WO2002001950A3 - Transgenic mice containing targeted gene disruptions - Google Patents

Transgenic mice containing targeted gene disruptions Download PDF

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Publication number
WO2002001950A3
WO2002001950A3 PCT/US2001/020795 US0120795W WO0201950A3 WO 2002001950 A3 WO2002001950 A3 WO 2002001950A3 US 0120795 W US0120795 W US 0120795W WO 0201950 A3 WO0201950 A3 WO 0201950A3
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WO
WIPO (PCT)
Prior art keywords
transgenic mice
targeted gene
mice containing
gene disruptions
containing targeted
Prior art date
Application number
PCT/US2001/020795
Other languages
French (fr)
Other versions
WO2002001950A2 (en
Inventor
Michael W Leviten
Thomas J Brennan
Catherine Guenther
Robert Klein
William Matthews
Mark Moore
Original Assignee
Deltagen Inc
Michael W Leviten
Thomas J Brennan
Catherine Guenther
Robert Klein
William Matthews
Mark Moore
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 Deltagen Inc, Michael W Leviten, Thomas J Brennan, Catherine Guenther, Robert Klein, William Matthews, Mark Moore filed Critical Deltagen Inc
Priority to JP2002506586A priority Critical patent/JP2004524002A/en
Priority to CA002416292A priority patent/CA2416292A1/en
Priority to EP01950688A priority patent/EP1304921A2/en
Priority to AU2001271656A priority patent/AU2001271656A1/en
Publication of WO2002001950A2 publication Critical patent/WO2002001950A2/en
Publication of WO2002001950A3 publication Critical patent/WO2002001950A3/en

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027New breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276Knockout animals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • C07K14/51Bone morphogenetic factor; Osteogenins; Osteogenic factor; Bone-inducing factor
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70567Nuclear receptors, e.g. retinoic acid receptor [RAR], RXR, nuclear orphan receptors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • C12N9/6427Chymotrypsins (3.4.21.1; 3.4.21.2); Trypsin (3.4.21.4)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/564Immunoassay; Biospecific binding assay; Materials therefor for pre-existing immune complex or autoimmune disease, i.e. systemic lupus erythematosus, rheumatoid arthritis, multiple sclerosis, rheumatoid factors or complement components C1-C9
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/072Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/20Animal model comprising regulated expression system
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0393Animal model comprising a reporter system for screening tests
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/30Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/435Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
    • G01N2333/46Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
    • G01N2333/47Assays involving proteins of known structure or function as defined in the subgroups
    • G01N2333/4701Details
    • G01N2333/4716Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2500/00Screening for compounds of potential therapeutic value

Abstract

The present invention relates to transgenic animals, as well as compositions and methods relating to the characterization of gene function. Specifically, the present invention provides transgenic mice comprising mutations in a target gene. Such transgenic mice are useful as models for disease and for identifying agents that modulate gene expression and gene function, and as potential treatments for various disease states and disease conditions.
PCT/US2001/020795 2000-06-29 2001-06-29 Transgenic mice containing targeted gene disruptions WO2002001950A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
JP2002506586A JP2004524002A (en) 2000-06-29 2001-06-29 Transgenic mice containing targeted gene disruption
CA002416292A CA2416292A1 (en) 2000-06-29 2001-06-29 Transgenic mice containing targeted gene disruptions
EP01950688A EP1304921A2 (en) 2000-06-29 2001-06-29 Transgenic mice containing targeted gene disruptions
AU2001271656A AU2001271656A1 (en) 2000-06-29 2001-06-29 Transgenic mice containing targeted gene disruptions

Applications Claiming Priority (18)

Application Number Priority Date Filing Date Title
US21546600P 2000-06-29 2000-06-29
US21546700P 2000-06-29 2000-06-29
US21540200P 2000-06-29 2000-06-29
US21536600P 2000-06-29 2000-06-29
US21517900P 2000-06-29 2000-06-29
US21540400P 2000-06-29 2000-06-29
US21517800P 2000-06-29 2000-06-29
US60/215,466 2000-06-29
US60/215,179 2000-06-29
US60/215,366 2000-06-29
US60/215,467 2000-06-29
US60/215,402 2000-06-29
US60/215,178 2000-06-29
US60/215,404 2000-06-29
US22166700P 2000-07-27 2000-07-27
US60/221,667 2000-07-27
US24408300P 2000-10-26 2000-10-26
US60/244,083 2000-10-26

Publications (2)

Publication Number Publication Date
WO2002001950A2 WO2002001950A2 (en) 2002-01-10
WO2002001950A3 true WO2002001950A3 (en) 2003-02-27

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PCT/US2001/020795 WO2002001950A2 (en) 2000-06-29 2001-06-29 Transgenic mice containing targeted gene disruptions

Country Status (5)

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EP (1) EP1304921A2 (en)
JP (1) JP2004524002A (en)
AU (1) AU2001271656A1 (en)
CA (1) CA2416292A1 (en)
WO (1) WO2002001950A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1215284A4 (en) * 1999-09-06 2004-05-19 Chugai Pharmaceutical Co Ltd Tsg-like gene
US7700274B2 (en) 2000-12-22 2010-04-20 Sagres Discovery, Inc. Compositions and methods in cancer associated with altered expression of KCNJ9
US20030216558A1 (en) * 2000-12-22 2003-11-20 Morris David W. Novel compositions and methods for cancer
US9986722B2 (en) * 2012-08-07 2018-06-05 Chiesi Farmaceutici S.P.A. Animal model of Krabbe's disease

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994026100A1 (en) * 1993-05-18 1994-11-24 Institut National De La Sante Et De La Recherche Medicale Genetically engineered mice containing alterations in the genes encoding retinoic acid receptor proteins
WO1996039511A2 (en) * 1995-06-05 1996-12-12 Incyte Pharmaceuticals, Inc. A C5a-LIKE SEVEN TRANSMEMBRANE RECEPTOR
WO1997028188A1 (en) * 1996-01-30 1997-08-07 The Scripps Research Institute A g protein-coupled receptor with an enlarged extracellular domain
EP0814158A2 (en) * 1996-06-17 1997-12-29 Smithkline Beecham Corporation Human C3a receptor variant; uses in diagnostics and therapeutics
WO1998039659A1 (en) * 1997-03-06 1998-09-11 Bion Diagnostic Sciences, Inc. Screening and treatment using complement regulator or receptor proteins
WO1998045465A1 (en) * 1997-04-09 1998-10-15 A+ Science Invest Ab Transgenic, non-human animal with protein deficiency, cells derived therefrom, use of said animal and cells, and method of producing said transgenic animal
WO2000009129A1 (en) * 1998-08-11 2000-02-24 Smithkline Beecham Corporation C3a receptor ligands

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994026100A1 (en) * 1993-05-18 1994-11-24 Institut National De La Sante Et De La Recherche Medicale Genetically engineered mice containing alterations in the genes encoding retinoic acid receptor proteins
WO1996039511A2 (en) * 1995-06-05 1996-12-12 Incyte Pharmaceuticals, Inc. A C5a-LIKE SEVEN TRANSMEMBRANE RECEPTOR
WO1997028188A1 (en) * 1996-01-30 1997-08-07 The Scripps Research Institute A g protein-coupled receptor with an enlarged extracellular domain
EP0814158A2 (en) * 1996-06-17 1997-12-29 Smithkline Beecham Corporation Human C3a receptor variant; uses in diagnostics and therapeutics
WO1998039659A1 (en) * 1997-03-06 1998-09-11 Bion Diagnostic Sciences, Inc. Screening and treatment using complement regulator or receptor proteins
WO1998045465A1 (en) * 1997-04-09 1998-10-15 A+ Science Invest Ab Transgenic, non-human animal with protein deficiency, cells derived therefrom, use of said animal and cells, and method of producing said transgenic animal
WO2000009129A1 (en) * 1998-08-11 2000-02-24 Smithkline Beecham Corporation C3a receptor ligands

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
HOLERS V M: "Phenotypes of complement knockouts.", IMMUNOPHARMACOLOGY. NETHERLANDS AUG 2000, vol. 49, no. 1-2, August 2000 (2000-08-01), pages 125 - 131, XP002204355, ISSN: 0162-3109 *
HOLLMANN T J ET AL: "Cloning, expression, sequence determination, and chromosome localization of the mouse complement C3a anaphylatoxin receptor gene.", MOLECULAR IMMUNOLOGY. ENGLAND FEB 1998, vol. 35, no. 3, February 1998 (1998-02-01), pages 137 - 148, XP002204354, ISSN: 0161-5890 *
HUMBLES ALISON A ET AL: "A role for the C3a anaphylatoxin receptor in the effector phase of asthma.", NATURE (LONDON), vol. 406, no. 6799, 31 August 2000 (2000-08-31), pages 998 - 1001, XP002204352, ISSN: 0028-0836 *
KILDSGAARD J ET AL: "Cutting edge: targeted disruption of the C3a receptor gene demonstrates a novel protective anti-inflammatory role for C3a in endotoxin-shock.", JOURNAL OF IMMUNOLOGY (BALTIMORE, MD.: 1950) UNITED STATES 15 NOV 2000, vol. 165, no. 10, 15 November 2000 (2000-11-15), pages 5406 - 5409, XP002204353, ISSN: 0022-1767 *

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Publication number Publication date
AU2001271656A1 (en) 2002-01-14
CA2416292A1 (en) 2002-01-10
WO2002001950A2 (en) 2002-01-10
JP2004524002A (en) 2004-08-12
EP1304921A2 (en) 2003-05-02

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