HRP20170380T1 - Životinje s humaniziranim il-5 - Google Patents

Životinje s humaniziranim il-5 Download PDF

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Publication number
HRP20170380T1
HRP20170380T1 HRP20170380TT HRP20170380T HRP20170380T1 HR P20170380 T1 HRP20170380 T1 HR P20170380T1 HR P20170380T T HRP20170380T T HR P20170380TT HR P20170380 T HRP20170380 T HR P20170380T HR P20170380 T1 HRP20170380 T1 HR P20170380T1
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Croatia
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human
mouse
exons
humanized
protein
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HRP20170380TT
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Jose F. Rojas
Ka-Man Venus Lai
Andrew J. Murphy
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Regeneron Pharmaceuticals, Inc.
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Publication of HRP20170380T1 publication Critical patent/HRP20170380T1/hr

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    • 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
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278Knock-in vertebrates, e.g. humanised vertebrates
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/54Interleukins [IL]
    • C07K14/5443IL-15
    • 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
    • A01K2217/07Animals genetically altered by homologous recombination
    • 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
    • A01K2217/072Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • 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
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • 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
    • A01K2267/035Animal model for multifactorial diseases
    • A01K2267/0387Animal model for diseases of the immune system
    • 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
    • C12N2015/8527Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Environmental Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Claims (12)

1. Genetički modificirani miš, naznačen time što mu genom sadrži zamjenu u endogenom mišjem lokusu IL-15, u mišjem genomskom fragmentu koji kodira zreli mišji polipeptid IL-15, ljudskim genomskim segmentom koji kodira zreli ljudski polipeptid IL-15, gdje ljudske egzone u IL-15 u navedenom ljudskom genomskom segmentu čine ljudski egzoni 3, 4, 5 i 6 koji kodiraju protein IL-15, kako bi se dobilo humanizirani gen za IL-15, gdje su egzoni u IL-15 tako obrojčani da je prvi kodirajući egzon egzon 1, te gdje je humanizirani gen za IL-15 pod kontrolom svih endogenih mišjih uzvodnih regulacijskih elemenata za IL-15 na endogenom mišjem lokusu IL-15.
2. Genetički modificirani miš u skladu s patentnim zahtjevom 1, naznačen time što humanizirani gen koji kodira IL-15 kodira protein koji sadrži aminokiselinski slijed sa SEQ ID NO: 5.
3. Genetički modificirani miš u skladu s patentnim zahtjevom 1, naznačen time što ljudski genomski segment dodatno sadrži slijed ljudskog introna uzvodno od naj-5′-smještenog ljudskog egzona i ljudski slijed koji ne kodira protein nizvodno od ljudskog stop kodona i nizvodno od ljudskog 3′ UTR.
4. Postupak dobivanja gentički modificiranog miša, naznačen time što se sastoji u modificiranju mišjeg genoma zamjenom mišjeg genomskog fragmenta koji kodira zreli mišji polipeptid IL-15 na endogenom mišjem lokusu IL-15 ljudskim genomskim segmentom koji kodira zreli ljudski polipeptid IL-15, gdje ljudske egzone u IL-15 u navedenom ljudskom genomskom segmentu čine ljudski egzoni 3, 4, 5 i 6 koji kodiraju protein IL-15, kako bi se dobilo humanizirani gen za IL-15, gdje su egzoni u IL-15 tako obrojčani da je prvi kodirajući egzon egzon 1, te gdje je humanizirani gen za IL-15 pod kontrolom svih endogenih mišjih uzvodnih regulacijskih elemenata za IL-15 na endogenom mišjem lokusu IL-15.
5. Postupak u skladu s patentnim zahtjevom 4, naznačen time što ljudski genomski segment dodatno sadrži slijed ljudskog introna uzvodno od naj-5′-smještenog ljudskog egzona i ljudski slijed koji ne kodira protein nizvodno od ljudskog stop kodona i nizvodno od ljudskog 3′ UTR.
6. Postupak u skladu s patentnim zahtjevom 4, naznačen time što humanizirani gen koji kodira IL-15 kodira protein koji sadrži aminokiselinski slijed sa SEQ ID NO: 5.
7. Genetički modificirani miš, naznačen time što mu genom sadrži humanizirani gen za IL-15, gdje navedeni humanizirani gen za IL-15 sadrži mišje egzone 1 i 2 koji kodiraju protein IL-15 i ljudski genomski segment, gdje ljudske egzone u IL-15 u navedenom ljudskom genomskom segmentu čine ljudski egzoni 3, 4, 5 i 6 koji kodiraju protein IL-15, gdje su egzoni u IL-15 tako obrojčani da je prvi kodirajući egzon egzon 1, gdje je humanizirani gen za IL-15 pod kontrolom svih mišjih regulacijskih elemenata za IL-15 uzvodno od endogenog mišjeg lokusa IL-15, te gdje navedeni humanizirani gen za IL-15 kodira protein koji sadrži slijed zrelog ljudskog polipeptida IL-15.
8. Genetički modificirani miš u skladu s patentnim zahtjevom 7, naznačen time što humanizirani gen koji kodira IL-15 kodira protein koji sadrži aminokiselinski slijed sa SEQ ID NO: 5.
9. Genetički modificirani miš u skladu s patentnim zahtjevom 7, naznačen time što ljudski genomski segment dodatno sadrži slijed ljudskog introna uzvodno od naj-5′-smještenog ljudskog egzona i ljudski slijed koji ne kodira protein nizvodno od ljudskog stop kodona i nizvodno od ljudskog 3′ UTR.
10. Genetički modificirani miš u skladu s patentnim zahtjevom 7, naznačen time što je humanizirani gen za IL-15 na položaju u mišjem genomu koji nije endogeni lokus IL-15.
11. Postupak dobivanja gentički modificiranog miša, naznačen time što se sastoji u uvođenju u mišji genom humaniziranog gena za IL-15 koji sadrži mišje egzone 1 i 2 koji kodiraju protein IL-15 i ljudski genomski segment, gdje ljudske egzone u IL-15 u navedenom ljudskom genomskom segmentu čine ljudski egzoni 3, 4, 5 i 6 u IL-15, gdje su egzoni u IL-15 tako obrojčani da je prvi kodirajući egzon egzon 1, gdje je humanizirani gen za IL-15 pod kontrolom svih mišjih regulacijskih elemenata za IL-15 uzvodno od endogenog mišjeg lokusa IL-15, te gdje navedeni humanizirani gen za IL-15 kodira protein koji sadrži slijed zrelog ljudskog polipeptida IL-15.
12. Postupak u skladu s patentnim zahtjevom 11, naznačen time što je humanizirani gen za IL-15 integriran u genom na položaju koji nije endogeni lokus IL-15.
HRP20170380TT 2013-10-15 2017-03-07 Životinje s humaniziranim il-5 HRP20170380T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361891013P 2013-10-15 2013-10-15
EP14790452.8A EP2908626B1 (en) 2013-10-15 2014-10-15 Humanized il-15 animals
PCT/US2014/060568 WO2015057758A1 (en) 2013-10-15 2014-10-15 Humanized il-15 animals

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HRP20170380T1 true HRP20170380T1 (hr) 2017-05-19

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EP (3) EP3138397B1 (hr)
JP (3) JP6492095B2 (hr)
KR (1) KR102376041B1 (hr)
CN (2) CN105744829B (hr)
AU (2) AU2014337526B2 (hr)
BR (1) BR112016008175A2 (hr)
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HK (1) HK1210659A1 (hr)
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HU (2) HUE033272T2 (hr)
IL (2) IL244525B (hr)
LT (2) LT3138397T (hr)
MX (1) MX2016004781A (hr)
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PT (2) PT2908626T (hr)
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SG (2) SG10201607768YA (hr)
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