HK1248465A1 - 具有c9orf72破壞的非人類動物 - Google Patents

具有c9orf72破壞的非人類動物

Info

Publication number
HK1248465A1
HK1248465A1 HK18107516.0A HK18107516A HK1248465A1 HK 1248465 A1 HK1248465 A1 HK 1248465A1 HK 18107516 A HK18107516 A HK 18107516A HK 1248465 A1 HK1248465 A1 HK 1248465A1
Authority
HK
Hong Kong
Prior art keywords
ac9orf72
disruption
human animals
animals
human
Prior art date
Application number
HK18107516.0A
Other languages
English (en)
Inventor
Amanda Atanasio
Burcin Ikiz
Guochun Gong
Michael L Lacroix-Fralish
Ka-Man Venus Lai
David M Valenzuela
Original Assignee
Regeneron Pharma
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 Regeneron Pharma filed Critical Regeneron Pharma
Publication of HK1248465A1 publication Critical patent/HK1248465A1/zh

Links

Classifications

    • 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
    • A01K67/0276—Knockout animals
    • 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
    • A01K67/02—Breeding 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • A01K2217/07—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; CARE OF BIRDS, FISHES, INSECTS; 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
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • 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
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0381—Animal model for diseases of the hematopoietic system
    • 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
    • A01K2267/0393—Animal model comprising a reporter system for screening tests

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Environmental Sciences (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Rheumatology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Toxicology (AREA)
  • Pathology (AREA)
  • Diabetes (AREA)
HK18107516.0A 2015-05-29 2018-06-08 具有c9orf72破壞的非人類動物 HK1248465A1 (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562168171P 2015-05-29 2015-05-29
US201562232658P 2015-09-25 2015-09-25
US201562245382P 2015-10-23 2015-10-23
PCT/US2016/034304 WO2016196185A1 (en) 2015-05-29 2016-05-26 Non-human animals having a disruption in a c9orf72 locus

Publications (1)

Publication Number Publication Date
HK1248465A1 true HK1248465A1 (zh) 2018-10-19

Family

ID=56194557

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18107516.0A HK1248465A1 (zh) 2015-05-29 2018-06-08 具有c9orf72破壞的非人類動物

Country Status (15)

Country Link
US (2) US10285388B2 (zh)
EP (2) EP3302048B1 (zh)
JP (3) JP6619822B2 (zh)
KR (2) KR102190661B1 (zh)
CN (1) CN107920498A (zh)
AU (2) AU2016270587B2 (zh)
BR (1) BR112017025507A2 (zh)
CA (1) CA2986048C (zh)
ES (1) ES2784360T3 (zh)
HK (1) HK1248465A1 (zh)
IL (2) IL255385A0 (zh)
MX (1) MX2017015369A (zh)
RU (2) RU2020115485A (zh)
SG (1) SG10201911257YA (zh)
WO (1) WO2016196185A1 (zh)

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JP6827113B2 (ja) 2017-01-21 2021-02-10 広州白雲山漢方現代薬業有限公司Ｇｕａｎｇｚｈｏｕ Ｈａｎｆａｎｇ Ｐｈａｒｍａｃｅｕｔｉｃａｌ Ｃｏ．，Ｌｔｄ． シェーグレン症候群の治療におけるペオニフロリン−６’−ｏ−ベンゼンスルホン酸の使用
CN107693542B (zh) * 2017-09-05 2020-03-10 中山大学附属第三医院 一种系统性红斑狼疮动物模型的构建方法及其应用
CN109452229B (zh) * 2018-11-19 2021-10-22 百奥赛图(北京)医药科技股份有限公司 狗源化pd-1基因改造动物模型的制备方法及应用
SG11202105189RA (en) 2018-12-20 2021-06-29 Regeneron Pharma Nuclease-mediated repeat expansion
CN113046390B (zh) * 2020-03-09 2024-01-09 百奥赛图江苏基因生物技术有限公司 Csf1r基因人源化的非人动物及其构建方法和应用

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Also Published As

Publication number Publication date
EP3302048A1 (en) 2018-04-11
US11547101B2 (en) 2023-01-10
EP3302048B1 (en) 2020-02-19
CA2986048C (en) 2021-10-26
RU2017145143A3 (zh) 2019-10-17
CN107920498A (zh) 2018-04-17
WO2016196185A1 (en) 2016-12-08
KR102190661B1 (ko) 2020-12-14
BR112017025507A2 (pt) 2018-08-07
MX2017015369A (es) 2018-07-06
US10285388B2 (en) 2019-05-14
AU2021201827A1 (en) 2021-04-22
SG10201911257YA (en) 2020-01-30
JP2019201671A (ja) 2019-11-28
KR20200140934A (ko) 2020-12-16
AU2016270587A1 (en) 2017-11-30
RU2020115485A (ru) 2020-06-11
RU2725737C2 (ru) 2020-07-03
JP6884831B2 (ja) 2021-06-09
EP3689139A1 (en) 2020-08-05
RU2017145143A (ru) 2019-07-02
NZ737000A (en) 2021-02-26
IL255385A0 (en) 2017-12-31
JP6619822B2 (ja) 2019-12-11
US20160345547A1 (en) 2016-12-01
KR102377189B1 (ko) 2022-03-22
IL286176A (en) 2021-10-31
CA2986048A1 (en) 2016-12-08
JP2021118745A (ja) 2021-08-12
KR20180012323A (ko) 2018-02-05
JP2018518165A (ja) 2018-07-12
ES2784360T3 (es) 2020-09-24
AU2021201827B2 (en) 2023-11-09
AU2016270587B2 (en) 2020-12-24
US20190216062A1 (en) 2019-07-18

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