AU2016233250C1 - Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception - Google Patents

Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception Download PDF

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AU2016233250C1
AU2016233250C1 AU2016233250A AU2016233250A AU2016233250C1 AU 2016233250 C1 AU2016233250 C1 AU 2016233250C1 AU 2016233250 A AU2016233250 A AU 2016233250A AU 2016233250 A AU2016233250 A AU 2016233250A AU 2016233250 C1 AU2016233250 C1 AU 2016233250C1
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cell
nucleic acid
rodent
endogenous
locus
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AU2016233250B2 (en
AU2016233250A1 (en
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Susan D. Croll
Burcin IKIZ
Michael LACROIX-FRALISH
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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    • 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
    • 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
    • A01K67/0276—Knock-out vertebrates
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70578—NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
    • G01N33/5058—Neurological cells
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5082—Supracellular entities, e.g. tissue, organisms
    • G01N33/5088—Supracellular entities, e.g. tissue, organisms of vertebrates
    • 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
    • A01K2217/075—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; 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
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
    • 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
    • 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
    • A01K2267/0393—Animal model comprising a reporter system for screening tests
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/61—Fusion polypeptide containing an enzyme fusion for detection (lacZ, luciferase)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2500/00—Screening for compounds of potential therapeutic value
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Cell Biology (AREA)
  • Biotechnology (AREA)
  • Environmental Sciences (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Toxicology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Food Science & Technology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Analytical Chemistry (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2016233250A 2015-03-16 2016-03-16 Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception Active AU2016233250C1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2022200784A AU2022200784B2 (en) 2015-03-16 2022-02-07 Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562133909P 2015-03-16 2015-03-16
US62/133,909 2015-03-16
US201562250229P 2015-11-03 2015-11-03
US62/250,229 2015-11-03
PCT/US2016/022685 WO2016149398A1 (en) 2015-03-16 2016-03-16 Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception

Related Child Applications (1)

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AU2022200784A Division AU2022200784B2 (en) 2015-03-16 2022-02-07 Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception

Publications (3)

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AU2016233250A1 AU2016233250A1 (en) 2017-09-14
AU2016233250B2 AU2016233250B2 (en) 2022-03-03
AU2016233250C1 true AU2016233250C1 (en) 2022-08-18

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EP (2) EP3270688B1 (enExample)
JP (4) JP6600694B2 (enExample)
KR (2) KR102616160B1 (enExample)
CN (1) CN107690279B (enExample)
AU (2) AU2016233250C1 (enExample)
BR (1) BR112017019620A2 (enExample)
CA (1) CA2978096C (enExample)
ES (2) ES2830173T3 (enExample)
IL (2) IL274285B (enExample)
MX (2) MX380521B (enExample)
RU (1) RU2744831C2 (enExample)
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IL294443A (en) * 2013-08-07 2022-09-01 Regeneron Pharma Lincrna-deficient non-human animals
US10781453B2 (en) * 2016-09-30 2020-09-22 Regeneron Pharmaceuticals, Inc. Non-human animals having a hexanucleotide repeat expansion in a C9ORF72 locus
CN111793607B (zh) * 2017-07-28 2022-05-17 杨涛 定向诱导hiPSC分化为神经细胞体系中培养神经元细胞的基础培养基
CN107586791B (zh) * 2017-10-26 2018-09-21 四川省人民医院 一种共济失调动物模型的构建方法以及应用

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Publication number Publication date
ES2830173T3 (es) 2021-06-03
HK1249360A1 (zh) 2018-11-02
JP2018512842A (ja) 2018-05-24
EP3685662B9 (en) 2023-10-04
IL274285B (en) 2022-07-01
CA2978096A1 (en) 2016-09-22
CA2978096C (en) 2022-06-28
CN107690279A (zh) 2018-02-13
BR112017019620A2 (pt) 2018-05-15
US10285387B2 (en) 2019-05-14
AU2022200784A1 (en) 2022-02-24
MX380521B (es) 2025-04-01
RU2017135557A3 (enExample) 2019-07-29
MX2017011918A (es) 2018-05-22
EP3270688B1 (en) 2020-08-12
EP3685662A1 (en) 2020-07-29
ES2955841T3 (es) 2023-12-07
AU2022200784B2 (en) 2024-02-15
IL274285A (en) 2020-06-30
AU2016233250B2 (en) 2022-03-03
RU2017135557A (ru) 2019-04-17
IL254333B (en) 2020-05-31
SG10201912899QA (en) 2020-02-27
NZ734906A (en) 2022-02-25
KR20170127459A (ko) 2017-11-21
MX2021002852A (es) 2021-05-28
KR102424119B1 (ko) 2022-07-25
CN107690279B (zh) 2021-07-02
KR20220108828A (ko) 2022-08-03
JP2020005660A (ja) 2020-01-16
IL254333A0 (en) 2017-11-30
AU2016233250A1 (en) 2017-09-14
RU2744831C2 (ru) 2021-03-16
EP3270688A1 (en) 2018-01-24
SG11201706809WA (en) 2017-09-28
US20210392865A1 (en) 2021-12-23
WO2016149398A1 (en) 2016-09-22
EP3685662B1 (en) 2023-06-07
KR102616160B1 (ko) 2023-12-22
JP2022048157A (ja) 2022-03-25
US20190261610A1 (en) 2019-08-29
US20160270377A1 (en) 2016-09-22
JP7193509B2 (ja) 2022-12-20
EP3685662C0 (en) 2023-06-07
JP2020198892A (ja) 2020-12-17
JP6600694B2 (ja) 2019-10-30

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