en) 2016-12-29
DK3152312T3 (da) 2020-04-06
AU2015269187A2 (en) 2017-01-19
SMT202000264T1 (it) 2020-07-08
LT3152312T (lt) 2020-04-27
AU2015269187B2 (en) 2021-06-17
US20150376650A1 (en) 2015-12-31
JP2017520243A (ja) 2017-07-27
AU2015269187A1 (en) 2017-01-12
EP3708671A1 (en) 2020-09-16
JP2020137521A (ja) 2020-09-03
RU2704283C2 (ru) 2019-10-25
HRP20200529T1 (hr) 2020-09-04
US20190225992A1 (en) 2019-07-25
CN113215196B (zh) 2024-09-03
NZ727481A (en) 2023-06-30
CA2950173C (en) 2023-10-10
ES3019688T3 (en) 2025-05-21
CY1122897T1 (el) 2021-05-05
BR112016028564A2 (pt) 2018-01-30
RU2016150168A3 (enExample) 2018-12-05
EP3152312A1 (en) 2017-04-12
KR102374379B1 (ko) 2022-03-17
CA2950173A1 (en) 2015-12-10
WO2015188109A1 (en) 2015-12-10
ES2784754T3 (es) 2020-09-30
JP6688231B2 (ja) 2020-04-28
CN106795521B (zh) 2021-06-04
SG10201913804WA (en) 2020-03-30
PL3152312T3 (pl) 2020-08-10
IL249042A0 (en) 2017-01-31

Similar Documents

Publication Publication Date Title
IL285565B (en) Methods and preparations for immune modulation
IL249042B (en) Methods and preparations for changing a targeted site
GB2535253B (en) Compositions and methods
GB201406151D0 (en) Method
GB201406155D0 (en) Method
IL249446A0 (en) Preparations and methods for production packaging
IL246879A0 (en) Preparations of Epilimod and methods of using them
GB201417828D0 (en) New methods and compositions
IL249868A0 (en) A method
IL249226A0 (en) Methods and preparations for immune modulation
GB201403470D0 (en) Method
GB201400756D0 (en) Blisk
GB201420832D0 (en) Location method
IL248513A0 (en) Preparations and methods for immunomodulation against lyst
GB201402915D0 (en) Compositions and methods
GB201403212D0 (en) Compositions and methods
GB201419012D0 (en) A composition
IL231539A0 (en) A model for the structure of the atom
GB201414050D0 (en) A composition
GB201406509D0 (en) Method
GB201406398D0 (en) Method
GB201406399D0 (en) Method
GB201406147D0 (en) Method
GB201406178D0 (en) Method
GB201405774D0 (en) Method