WO2003095640A3 - Transgene ratte als tiermodell für die humane chorea huntington erkrankung - Google Patents

Transgene ratte als tiermodell für die humane chorea huntington erkrankung Download PDF

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Publication number
WO2003095640A3
WO2003095640A3 PCT/DE2003/001546 DE0301546W WO03095640A3 WO 2003095640 A3 WO2003095640 A3 WO 2003095640A3 DE 0301546 W DE0301546 W DE 0301546W WO 03095640 A3 WO03095640 A3 WO 03095640A3
Authority
WO
WIPO (PCT)
Prior art keywords
human
disease
huntingdon
cag
progredient
Prior art date
Application number
PCT/DE2003/001546
Other languages
English (en)
French (fr)
Other versions
WO2003095640A2 (de
Inventor
Olaf Riess
Hoersten Stephan Von
Original Assignee
Hannover Med Hochschule
Olaf Riess
Hoersten Stephan Von
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 Hannover Med Hochschule, Olaf Riess, Hoersten Stephan Von filed Critical Hannover Med Hochschule
Priority to AU2003243893A priority Critical patent/AU2003243893A1/en
Priority to DE10393086T priority patent/DE10393086D2/de
Priority to EP03749850A priority patent/EP1534826A2/de
Priority to CA002523888A priority patent/CA2523888A1/en
Priority to US10/514,512 priority patent/US20070044162A1/en
Publication of WO2003095640A2 publication Critical patent/WO2003095640A2/de
Publication of WO2003095640A3 publication Critical patent/WO2003095640A3/de

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Classifications

    • 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
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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)
    • 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
    • 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
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's

Abstract

Die Chorea Huntington (Huntington's Disease, HD) ist eine autosomal dominant vererbte progrediente neurodegenerative Erkrankung aus der Gruppe der 'CAG-repeat'/Polyglutamin-Erkrankungen und imponiert durch eine Trias aus psychiatrische Veränderungen, Demenz und Motorfunktionsstörungen. Auf subzellulärer Ebene ist eine Mutation mit verlängerten CAG-Trinukleotidrepeats als Ursache der Chorea Huntington identifziert worden. Die therapeutischen Effekte bestimmter Substanzen können auf die in neurochemisch-indizierten und transgenen Tiermodellen mit expandierte CAG-Repeats getestet werden. In der vorliegenden Erfindung wurden daher transgene Ratten für die humane HD generiert und charakterisiert. Dieses Rattenmodell für die humane HD und andere Erkrankungen des ZNS trägt 51 CAG Repeats unter der Kontrolle eines Rattenpromotors und weist einen langsam progredienten neurologischen Phänotyp auf, der eng das humane HD-Syndrom widerspiegelt. Die Vergleichbarkeit des Rattenmodells zur humanen HD zeigt sich in neuropathologischen, neuroradiologischen und neurochemischen Veränderungen einhergehend mit typischen Verhaltensauffälligkeiten.
PCT/DE2003/001546 2002-05-14 2003-05-14 Transgene ratte als tiermodell für die humane chorea huntington erkrankung WO2003095640A2 (de)

Priority Applications (5)

Application Number Priority Date Filing Date Title
AU2003243893A AU2003243893A1 (en) 2002-05-14 2003-05-14 Transgenic rat as animal model for human Huntingdon's Disease
DE10393086T DE10393086D2 (de) 2002-05-14 2003-05-14 Transgene Ratte und ihre Verwendung im Tiermodell für die humane chorea huntington Erkrankung sowie Nukleinsäurekonstrukte, Vektoren und Zellen zu ihrer Erzeugung
EP03749850A EP1534826A2 (de) 2002-05-14 2003-05-14 Transgene ratte und ihre verwendung im tiermodell f r die hu mane chorea huntington erkrankung sowie nukleins urekonstruk te, vektoren und zellen zu ihrer erzeugung
CA002523888A CA2523888A1 (en) 2002-05-14 2003-05-14 Transgenic rat and the use thereof in the animal model for human huntingdon's disease and nuclear constructs, vectors and cells for the production thereof
US10/514,512 US20070044162A1 (en) 2002-05-14 2003-05-14 Transgenic rat as animal model for human huntingdon's disease

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
DE10221344A DE10221344A1 (de) 2002-05-14 2002-05-14 Transgene Ratte und ihre Verwendung im Tiermodell für die humane Chorea Huntington Erkrankung sowie Nukleinsäurekonstrukte, Vektoren und Zellen zu ihrer Erzeugung
DE10221344.5 2002-05-14

Publications (2)

Publication Number Publication Date
WO2003095640A2 WO2003095640A2 (de) 2003-11-20
WO2003095640A3 true WO2003095640A3 (de) 2004-01-22

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/DE2003/001546 WO2003095640A2 (de) 2002-05-14 2003-05-14 Transgene ratte als tiermodell für die humane chorea huntington erkrankung

Country Status (6)

Country Link
US (1) US20070044162A1 (de)
EP (1) EP1534826A2 (de)
AU (1) AU2003243893A1 (de)
CA (1) CA2523888A1 (de)
DE (2) DE10221344A1 (de)
WO (1) WO2003095640A2 (de)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8150629B2 (en) 2005-11-10 2012-04-03 In Silico Biosciences Method and apparatus for computer modeling of the interaction between and among cortical and subcortical areas in the human brain for the purpose of predicting the effect of drugs in psychiatric and cognitive diseases
US9314005B2 (en) 2009-07-01 2016-04-19 Transposagen Biopharmaceuticals, Inc. Genetically modified rat models for severe combined immunodeficiency (SCID)
WO2011014721A2 (en) * 2009-07-30 2011-02-03 Transposagen Biopharmaceuticals, Inc. Genetically modified rat models for pharmacokinetics
WO2011046189A1 (ja) * 2009-10-15 2011-04-21 国立大学法人京都大学 神経変性疾患のモデル細胞、その製造方法及びその用途
US9617230B2 (en) 2014-12-22 2017-04-11 Farmington Pharma Development Creatine prodrugs, compositions and methods of use thereof
US20210092942A1 (en) * 2017-03-31 2021-04-01 Hera Testing Laboratories, Inc. Novel immunodeficient rat for modeling human cancer
CN111417620B (zh) 2017-12-01 2023-08-25 奥特吉尼克斯制药公司 肌酸前药、其组合物以及使用方法

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
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51st Annual Meeting of the American Society of Human Genetics; San Diego, California, USA; October 12-16, 2001 *
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RIESS O; ET AL.: "Slowly progressive cerebral dysfunction with selective striatal neuronal inclusions in a rat model transgenic for the Huntington disease mutation", AMERICAN JOURNAL OF HUMAN GENETICS, vol. 69, no. 4, October 2001 (2001-10-01), pages 616, XP001170363 *
RIESS O; NGUYEN H P; SCHMITT I; HOLZMANN C; WALTHER T; BADER M; KASK A; VAARMANN A;ET AL.: "TRANSGENIC RAT MODEL OF HUNTINGTON 'S DISEASE.", SOCIETY FOR NEUROSCIENCE ABSTRACT VIEWER AND ITINERARY PLANNER, vol. 2002, November 2002 (2002-11-01), pages ABSTRACT NO. 417.6, XP001170362 *
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Also Published As

Publication number Publication date
EP1534826A2 (de) 2005-06-01
DE10393086D2 (de) 2005-05-12
DE10221344A1 (de) 2003-12-04
WO2003095640A2 (de) 2003-11-20
CA2523888A1 (en) 2003-11-20
AU2003243893A1 (en) 2003-11-11
US20070044162A1 (en) 2007-02-22

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