EA201590232A1 - Животная модель болезни краббе - Google Patents

Животная модель болезни краббе

Info

Publication number
EA201590232A1
EA201590232A1 EA201590232A EA201590232A EA201590232A1 EA 201590232 A1 EA201590232 A1 EA 201590232A1 EA 201590232 A EA201590232 A EA 201590232A EA 201590232 A EA201590232 A EA 201590232A EA 201590232 A1 EA201590232 A1 EA 201590232A1
Authority
EA
Eurasian Patent Office
Prior art keywords
animal model
crabbe
disease
globoid cell
cell leukodystrophy
Prior art date
Application number
EA201590232A
Other languages
English (en)
Other versions
EA030305B1 (ru
Inventor
Йенс Фог
Клаэс Андерссон
Original Assignee
Эйс Байосайенсиз А/С
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 Эйс Байосайенсиз А/С filed Critical Эйс Байосайенсиз А/С
Publication of EA201590232A1 publication Critical patent/EA201590232A1/ru
Publication of EA030305B1 publication Critical patent/EA030305B1/ru

Links

Classifications

    • 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
    • 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
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • 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; 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01046—Galactosylceramidase (3.2.1.46)

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Environmental Sciences (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Animal Husbandry (AREA)
  • Medicinal Chemistry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Rheumatology (AREA)
  • Public Health (AREA)
  • Diabetes (AREA)
  • Toxicology (AREA)
  • Urology & Nephrology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Plant Pathology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Investigating Or Analysing Biological Materials (AREA)

Abstract

Настоящее изобретение относится к животной модели детской глобоидно-клеточной лейкодистрофии и к применению указанной животной модели для скрининга и/или оценки агентов, которые могут быть полезны в качестве лекарственного средства для лечения глобоидно-клеточной лейкодистрофии.
EA201590232A 2012-08-07 2013-08-07 Животная модель болезни краббе EA030305B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
DKPA201270467 2012-08-07
PCT/DK2013/050260 WO2014023314A1 (en) 2012-08-07 2013-08-07 Animal model of krabbe's disease

Publications (2)

Publication Number Publication Date
EA201590232A1 true EA201590232A1 (ru) 2015-07-30
EA030305B1 EA030305B1 (ru) 2018-07-31

Family

ID=48985927

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201590232A EA030305B1 (ru) 2012-08-07 2013-08-07 Животная модель болезни краббе

Country Status (6)

Country Link
US (1) US9986722B2 (ru)
EP (1) EP2882284B1 (ru)
CN (1) CN104735976B (ru)
EA (1) EA030305B1 (ru)
HK (1) HK1211790A1 (ru)
WO (1) WO2014023314A1 (ru)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018210326B9 (en) * 2017-01-20 2024-04-04 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Treatment of krabbe disease with umbilical cord blood transplantion (UCBT) and increased galactocerebrosidase (GALC) expression
WO2020176562A1 (en) * 2019-02-26 2020-09-03 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
EP4153756A1 (en) 2020-05-22 2023-03-29 NEUWAY Pharma GmbH Vlp for the treatment of leukodystrophies
CN113265461B (zh) * 2021-07-02 2022-05-13 北京华诺奥美医学检验实验室有限公司 一种用于检测高频基因致病变异的引物组和探针组及试剂盒

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH01149718A (ja) * 1987-12-07 1989-06-12 Yoshikatsu Eto 脳血液関門を通過し易いリポゾーム製剤
JP2004524002A (ja) * 2000-06-29 2004-08-12 デルタジェン　インコーポレイテッド 標的化された遺伝子破壊を含むトランスジェニックマウス
GB0718824D0 (en) * 2007-09-26 2007-11-07 Univ Ramot Methods of treating lysosomal storage disorders
EP2588132A4 (en) * 2010-06-25 2014-10-15 Shire Human Genetic Therapies METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS

Also Published As

Publication number Publication date
CN104735976A (zh) 2015-06-24
EP2882284B1 (en) 2017-11-15
WO2014023314A1 (en) 2014-02-13
HK1211790A1 (en) 2016-06-03
EP2882284A1 (en) 2015-06-17
CN104735976B (zh) 2017-03-01
US20150196014A1 (en) 2015-07-16
EA030305B1 (ru) 2018-07-31
US9986722B2 (en) 2018-06-05

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Legal Events

Date Code Title Description
MM4A Lapse of a eurasian patent due to non-payment of renewal fees within the time limit in the following designated state(s)

Designated state(s): AM AZ BY KZ KG TJ TM

MM4A Lapse of a eurasian patent due to non-payment of renewal fees within the time limit in the following designated state(s)

Designated state(s): RU