[go: up one dir, main page]

MX2018014172A - Métodos para romper la tolerancia inmunológica usando múltiples arn guías. - Google Patents

Métodos para romper la tolerancia inmunológica usando múltiples arn guías.

Info

Publication number
MX2018014172A
MX2018014172A MX2018014172A MX2018014172A MX2018014172A MX 2018014172 A MX2018014172 A MX 2018014172A MX 2018014172 A MX2018014172 A MX 2018014172A MX 2018014172 A MX2018014172 A MX 2018014172A MX 2018014172 A MX2018014172 A MX 2018014172A
Authority
MX
Mexico
Prior art keywords
methods
interest
antigen
guide rnas
multiple guide
Prior art date
Application number
MX2018014172A
Other languages
English (en)
Inventor
J Murphy Andrew
Macdonald Lynn
Frendewey David
Zambrowicz Brian
Droguett Gustavo
Voronina Vera
Prissette Marine
Badithe Ashok
Man Venus Lai Ka-
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 MX2018014172A publication Critical patent/MX2018014172A/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0271—Chimeric vertebrates, e.g. comprising exogenous 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
    • A01K67/0276—Knock-out vertebrates
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6489—Metalloendopeptidases (3.4.24)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/24—Metalloendopeptidases (3.4.24)
    • C12Y304/24046—Adamalysin (3.4.24.46)
    • 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
    • A01K2207/05—Animals modified by non-integrating nucleic acids, e.g. antisense, RNAi, morpholino, episomal vector, for non-therapeutic purpose
    • 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
    • A01K2207/12—Animals modified by administration of exogenous 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
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • 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
    • A01K2267/01—Animal expressing industrially exogenous proteins
    • 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
    • A01K2267/02—Animal zootechnically ameliorated
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/21—Immunoglobulins specific features characterized by taxonomic origin from primates, e.g. man
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/33—Crossreactivity, e.g. for species or epitope, or lack of said crossreactivity
    • 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
    • C12N2015/8518—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic expressing industrially exogenous proteins, e.g. for pharmaceutical use, human insulin, blood factors, immunoglobulins, pseudoparticles
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Environmental Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Information Retrieval, Db Structures And Fs Structures Therefor (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

Se proporcionan métodos y composiciones para generar animales no humanos con menor tolerancia de un antígeno foráneo de interés y generar proteínas de unión al antígeno contra dicho antígeno foráneo de interés usando dichos animales. Los métodos y las composiciones emplean sistemas CRISPR/Cas9 usando múltiples ARN guía para reducir o eliminar la expresión de un auto antígeno homologo a o que comparte un epitopo de interés con el antígeno foráneo de interés o para reducir o eliminar la expresión de un epitopo en el auto antígeno que es compartido con el antígeno foráneo de interés.
MX2018014172A 2016-05-20 2017-05-19 Métodos para romper la tolerancia inmunológica usando múltiples arn guías. MX2018014172A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662339472P 2016-05-20 2016-05-20
US201662368604P 2016-07-29 2016-07-29
PCT/US2017/033648 WO2017201476A1 (en) 2016-05-20 2017-05-19 Methods for breaking immunological tolerance using multiple guide rnas

Publications (1)

Publication Number Publication Date
MX2018014172A true MX2018014172A (es) 2019-08-22

Family

ID=59055266

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018014172A MX2018014172A (es) 2016-05-20 2017-05-19 Métodos para romper la tolerancia inmunológica usando múltiples arn guías.

Country Status (15)

Country Link
US (3) US20170332610A1 (es)
EP (2) EP3457840B1 (es)
JP (3) JP7324583B2 (es)
KR (3) KR102661748B1 (es)
CN (2) CN113831407B (es)
AU (2) AU2017268458B2 (es)
BR (1) BR112018073750A2 (es)
CA (1) CA3022997C (es)
ES (1) ES2977547T3 (es)
IL (2) IL323024A (es)
MX (1) MX2018014172A (es)
NZ (1) NZ747857A (es)
RU (1) RU2745563C2 (es)
SG (2) SG11201809552SA (es)
WO (1) WO2017201476A1 (es)

Families Citing this family (51)

* 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
US20150044192A1 (en) 2013-08-09 2015-02-12 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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
EP4079847A1 (en) 2014-07-30 2022-10-26 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
RU2734770C2 (ru) 2014-11-21 2020-10-23 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации с использованием парных гидовых рнк
JP7067793B2 (ja) 2015-10-23 2022-05-16 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 核酸塩基編集因子およびその使用
RU2745563C2 (ru) 2016-05-20 2021-03-29 Регенерон Фармасьютикалс, Инк. Способы преодоления иммунологической толерантности с использованием множества направляющих рнк
IL264565B2 (en) 2016-08-03 2024-07-01 Harvard College Adenosine nucleobase editors and uses thereof
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
JP6958917B2 (ja) * 2016-08-10 2021-11-02 国立大学法人 東京医科歯科大学 遺伝子ノックイン細胞の作製方法
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
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
EP3592777A1 (en) 2017-03-10 2020-01-15 President and Fellows of Harvard College Cytosine to guanine base editor
SG11201908658TA (en) 2017-03-23 2019-10-30 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
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
WO2019173615A1 (en) * 2018-03-07 2019-09-12 Icahn School Of Medicine At Mount Sinai Sphingolipid-metabolizing proteins enhance the efficiency of gene editing in cells
TW202506730A (zh) 2018-03-24 2025-02-16 美商再生元醫藥公司 用於產生抗肽-mhc複合物之治療性抗體的經基因修飾的非人類動物、其製法與用途
CN116420679B (zh) 2018-03-26 2025-10-03 瑞泽恩制药公司 用于测试治疗剂的人源化啮齿动物
US20200208177A1 (en) * 2018-04-18 2020-07-02 Ligandal, Inc. Methods and compositions for genome editing
JP2021523745A (ja) * 2018-05-16 2021-09-09 シンテゴ コーポレイション ガイドｒｎａ設計および使用のための方法およびシステム
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3814520A1 (en) * 2018-06-26 2021-05-05 The Broad Institute, Inc. Crispr double nickase based amplification compositions, systems, and methods
WO2020005341A1 (en) 2018-06-29 2020-01-02 Icahn School Of Medicine At Mount Sinai Anc80 encoding sphingolipid-metabolizing proteins
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
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020169022A1 (en) 2019-02-18 2020-08-27 Beijing Biocytogen Co., Ltd Genetically modified non-human animals with humanized immunoglobulin locus
WO2020191246A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12467043B2 (en) 2019-07-12 2025-11-11 Duke University 3′ UTR CRISPR-DCAS 13 engineering system and methods of using same
EP3785536B2 (en) * 2019-08-28 2025-09-17 Trianni, Inc. Adam6 knockin mice
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
EP4069722A1 (en) 2019-12-02 2022-10-12 Regeneron Pharmaceuticals, Inc. Peptide-mhc ii protein constructs and uses thereof
KR20210095781A (ko) 2020-01-24 2021-08-03 주식회사 에이프릴바이오 항원결합 단편 및 생리활성 이펙터 모이어티로 구성된 융합 컨스트럭트를 포함하는 다중결합항체 및 이를 포함하는 약학조성물
GB2614813B (en) 2020-05-08 2025-05-07 Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
KR20230018439A (ko) 2020-06-02 2023-02-07 바이오사이토젠 파마슈티컬스 (베이징) 컴퍼니 리미티드 공동 경쇄 면역글로불린 좌위를 갖는 유전자 변형 비인간 동물
JP7419168B2 (ja) * 2020-06-10 2024-01-22 株式会社東芝 改変型ｐｉｇｇｙＢａｃトランスポゼースのポリペプチド、それをコードするポリヌクレオチド、導入キャリア、キット、細胞のゲノムに目的配列を組込む方法及び細胞製造方法
EP4130263A1 (en) * 2021-08-04 2023-02-08 Eberhard Karls Universität Tübingen Medizinische Fakultät Compound and method for a specific targeting of the hax1 gene
JP2024540086A (ja) * 2021-10-28 2024-10-31 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 Ｃ５をノックアウトするためのｃｒｉｓｐｒ／ｃａｓ関連方法及び組成物
CN118475693A (zh) * 2021-12-24 2024-08-09 国立大学法人大阪大学 利用了同源重组的基因组编辑细胞的制造方法

Family Cites Families (159)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5523226A (en) * 1993-05-14 1996-06-04 Biotechnology Research And Development Corp. Transgenic swine compositions and methods
EP0721346B1 (en) 1993-09-02 1998-06-10 Trustees of Dartmouth College Methods for inducing antigen-specific t cell tolerance
US7119248B1 (en) * 1994-04-12 2006-10-10 Miltenyi Biotec Gmbh Antibodies against epitopes with homology to self antigens, methods of preparation and applications thereof
WO1999005266A2 (en) 1997-07-26 1999-02-04 Wisconsin Alumni Research Foundation Trans-species nuclear transfer
DE60133104T2 (de) 2000-05-31 2009-03-12 Novartis Vaccines and Diagnostics, Inc., Emeryville Verfahren und zusammensetzungen zur behandlung von neoplastischen erkrankungen mittels chemotherapie und strahlungssensibilisatoren
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
AUPR451401A0 (en) 2001-04-20 2001-05-24 Monash University A method of nuclear transfer
US7612250B2 (en) 2002-07-29 2009-11-03 Trustees Of Tufts College Nuclear transfer embryo formation method
BRPI0417056A (pt) 2003-12-01 2007-02-06 Kudos Pharm Ltd inibidores de reparo de dano no dna para tratamento de cáncer
PL2767161T3 (pl) 2004-10-19 2018-09-28 Regeneron Pharmaceuticals, Inc. Sposób wytwarzania zwierzęcia homozygotycznego pod względem modyfikacji genetycznej
JP2006187215A (ja) * 2004-12-28 2006-07-20 Univ Of Tsukuba ベクターによる遺伝子改変型樹状細胞を用いた抗体作製方法
HRP20120175T1 (hr) 2006-06-02 2012-03-31 Regeneron Pharmaceuticals Antitijela s visokim afinitetom za humani il-6 receptor
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
US7732195B2 (en) * 2006-11-01 2010-06-08 Facet Biotech Corporation Tethered vectors for cell surface immunoglobulin display
CN102037594A (zh) 2008-04-11 2011-04-27 Utc电力公司 燃料电池和具有歧管贮槽的双极板
MY164121A (en) 2009-06-26 2017-11-30 Regeneron Pharma Readily isolated bispecific antibodies with native immunoglobulin format
AU2010269978B2 (en) 2009-07-08 2016-11-03 Kymab Limited Animal models and therapeutic molecules
RU2425880C2 (ru) * 2009-07-30 2011-08-10 Учреждение Российской академии наук Институт общей генетики им. Н.И. Вавилова РАН Способ получения трансгенных мышей
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
US8518392B2 (en) 2009-08-14 2013-08-27 Regeneron Pharmaceuticals, Inc. Promoter-regulated differentiation-dependent self-deleting cassette
AU2010319894B2 (en) 2009-10-29 2015-03-05 Regeneron Pharmaceuticals, Inc. Multifunctional alleles
ES2595376T3 (es) 2009-12-10 2016-12-29 Regeneron Pharmaceuticals, Inc. Ratones que producen anticuerpos de cadena pesada
TR201808011T4 (tr) 2009-12-21 2018-06-21 Regeneron Pharma Hümanize fc gama r fareleri.
EP2525649B1 (en) 2010-01-22 2020-01-08 Dow AgroSciences, LLC Excision of transgenes in genetically modified organisms
US20130045492A1 (en) 2010-02-08 2013-02-21 Regeneron Pharmaceuticals, Inc. Methods For Making Fully Human Bispecific Antibodies Using A Common Light Chain
US20120021409A1 (en) 2010-02-08 2012-01-26 Regeneron Pharmaceuticals, Inc. Common Light Chain Mouse
US9796788B2 (en) 2010-02-08 2017-10-24 Regeneron Pharmaceuticals, Inc. Mice expressing a limited immunoglobulin light chain repertoire
PL2501817T5 (pl) 2010-02-08 2021-08-16 Regeneron Pharmaceuticals, Inc. Mysz o wspólnym łańcuchu lekkim
US20130185821A1 (en) 2010-02-08 2013-07-18 Regeneron Pharmaceuticals, Inc. Common Light Chain Mouse
KR101945352B1 (ko) 2010-06-22 2019-02-07 리제너론 파아마슈티컬스, 인크. 사람 람다 가변 영역 및 마우스 불변 영역을 갖는 경쇄를 발현하는 마우스
CA2805442C (en) 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus
CN105753979B (zh) 2010-08-02 2021-05-07 瑞泽恩制药公司 制造包含vl结构域的结合蛋白的小鼠
CN103492575A (zh) 2011-01-18 2014-01-01 安姆根有限公司 NaV1.7敲除小鼠及其用途
SG10201913155QA (en) * 2011-02-25 2020-02-27 Regeneron Pharma Adam6 mice
CA2837169C (en) 2011-05-24 2021-11-09 Zyngenia, Inc. Multispecific complexes comprising angiopoietin-2-binding peptide and their uses
SMT202100357T1 (it) 2011-08-05 2021-07-12 Regeneron Pharma Topi di catena leggera universale umanizzata
DK3311661T3 (da) 2011-09-19 2022-07-11 Kymab Ltd Manipulation af immunoglobulin-gendiversitet og terapeutiske lægemidler med flere antistoffer
KR20140082824A (ko) 2011-10-17 2014-07-02 리제너론 파아마슈티컬스, 인크. 제한된 면역글로불린 중쇄 마우스
HUE044266T2 (hu) 2011-12-20 2019-10-28 Regeneron Pharma Humanizált könnyûláncú egerek
SI2809150T1 (sl) 2012-02-01 2019-11-29 Regeneron Pharma Humanizirane miši, ki izražajo težke verige z domenami VL
US20140013456A1 (en) 2012-03-16 2014-01-09 Regeneron Pharmaceuticals, Inc. Histidine Engineered Light Chain Antibodies and Genetically Modified Non-Human Animals for Generating the Same
SG10201607727PA (en) 2012-03-16 2016-11-29 Regeneron Pharma Mice that produce antigen-binding proteins with ph-dependent binding characteristics
EP2825036B1 (en) 2012-03-16 2018-05-02 Regeneron Pharmaceuticals, Inc. Histidine engineered light chain antibodies and genetically modified rodents for generating the same
HUE045537T2 (hu) 2012-03-16 2019-12-30 Regeneron Pharma PH-érzékeny immunglobulin-szekvenciákat expresszáló rágcsálók
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
US10251377B2 (en) * 2012-03-28 2019-04-09 Kymab Limited Transgenic non-human vertebrate for the expression of class-switched, fully human, antibodies
KR102116153B1 (ko) 2012-05-07 2020-05-27 상가모 테라퓨틱스, 인코포레이티드 도입유전자의 뉴클레아제-매개 표적화된 통합을 위한 방법 및 조성물
US20160046961A1 (en) 2012-05-25 2016-02-18 Emmanuelle Charpentier Methods and Compositions for RNA-Directed Target DNA Modification and For RNA-Directed Modulation of Transcription
RU2656155C2 (ru) * 2012-06-12 2018-05-31 Регенерон Фармасьютикалз, Инк. Гуманизированные не относящиеся к человеку животные с ограниченными локусами тяжелой цепи иммуноглобулина
DK3494997T3 (da) 2012-07-25 2019-12-02 Broad Inst Inc Inducerbare dna-bindende proteiner og værktøjer til genomperturbation samt anvendelser deraf
WO2014033644A2 (en) 2012-08-28 2014-03-06 Novartis Ag Methods of nuclease-based genetic engineering
EP2912175B1 (en) 2012-10-23 2018-08-22 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
CN108913676B (zh) 2012-12-06 2023-11-03 西格马-奥尔德里奇有限责任公司 基于crispr的基因组修饰和调控
ES2576128T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones para la manipulación de secuencias con dominios funcionales
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
KR20150105956A (ko) * 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
SG10201912991WA (en) 2012-12-17 2020-03-30 Harvard College Rna-guided human genome engineering
EP3561050B1 (en) * 2013-02-20 2021-12-08 Regeneron Pharmaceuticals, Inc. Genetic modification of rats
KR101764800B1 (ko) 2013-02-20 2017-08-04 리제너론 파마슈티칼스 인코포레이티드 변형된 면역글로불린 중쇄 서열을 갖는 비인간 동물
WO2014131833A1 (en) 2013-02-27 2014-09-04 Helmholtz Zentrum München Deutsches Forschungszentrum Für Gesundheit Und Umwelt (Gmbh) Gene editing in the oocyte by cas9 nucleases
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
GB2527450A (en) 2013-03-14 2015-12-23 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
CN113563476A (zh) 2013-03-15 2021-10-29 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US9788534B2 (en) 2013-03-18 2017-10-17 Kymab Limited Animal models and therapeutic molecules
JP2016522679A (ja) * 2013-04-04 2016-08-04 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
HRP20181648T1 (hr) * 2013-04-16 2019-01-25 Regeneron Pharmaceuticals, Inc. Ciljana modifikacija genoma štakora
WO2014172470A2 (en) 2013-04-16 2014-10-23 Whitehead Institute For Biomedical Research Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal
JP2016518142A (ja) 2013-05-10 2016-06-23 サンガモ バイオサイエンシーズ， インコーポレイテッド ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
EP2997133B1 (en) * 2013-05-13 2023-08-23 Cellectis Methods for engineering highly active t cell for immunotherapy
ES2670531T3 (es) 2013-05-29 2018-05-30 Cellectis S.A. Un método para producir una escisión de ADN precisa utilizando la actividad nickasa de Cas9
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CN113425857B (zh) 2013-06-17 2025-05-16 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
KR20160019553A (ko) 2013-06-17 2016-02-19 더 브로드 인스티튜트, 인코퍼레이티드 유사분열 후 세포의 질병 및 장애를 표적화하고 모델링하기 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
CN106062197A (zh) 2013-06-17 2016-10-26 布罗德研究所有限公司 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化
EP3011035B1 (en) 2013-06-17 2020-05-13 The Broad Institute, Inc. Assay for quantitative evaluation of target site cleavage by one or more crispr-cas guide sequences
EP3011034B1 (en) 2013-06-17 2019-08-07 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
JP2016528890A (ja) 2013-07-09 2016-09-23 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
US11060083B2 (en) 2013-07-19 2021-07-13 Larix Bioscience Llc Methods and compositions for producing double allele knock outs
EP3022304B1 (en) 2013-07-19 2018-12-26 Larix Biosciences LLC Methods and compositions for producing double allele knock outs
US9925248B2 (en) 2013-08-29 2018-03-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
ES3013744T3 (en) 2013-09-18 2025-04-15 Kymab Ltd Methods, cells and organisms
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
CN110713995B (zh) 2013-10-17 2023-08-01 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
WO2015068785A1 (ja) 2013-11-06 2015-05-14 国立大学法人広島大学 核酸挿入用ベクター
DK3066201T3 (en) 2013-11-07 2018-06-06 Editas Medicine Inc CRISPR-RELATED PROCEDURES AND COMPOSITIONS WITH LEADING GRADES
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US10450586B2 (en) 2013-11-28 2019-10-22 Horizon Discovery Limited Somatic haploid human cell line
MX388127B (es) * 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
MX2016007326A (es) 2013-12-12 2017-02-13 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
CN111206032B (zh) 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
EP4219699A1 (en) 2013-12-12 2023-08-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
EP3080261B1 (en) 2013-12-12 2019-05-22 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
US20150291969A1 (en) 2014-01-30 2015-10-15 Chromatin, Inc. Compositions for reduced lignin content in sorghum and improving cell wall digestibility, and methods of making the same
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
EP3110454B1 (en) 2014-02-24 2020-11-18 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
WO2015138739A2 (en) 2014-03-12 2015-09-17 Precision Biosciences, Inc. Dystrophin gene oxon deletion using engineered nucleases
US20170081674A1 (en) 2014-03-14 2017-03-23 Cibus Us Llc Methods and compositions for increasing efficiency of targeted gene modification using oligonucleotide-mediated gene repair
CA3124228C (en) * 2014-03-21 2024-05-14 Regeneron Pharmaceuticals, Inc. Non-human animals that make single domain binding proteins
US10349639B2 (en) 2014-03-26 2019-07-16 University Of Maryland, College Park Targeted genome editing in zygotes of domestic large animals
GB201406968D0 (en) 2014-04-17 2014-06-04 Green Biologics Ltd Deletion mutants
EP3142706A1 (en) 2014-05-16 2017-03-22 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
WO2015184259A1 (en) 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods to treat latent viral infections
MX385689B (es) * 2014-06-06 2025-03-18 Regeneron Pharma Métodos y composiciones para modificar un locus dirigido.
EP3155101B1 (en) 2014-06-16 2020-01-29 The Johns Hopkins University Compositions and methods for the expression of crispr guide rnas using the h1 promoter
US20150376586A1 (en) 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
ES2694629T3 (es) 2014-06-26 2018-12-26 Regeneron Pharmaceuticals, Inc. Métodos y composiciones para modificaciones genéticas objetivo y métodos de uso
ES2959683T3 (es) 2014-07-15 2024-02-27 Juno Therapeutics Inc Células manipuladas para terapia celular adoptiva
WO2016011428A1 (en) 2014-07-17 2016-01-21 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of treating cells containing fusion genes
WO2016033246A1 (en) 2014-08-27 2016-03-03 Caribou Biosciences, Inc. Methods for increasing cas9-mediated engineering efficiency
EP3188763B1 (en) 2014-09-02 2020-05-13 The Regents of The University of California Methods and compositions for rna-directed target dna modification
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
WO2016061073A1 (en) 2014-10-14 2016-04-21 Memorial Sloan-Kettering Cancer Center Composition and method for in vivo engineering of chromosomal rearrangements
CA2964796C (en) 2014-10-17 2022-01-11 The Penn State Research Foundation Methods and compositions for multiplex rna guided genome editing and other rna technologies
US12180263B2 (en) 2014-11-06 2024-12-31 President And Fellows Of Harvard College Cells lacking B2M surface expression and methods for allogeneic administration of such cells
US11680268B2 (en) 2014-11-07 2023-06-20 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
RU2734770C2 (ru) 2014-11-21 2020-10-23 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации с использованием парных гидовых рнк
EP3224363B1 (en) 2014-11-27 2021-11-03 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Nucleic acid constructs for genome editing
WO2016089866A1 (en) 2014-12-01 2016-06-09 President And Fellows Of Harvard College Rna-guided systems for in vivo gene editing
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
US10863730B2 (en) 2014-12-26 2020-12-15 Riken Gene knockout method
US20180155708A1 (en) 2015-01-08 2018-06-07 President And Fellows Of Harvard College Split Cas9 Proteins
CN107406842B (zh) 2015-01-09 2021-05-11 生物辐射实验室股份有限公司 检测基因组编辑
WO2016130697A1 (en) 2015-02-11 2016-08-18 Memorial Sloan Kettering Cancer Center Methods and kits for generating vectors that co-express multiple target molecules
CA2977455A1 (en) 2015-02-23 2019-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
GB201504223D0 (en) 2015-03-12 2015-04-29 Genome Res Ltd Biallelic genetic modification
JP2018508224A (ja) 2015-03-19 2018-03-29 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． 抗原を結合する軽鎖可変領域を選択する非ヒト動物
WO2016154579A2 (en) 2015-03-26 2016-09-29 Editas Medicine, Inc. Crispr/cas-mediated gene conversion
WO2016160721A1 (en) 2015-03-27 2016-10-06 President And Fellows Of Harvard College Modified t cells and methods of making and using the same
CA2981508A1 (en) 2015-04-01 2016-10-06 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
US10155938B2 (en) 2015-04-14 2018-12-18 City Of Hope Coexpression of CAS9 and TREX2 for targeted mutagenesis
EP3286301B1 (en) 2015-04-22 2021-07-28 Sonic Master Limited Generation of muscle-lineage cells from stem cells
EP3289081B1 (en) 2015-04-27 2019-03-27 Genethon Compositions and methods for the treatment of nucleotide repeat expansion disorders
US20190275168A1 (en) 2015-04-30 2019-09-12 The Trustees Of Columbia University In The City Of New York Gene therapy for autosomal dominant diseases
RU2759335C2 (ru) 2015-05-16 2021-11-12 Джензим Корпорейшн Генное редактирование глубоких интронных мутаций
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016335698B2 (en) 2015-10-08 2022-12-01 President And Fellows Of Harvard College Multiplexed genome editing
KR20180069832A (ko) 2015-10-20 2018-06-25 파이어니어 하이 부렛드 인터내쇼날 인코포레이팃드 유도 cas 시스템을 통한 비기능성 유전자 산물에 대한 기능 회복 및 이용 방법
CA3004497A1 (en) 2015-11-06 2017-05-11 The Jackson Laboratory Large genomic dna knock-in and uses thereof
CA3014208A1 (en) 2016-02-16 2017-08-24 Regeneron Pharamaceuticals, Inc. Non-human animals having a mutant kynureninase gene
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017173004A1 (en) 2016-03-30 2017-10-05 Mikuni Takayasu A method for in vivo precise genome editing
EP3443080B1 (en) 2016-04-13 2021-08-11 University of Massachusetts Repairing compound heterozygous recessive mutations by allele exchange
RU2745563C2 (ru) 2016-05-20 2021-03-29 Регенерон Фармасьютикалс, Инк. Способы преодоления иммунологической толерантности с использованием множества направляющих рнк
EP3546575B1 (en) 2016-11-28 2024-07-17 Osaka University Genome editing method

Also Published As

Publication number Publication date
NZ747857A (en) 2023-01-27
JP7324583B2 (ja) 2023-08-10
KR20240016444A (ko) 2024-02-06
KR102661748B1 (ko) 2024-05-31
CN109475109A (zh) 2019-03-15
KR20220018613A (ko) 2022-02-15
AU2017268458A1 (en) 2018-11-22
RU2018143655A3 (es) 2020-10-07
EP3457840B1 (en) 2024-04-10
US20250280805A1 (en) 2025-09-11
ES2977547T3 (es) 2024-08-26
WO2017201476A1 (en) 2017-11-23
EP3457840C0 (en) 2024-04-10
EP4368637A2 (en) 2024-05-15
IL262888B1 (en) 2025-10-01
KR102356542B1 (ko) 2022-01-28
SG10202102885UA (en) 2021-05-28
CA3022997A1 (en) 2017-11-23
KR102628909B1 (ko) 2024-01-25
RU2018143655A (ru) 2020-06-22
EP3457840A1 (en) 2019-03-27
IL323024A (en) 2025-10-01
JP2025159002A (ja) 2025-10-17
IL262888A (en) 2018-12-31
JP2019518454A (ja) 2019-07-04
US20220167600A1 (en) 2022-06-02
AU2022246414A1 (en) 2022-11-03
CA3022997C (en) 2023-07-18
US20170332610A1 (en) 2017-11-23
RU2021106817A (ru) 2021-04-06
BR112018073750A2 (pt) 2019-02-26
CN113831407B (zh) 2024-06-11
EP4368637A3 (en) 2024-07-10
SG11201809552SA (en) 2018-11-29
AU2022246414B2 (en) 2024-11-21
AU2017268458B2 (en) 2022-07-21
JP2023078479A (ja) 2023-06-06
CN113831407A (zh) 2021-12-24
RU2745563C2 (ru) 2021-03-29
AU2022246414C1 (en) 2025-05-08
US12342801B2 (en) 2025-07-01
KR20190019931A (ko) 2019-02-27
CN109475109B (zh) 2021-10-29

Similar Documents

Publication Publication Date Title
MX2018014172A (es) Métodos para romper la tolerancia inmunológica usando múltiples arn guías.
UY40829A (es) Anticuerpos anti-phf-tau y sus usos
CO2018006301A2 (es) Mutantes de proteína f de rsv
CL2018000162A1 (es) Composiciones ingeribles por abejas, procedimientos para el uso de las mismas para producir miel y miel producida de esta manera
MX2023007726A (es) Antifolatos poliglutamados alfa y gamma-d y sus usos.
MX391171B (es) Anticuerpos anti-cd19 humano con alta afinidad
CR20160379A (es) Anticuerpos biespecificos anti-il 13/il-17 y sus usos
CR20180605A (es) Anticuerpos anti-cd40 y sus usos
DOP2018000219A (es) Anticuerpos anti-factor bb del complemento y usos de estos
MX390482B (es) Pirimidinas y variantes de estas, y usos de estas.
CR20170514A (es) Combinaciones herbicidas que comprenden l-glufosinato e indiziflam.
MX2017003121A (es) Formulaciones de anticuerpos.
MX378368B (es) Receptores de antigeno y usos de los mismos.
CO2020016628A2 (es) Medios y métodos para el clivaje de la zearalenona
PL3939195T3 (pl) Metody poprawy niezawodności aktualizacji wiązki wyzwalanej przez dci w 5g nr
AR100716A1 (es) Producción de fragmentos fc
CO2018013257A2 (es) Formulaciones de fulvestrant y métodos para su uso
CL2020003447A1 (es) Composiciones y métodos para el tratamiento de enfermedades o condiciones relacionadas con inflamasomas
CO2017007249A2 (es) Modificación cristalina de propanil
CR20160392A (es) Proteínas de fusión tatk-cdkl5, composiciones, formulaciones y uso de esta
ECSP19008417A (es) Anticuerpos con inmunogenicidad baja y usos de estos
CO2017001902A2 (es) Adyuvantes acondicionadores del agua que promueven una baja volatilidad
BR112018067851A2 (pt) composições e métodos para o tratamento de artrite reumatoide
BR112017020513A2 (pt) método de produção de peixe para transplante, peixe para transplante, método de produção de espécie híbrida de peixe e espécie híbrida de peixe
MX2019003212A (es) Composiciones que comprenden tensioactivos especificos y altos niveles de glicerina.