WO2001016176A3 - Transgenic animal model for neurodegenerative diseases - Google Patents

Transgenic animal model for neurodegenerative diseases Download PDF

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Publication number
WO2001016176A3
WO2001016176A3 PCT/EP2000/008071 EP0008071W WO0116176A3 WO 2001016176 A3 WO2001016176 A3 WO 2001016176A3 EP 0008071 W EP0008071 W EP 0008071W WO 0116176 A3 WO0116176 A3 WO 0116176A3
Authority
WO
WIPO (PCT)
Prior art keywords
neurodegenerative diseases
transgenic animal
human
animal model
sequence
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/EP2000/008071
Other languages
French (fr)
Other versions
WO2001016176A2 (en
Inventor
Hermann Luebbert
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Biofrontera Bioscience GmbH
Original Assignee
Biofrontera Pharmaceuticals GmbH
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 Biofrontera Pharmaceuticals GmbH filed Critical Biofrontera Pharmaceuticals GmbH
Priority to ES00956461T priority Critical patent/ES2304973T3/en
Priority to DE60038966T priority patent/DE60038966D1/en
Priority to CA002383564A priority patent/CA2383564A1/en
Priority to JP2001520735A priority patent/JP2003512026A/en
Priority to EP00956461A priority patent/EP1208200B1/en
Publication of WO2001016176A2 publication Critical patent/WO2001016176A2/en
Publication of WO2001016176A3 publication Critical patent/WO2001016176A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93Ligases (6)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/0004Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • 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; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00Modified animals
    • A01K2207/15Humanized animals
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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
    • A01K2267/0306Animal model for genetic diseases
    • A01K2267/0318Animal model for neurodegenerative disease, e.g. non- Alzheimer's

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Pathology (AREA)
  • Urology & Nephrology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Rheumatology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

The present application refers to a mouse parkin2 DNA- and protein sequence, containing mutations or deletions causing Parkinson's disease in a human if occurring in the according human sequence, the construction of a transgenic non-human animal containing such a mutated DNA sequence and therefore expressing no or a less active or non-active parkin protein as well as the use of this transgenic animal as a model for neurodegenerative diseases.
PCT/EP2000/008071 1999-08-30 2000-08-18 Transgenic animal model for neurodegenerative diseases Ceased WO2001016176A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
ES00956461T ES2304973T3 (en) 1999-08-30 2000-08-18 TRANSGENIC ANIMAL MODEL FOR NEURODEGENERATIVE DISEASES.
DE60038966T DE60038966D1 (en) 1999-08-30 2000-08-18 Transgenic animal model for neurodegenerative diseases
CA002383564A CA2383564A1 (en) 1999-08-30 2000-08-18 Transgenic animal model for neurodegenerative diseases
JP2001520735A JP2003512026A (en) 1999-08-30 2000-08-18 Transgenic animal models for neurodegenerative diseases
EP00956461A EP1208200B1 (en) 1999-08-30 2000-08-18 Transgenic animal model for neurodegenerative diseases

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP99116766.9 1999-08-30
EP99116766A EP1081225A1 (en) 1999-08-30 1999-08-30 Transgenic animal model for neurodegenerative diseases

Publications (2)

Publication Number Publication Date
WO2001016176A2 WO2001016176A2 (en) 2001-03-08
WO2001016176A3 true WO2001016176A3 (en) 2001-09-27

Family

ID=8238860

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2000/008071 Ceased WO2001016176A2 (en) 1999-08-30 2000-08-18 Transgenic animal model for neurodegenerative diseases

Country Status (7)

Country Link
EP (2) EP1081225A1 (en)
JP (1) JP2003512026A (en)
AT (1) ATE396263T1 (en)
CA (1) CA2383564A1 (en)
DE (1) DE60038966D1 (en)
ES (1) ES2304973T3 (en)
WO (1) WO2001016176A2 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2845245A1 (en) * 2002-10-07 2004-04-09 Aventis Pharma Sa Transgenic animal model of Parkinson's disease, useful in screening for agents effective against neurodegenerative diseases, contains a parkin gene with a deletion in exon 3
US6943278B2 (en) * 2002-10-15 2005-09-13 Genexel, Inc. Transgenic Drosophila having a disrupted Parkin gene and exhibits reduced climbing ability
CN113373150B (en) * 2021-06-18 2022-11-22 南方科技大学 sgRNA of targeting dat gene and application thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998059050A1 (en) * 1997-06-25 1998-12-30 The Government Of The United States Of America Represented By The Secretary, Department Of Health And Human Services Cloning of a gene mutation for parkinson's disease
WO1999040191A1 (en) * 1998-02-09 1999-08-12 Boehringer Ingelheim International Gmbh Dnas or genes participating in parkinson's disease
WO2000031253A2 (en) * 1998-11-19 2000-06-02 Aventis Pharma S.A. Mutation of the parkin gene, compositions, methods and uses

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998059050A1 (en) * 1997-06-25 1998-12-30 The Government Of The United States Of America Represented By The Secretary, Department Of Health And Human Services Cloning of a gene mutation for parkinson's disease
WO1999040191A1 (en) * 1998-02-09 1999-08-12 Boehringer Ingelheim International Gmbh Dnas or genes participating in parkinson's disease
WO2000031253A2 (en) * 1998-11-19 2000-06-02 Aventis Pharma S.A. Mutation of the parkin gene, compositions, methods and uses

Non-Patent Citations (10)

* Cited by examiner, † Cited by third party
Title
ABBAS ET AL: "A wide variety of mutations in the parkin gene are responsible for autosomal recessive parkinsonism in Europe", HUMAN MOLECULAR GENETICS,GB,OXFORD UNIVERSITY PRESS, SURREY, vol. 8, no. 4, April 1999 (1999-04-01), pages 567 - 574, XP002108471, ISSN: 0964-6906 *
DALIE J E (PROGRAM MANAGER - SOCIETY FOR NEUROSCIENCE): "Publication dates for the 1999 Abstract Volumes", SOCIETY FOR NEUROSCIENCE ABSTRACTS, 16 August 1999 (1999-08-16), XP002157614 *
DATABASE EMROD E.M.B.L. Databases; 13 July 1999 (1999-07-13), SHIMIZU N ET AL: "Mus musculus mRNA for parkin, complete cds", XP002131476 *
GOLDBERG M S ET AL: "STUDIES OF WILD-TYPE AND MUTANT ALPHA-SYNUCLEIN IN TRANSGENIC MICE", ANNUAL MEETING SOCIETY NEUROSCIENCE,XX,XX, vol. 24, no. 1/02, 1998, pages 966, XP000884112 *
HATTORI ET AL: "Point Mutations (Thr240Arg and Ala311Stop) in the Parkin Gene", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS,US,ACADEMIC PRESS INC. ORLANDO, FL, vol. 249, no. 3, 1998, pages 754 - 758, XP002108468, ISSN: 0006-291X *
HATTORI N ET AL: "Molecular genetic analysis of a novel Parkin gene in Japanese families with autosomal recessive juvenile parkinsonism: evidence for variable homozygous deletions in the Parkin gene in affected individuals", ANN NEUROL., vol. 44, no. 6, December 1998 (1998-12-01), pages 935 - 941, XP000877155 *
KESSLER J ET AL: "Investigation of the pathogenic mechanism of parkin mutations", SOCIETY FOR NEUROSCIENCE ABSTRACTS, vol. 25, no. 1-2, 1999, pages 52 - Abstract 27.20, XP000884113 *
KITADA ET AL: "Mutations in the parkin gene cause autosomal recessive juvenile parkinsonism", NATURE,GB,MACMILLAN JOURNALS LTD. LONDON, vol. 392, no. 6676, 9 April 1998 (1998-04-09), pages 605 - 608, XP002108469, ISSN: 0028-0836 *
LEROY ET AL: "Deletions in the Parkin gene and genetic heterogeneity in a Greek family with early onset Parkinson's disease", HUMAN GENETICS,DE,BERLIN, vol. 103, no. 4, October 1998 (1998-10-01), pages 424 - 427, XP002108470 *
LÜCKING ET AL: "Homozygous deletions in parkin gene in European and North African families with autosomal recessive juvenile parkinsonism", LANCET THE,GB,LANCET LIMITED. LONDON, vol. 352, no. 9137, 24 October 1998 (1998-10-24), pages 1355 - 1356, XP002108466, ISSN: 0140-6736 *

Also Published As

Publication number Publication date
JP2003512026A (en) 2003-04-02
ATE396263T1 (en) 2008-06-15
CA2383564A1 (en) 2001-03-08
DE60038966D1 (en) 2008-07-03
WO2001016176A2 (en) 2001-03-08
EP1208200B1 (en) 2008-05-21
EP1081225A1 (en) 2001-03-07
EP1208200A2 (en) 2002-05-29
ES2304973T3 (en) 2008-11-01

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