WO2010062604A3 - Models of thrombotic thrombocytopenic purpura and methods of use thereof - Google Patents

Models of thrombotic thrombocytopenic purpura and methods of use thereof Download PDF

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Publication number
WO2010062604A3
WO2010062604A3 PCT/US2009/062228 US2009062228W WO2010062604A3 WO 2010062604 A3 WO2010062604 A3 WO 2010062604A3 US 2009062228 W US2009062228 W US 2009062228W WO 2010062604 A3 WO2010062604 A3 WO 2010062604A3
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WO
WIPO (PCT)
Prior art keywords
ttp
thrombocytopenic purpura
methods
thrombotic thrombocytopenic
models
Prior art date
Application number
PCT/US2009/062228
Other languages
French (fr)
Other versions
WO2010062604A2 (en
Inventor
Hans-Peter Schwarz
Eva-Maria Muchitsch
Peter Turecek
Original Assignee
Baxter International Inc.
Baxter Healthcare S.A.
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 Baxter International Inc., Baxter Healthcare S.A. filed Critical Baxter International Inc.
Priority to EP09752581.0A priority Critical patent/EP2350294B1/en
Priority to ES09752581T priority patent/ES2713270T3/en
Priority to NZ592905A priority patent/NZ592905A/en
Priority to JP2011533425A priority patent/JP5749649B2/en
Priority to KR1020117011998A priority patent/KR101785118B1/en
Priority to AU2009320088A priority patent/AU2009320088B2/en
Priority to EP18202001.6A priority patent/EP3495488A1/en
Priority to DK09752581.0T priority patent/DK2350294T3/en
Priority to CA2741634A priority patent/CA2741634A1/en
Publication of WO2010062604A2 publication Critical patent/WO2010062604A2/en
Publication of WO2010062604A3 publication Critical patent/WO2010062604A3/en

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027—New breeds of vertebrates
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027—New breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027—New breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knockout animals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • 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
    • 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; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/054—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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
    • A01K2267/0306—Animal model for genetic diseases
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0325—Animal model for autoimmune diseases
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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
    • A01K2267/0381—Animal model for diseases of the hematopoietic system

Abstract

The invention relates to the development of an animal model for testing various agents in the treatment of a clotting disorder. More specifically, the invention relates to the use of ultra-large molecular weight multimers of von Willebrand factor (VWF) in various mouse strains to induce thrombotic thrombocytopenic purpura (TTP)-like symptoms for the development of a mouse model of TTP. The invention also provides methods for generating such animal disease models and screening methods for identifying biologically active compounds which are effective in the treatment of TTP.
PCT/US2009/062228 2008-10-27 2009-10-27 Models of thrombotic thrombocytopenic purpura and methods of use thereof WO2010062604A2 (en)

Priority Applications (9)

Application Number Priority Date Filing Date Title
EP09752581.0A EP2350294B1 (en) 2008-10-27 2009-10-27 Mouse models of thrombotic thrombocytopenic purpura and methods of use thereof
ES09752581T ES2713270T3 (en) 2008-10-27 2009-10-27 Mouse models of thrombotic thrombocytopenic purpura and methods for its use
NZ592905A NZ592905A (en) 2008-10-27 2009-10-27 Models of thrombotic thrombocytopenic purpura and methods of use thereof
JP2011533425A JP5749649B2 (en) 2008-10-27 2009-10-27 Model of thrombotic thrombocytopenic purpura and method of use thereof
KR1020117011998A KR101785118B1 (en) 2008-10-27 2009-10-27 Models of thrombotic thrombocytopenic purpura and methods of use thereof
AU2009320088A AU2009320088B2 (en) 2008-10-27 2009-10-27 Models of thrombotic thrombocytopenic purpura and methods of use thereof
EP18202001.6A EP3495488A1 (en) 2008-10-27 2009-10-27 Models of thrombotic thrombocytopenic purpura and methods of use thereof
DK09752581.0T DK2350294T3 (en) 2008-10-27 2009-10-27 MOUSE MODELS OF TROMBOTIC TROMBOCYTPENIC PURPURA AND METHOD OF USING THEREOF
CA2741634A CA2741634A1 (en) 2008-10-27 2009-10-27 Models of thrombotic thrombocytopenic purpura and methods of use thereof

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US10878108P 2008-10-27 2008-10-27
US61/108,781 2008-10-27
US15676809P 2009-03-02 2009-03-02
US61/156,768 2009-03-02

Publications (2)

Publication Number Publication Date
WO2010062604A2 WO2010062604A2 (en) 2010-06-03
WO2010062604A3 true WO2010062604A3 (en) 2010-07-22

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2009/062228 WO2010062604A2 (en) 2008-10-27 2009-10-27 Models of thrombotic thrombocytopenic purpura and methods of use thereof

Country Status (10)

Country Link
US (2) US9587249B2 (en)
EP (2) EP2350294B1 (en)
JP (1) JP5749649B2 (en)
KR (1) KR101785118B1 (en)
AU (1) AU2009320088B2 (en)
CA (1) CA2741634A1 (en)
DK (1) DK2350294T3 (en)
ES (1) ES2713270T3 (en)
NZ (3) NZ592905A (en)
WO (1) WO2010062604A2 (en)

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ES2685503T3 (en) 2008-12-05 2018-10-09 Baxalta GmbH Methods of measuring in vivo division of Factor von Willebrand mediated by ADAMTS13 and their use
RU2570162C1 (en) * 2014-10-21 2015-12-10 Общество с ограниченной ответственностью "АптаНова" Method for simulating thrombus formation in mice for assessing effect of anticoagulant preparations
US11597778B2 (en) 2015-04-07 2023-03-07 The Trustees Of The University Of Pennsylvania Human monoclonal autoantibodies to ADAMTS13 and uses thereof
CN115413627B (en) * 2022-10-21 2023-03-24 首都医科大学附属北京儿童医院 Construction method and application of animal model of abdominal anaphylactoid purpura

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