PL2747551T3 - Szczury z niedoborem hydrolazy fumaryloacetooctanowej (fah) i z niedoborem odporności oraz ich zastosowanie - Google Patents

Szczury z niedoborem hydrolazy fumaryloacetooctanowej (fah) i z niedoborem odporności oraz ich zastosowanie

Info

Publication number
PL2747551T3
PL2747551T3 PL12828793T PL12828793T PL2747551T3 PL 2747551 T3 PL2747551 T3 PL 2747551T3 PL 12828793 T PL12828793 T PL 12828793T PL 12828793 T PL12828793 T PL 12828793T PL 2747551 T3 PL2747551 T3 PL 2747551T3
Authority
PL
Poland
Prior art keywords
fah
deficient
fumarylacetoacetate hydrolase
immunodeficient rats
immunodeficient
Prior art date
Application number
PL12828793T
Other languages
English (en)
Inventor
John R. BIAL
Elizabeth M. Wilson
Aron M. Geurts
Original Assignee
Yecuris Corporation
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 Yecuris Corporation filed Critical Yecuris Corporation
Publication of PL2747551T3 publication Critical patent/PL2747551T3/pl

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Classifications

    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • C—CHEMISTRY; METALLURGY
    • 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
    • C07K14/715—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
    • C07K14/7155—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons for interleukins [IL]
    • 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)
    • 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
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/6456—Plasminogen activators
    • C12N9/6462—Plasminogen activators u-Plasminogen activator (3.4.21.73), i.e. urokinase
    • 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)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21073—Serine endopeptidases (3.4.21) u-Plasminogen activator (3.4.21.73), i.e. urokinase
    • 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
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y307/00—Hydrolases acting on carbon-carbon bonds (3.7)
    • C12Y307/01—Hydrolases acting on carbon-carbon bonds (3.7) in ketonic substances (3.7.1)
    • C12Y307/01002—Fumarylacetoacetase (3.7.1.2)

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Environmental Sciences (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Animal Husbandry (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PL12828793T 2011-08-26 2012-08-24 Szczury z niedoborem hydrolazy fumaryloacetooctanowej (fah) i z niedoborem odporności oraz ich zastosowanie PL2747551T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161527865P 2011-08-26 2011-08-26
PCT/US2012/052306 WO2013032918A1 (en) 2011-08-26 2012-08-24 Fumarylacetoacetate hydrolase (fah)-deficient and immunodeficient rats and uses thereof
EP12828793.5A EP2747551B1 (en) 2011-08-26 2012-08-24 Fumarylacetoacetate hydrolase (fah)-deficient and immunodeficient rats and uses thereof

Publications (1)

Publication Number Publication Date
PL2747551T3 true PL2747551T3 (pl) 2020-09-21

Family

ID=47756758

Family Applications (1)

Application Number Title Priority Date Filing Date
PL12828793T PL2747551T3 (pl) 2011-08-26 2012-08-24 Szczury z niedoborem hydrolazy fumaryloacetooctanowej (fah) i z niedoborem odporności oraz ich zastosowanie

Country Status (8)

Country Link
US (3) US20140373187A1 (pl)
EP (2) EP3578042A1 (pl)
JP (2) JP6484444B2 (pl)
CY (1) CY1122857T1 (pl)
DK (1) DK2747551T3 (pl)
ES (1) ES2791829T3 (pl)
PL (1) PL2747551T3 (pl)
WO (1) WO2013032918A1 (pl)

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WO2013096955A1 (en) * 2011-12-23 2013-06-27 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
EP3561050B1 (en) 2013-02-20 2021-12-08 Regeneron Pharmaceuticals, Inc. Genetic modification of rats
SI2986729T1 (sl) 2013-04-16 2019-02-28 Regeneron Pharmaceuticals, Inc. Ciljana sprememba genoma podgane
IL302370B2 (en) * 2015-04-13 2025-10-01 Regeneron Pharma Mice genetically engineered to express alpha signal modulator protein and interleukin 15 and methods for using them
AU2017289194B2 (en) * 2016-06-27 2019-12-05 Baylor College Of Medicine Human liver chimeric non-human animal with deficient P450 oxidoreductase and methods of using same
WO2018033542A1 (en) * 2016-08-15 2018-02-22 Danmarks Tekniske Universitet Method for reducing ammonium and lactate production in cho cells
EP3583201B1 (en) * 2017-02-14 2024-01-17 University of Pittsburgh - Of the Commonwealth System of Higher Education Methods of engineering human induced pluripotent stem cells to produce liver tissue
CN109136273B (zh) * 2017-06-16 2022-09-27 中国科学院分子细胞科学卓越创新中心 制备免疫缺陷的大鼠的方法及其应用
CN114206107A (zh) * 2019-07-26 2022-03-18 安必思医药公司 用于产生肝细胞的方法和组合物
CN113440502B (zh) * 2020-11-30 2022-10-18 先见明创医药科技(苏州)有限公司 Fah基因缺失动物的程序化慢性肝损伤维持及其在制备异源化肝脏模型中的应用
WO2023086958A1 (en) * 2021-11-12 2023-05-19 Ambys Medicines, Inc. Methods of producing and using human hepatocytes and related compositions
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WO2024073679A1 (en) 2022-09-29 2024-04-04 Regeneron Pharmaceuticals, Inc. Correction of hepatosteatosis in humanized liver animals through restoration of il6/il6r/gp130 signaling in human hepatocytes
WO2025250495A1 (en) 2024-05-28 2025-12-04 Regeneron Pharmaceuticals, Inc. Acceleration of human hepatocyte engraftment in humanized liver animals by supplementing paracrine ligands or agonists that activate human liver regeneration signals

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PL2747551T3 (pl) 2011-08-26 2020-09-21 Yecuris Corporation Szczury z niedoborem hydrolazy fumaryloacetooctanowej (fah) i z niedoborem odporności oraz ich zastosowanie

Also Published As

Publication number Publication date
EP3578042A1 (en) 2019-12-11
JP6484444B2 (ja) 2019-03-13
EP2747551A1 (en) 2014-07-02
JP2014525248A (ja) 2014-09-29
DK2747551T3 (da) 2020-05-18
US11793178B2 (en) 2023-10-24
JP6573924B2 (ja) 2019-09-11
EP2747551A4 (en) 2015-07-15
US20200008406A1 (en) 2020-01-09
EP2747551B1 (en) 2020-02-12
US10470445B2 (en) 2019-11-12
CY1122857T1 (el) 2021-05-05
US20140373187A1 (en) 2014-12-18
WO2013032918A1 (en) 2013-03-07
ES2791829T3 (es) 2020-11-06
JP2017131227A (ja) 2017-08-03
US20160249591A1 (en) 2016-09-01

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