HK1211790A1 - 克拉伯病的動物模型 - Google Patents

克拉伯病的動物模型

Info

Publication number
HK1211790A1
HK1211790A1 HK15112630.4A HK15112630A HK1211790A1 HK 1211790 A1 HK1211790 A1 HK 1211790A1 HK 15112630 A HK15112630 A HK 15112630A HK 1211790 A1 HK1211790 A1 HK 1211790A1
Authority
HK
Hong Kong
Prior art keywords
animal model
krabbes disease
krabbes
disease
animal
Prior art date
Application number
HK15112630.4A
Other languages
English (en)
Inventor
延斯‧福格
克拉斯‧安德松
Original Assignee
Chiesi Farma Spa
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 Chiesi Farma Spa filed Critical Chiesi Farma Spa
Publication of HK1211790A1 publication Critical patent/HK1211790A1/zh

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)
  • Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Rheumatology (AREA)
  • Toxicology (AREA)
  • Urology & Nephrology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Diabetes (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)
HK15112630.4A 2012-08-07 2015-12-23 克拉伯病的動物模型 HK1211790A1 (zh)

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 (1)

Publication Number Publication Date
HK1211790A1 true HK1211790A1 (zh) 2016-06-03

Family

ID=48985927

Family Applications (1)

Application Number Title Priority Date Filing Date
HK15112630.4A HK1211790A1 (zh) 2012-08-07 2015-12-23 克拉伯病的動物模型

Country Status (6)

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

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190336540A1 (en) * 2017-01-20 2019-11-07 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
TW202045728A (zh) * 2019-02-26 2020-12-16 賓州大學委員會 用於治療克拉培氏病之組成物
CA3184307A1 (en) 2020-05-22 2021-11-25 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
CA2416292A1 (en) * 2000-06-29 2002-01-10 Deltagen, Inc. Transgenic mice containing targeted gene disruptions
GB0718824D0 (en) * 2007-09-26 2007-11-07 Univ Ramot Methods of treating lysosomal storage disorders
US20110318324A1 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of b-galactocerebrosidase

Also Published As

Publication number Publication date
EP2882284B1 (en) 2017-11-15
US9986722B2 (en) 2018-06-05
WO2014023314A1 (en) 2014-02-13
EP2882284A1 (en) 2015-06-17
EA030305B1 (ru) 2018-07-31
US20150196014A1 (en) 2015-07-16
EA201590232A1 (ru) 2015-07-30
CN104735976A (zh) 2015-06-24
CN104735976B (zh) 2017-03-01

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Legal Events

Date Code Title Description
PC Patent ceased (i.e. patent has lapsed due to the failure to pay the renewal fee)

Effective date: 20210801