AU2008206936B2 - In vivo screening models for treatment of Alzheimer's disease and other Qpct-related disorders - Google Patents

In vivo screening models for treatment of Alzheimer's disease and other Qpct-related disorders Download PDF

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Publication number
AU2008206936B2
AU2008206936B2 AU2008206936A AU2008206936A AU2008206936B2 AU 2008206936 B2 AU2008206936 B2 AU 2008206936B2 AU 2008206936 A AU2008206936 A AU 2008206936A AU 2008206936 A AU2008206936 A AU 2008206936A AU 2008206936 B2 AU2008206936 B2 AU 2008206936B2
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Prior art keywords
glutaminyl cyclase
transgenic
mouse
transgene
human animal
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AU2008206936A
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AU2008206936A1 (en
Inventor
Holger Cynis
Hans-Ulrich Demuth
Torsten Hoffmann
Stephan Schilling
Katrin Schulz
Michael Wermann
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Vivoryon Therapeutics AG
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Probiodrug AG
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    • 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
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/14—Prodigestives, e.g. acids, enzymes, appetite stimulants, antidyspeptics, tonics, antiflatulents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • A61P15/08—Drugs for genital or sexual disorders; Contraceptives for gonadal disorders or for enhancing fertility, e.g. inducers of ovulation or of spermatogenesis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/06—Antipsoriatics
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/20—Hypnotics; Sedatives
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
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    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1025—Acyltransferases (2.3)
    • C12N9/104—Aminoacyltransferases (2.3.2)
    • 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
    • A01K2217/05—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; 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
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0312—Animal model for Alzheimer's disease
    • 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
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's

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  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Environmental Sciences (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Reproductive Health (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Rheumatology (AREA)
  • Animal Husbandry (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Plant Pathology (AREA)
  • Endocrinology (AREA)
  • Biophysics (AREA)
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AU2008206936A 2007-01-19 2008-01-18 In vivo screening models for treatment of Alzheimer's disease and other Qpct-related disorders Ceased AU2008206936B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US88564907P 2007-01-19 2007-01-19
US60/885,649 2007-01-19
PCT/EP2008/050532 WO2008087197A1 (en) 2007-01-19 2008-01-18 In vivo screening models for treatment of alzheimer's disease and other qpct-related disorders

Publications (2)

Publication Number Publication Date
AU2008206936A1 AU2008206936A1 (en) 2008-07-24
AU2008206936B2 true AU2008206936B2 (en) 2013-03-14

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AU2008206936A Ceased AU2008206936B2 (en) 2007-01-19 2008-01-18 In vivo screening models for treatment of Alzheimer's disease and other Qpct-related disorders

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US (1) US20080200567A1 (enExample)
EP (2) EP2395095A1 (enExample)
JP (1) JP2010516235A (enExample)
AT (1) ATE521711T1 (enExample)
AU (1) AU2008206936B2 (enExample)
CA (1) CA2675192A1 (enExample)
DK (1) DK2121944T3 (enExample)
ES (1) ES2372229T3 (enExample)
HR (1) HRP20110651T1 (enExample)
NZ (1) NZ578513A (enExample)
SI (1) SI2121944T1 (enExample)
WO (1) WO2008087197A1 (enExample)
ZA (1) ZA200904353B (enExample)

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ES2468551T3 (es) * 2007-04-18 2014-06-16 Probiodrug Ag Derivados de nitrovinil-diamina como inhibidores de la glutaminil ciclasa
WO2008128985A1 (en) * 2007-04-18 2008-10-30 Probiodrug Ag Thiourea derivatives as glutaminyl cyclase inhibitors
JP5675343B2 (ja) * 2007-04-18 2015-02-25 プロビオドルグ エージー グルタミニルシクラーゼ阻害剤としての尿素誘導体
EP2142515B1 (en) * 2007-04-18 2014-03-26 Probiodrug AG Nitrovinyl-diamine derivatives as glutaminyl cyclase inhibitors
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WO2009090190A1 (en) * 2008-01-14 2009-07-23 Probiodrug Ag Mouse models carrying a knock-out mutation of the glutaminyl cyclase gene
EA025107B1 (ru) * 2008-07-31 2016-11-30 Пробиодруг Аг Способ диагностирования болезни альцгеймера, нейродегенерации при синдроме дауна или легкого когнитивного нарушения у субъекта (варианты) и набор для осуществления способа
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
ES2540467T3 (es) 2010-08-19 2015-07-09 Probiodrug Ag Estructura cristalina de glutaminil ciclasa
JP2013541938A (ja) 2010-09-02 2013-11-21 プロビオドルグ エージー アイソqc関連障害の治療のためのインビボスクリーニングモデル
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WO2013024043A2 (en) 2011-08-12 2013-02-21 Probiodrug Ag In vivo screening models for treatment of qc-related disorders
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WO2008087197A1 (en) 2008-07-24
NZ578513A (en) 2012-01-12
EP2121944B1 (en) 2011-08-24
SI2121944T1 (sl) 2011-12-30
JP2010516235A (ja) 2010-05-20
EP2395095A1 (en) 2011-12-14
ES2372229T3 (es) 2012-01-17
DK2121944T3 (da) 2011-10-31
US20080200567A1 (en) 2008-08-21
ATE521711T1 (de) 2011-09-15
HRP20110651T1 (hr) 2011-10-31
AU2008206936A1 (en) 2008-07-24
EP2121944A1 (en) 2009-11-25
CA2675192A1 (en) 2008-07-24
ZA200904353B (en) 2010-09-29

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