JP7669113B2 - トランスフェリン受容体トランスジェニックモデル - Google Patents

トランスフェリン受容体トランスジェニックモデル Download PDF

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JP7669113B2
JP7669113B2 JP2019545322A JP2019545322A JP7669113B2 JP 7669113 B2 JP7669113 B2 JP 7669113B2 JP 2019545322 A JP2019545322 A JP 2019545322A JP 2019545322 A JP2019545322 A JP 2019545322A JP 7669113 B2 JP7669113 B2 JP 7669113B2
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JP2020510418A (ja
JP2020510418A5 (enExample
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マーク エス. デニス
アダム ピー. シルバーマン
ジョイ ユ ズチェロ
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Denali Therapeutics Inc
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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
    • 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
    • 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
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70582CD71
    • 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
    • C07K14/79Transferrins, e.g. lactoferrins, ovotransferrins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008Chemical 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
    • G01N33/5082Supracellular entities, e.g. tissue, organisms
    • G01N33/5088Supracellular entities, e.g. tissue, organisms of vertebrates
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/5308Immunoassay; Biospecific binding assay; Materials therefor for analytes not provided for elsewhere, e.g. nucleic acids, uric acid, worms, mites
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • 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
    • 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
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
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    • 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
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2500/00Screening for compounds of potential therapeutic value
    • G01N2500/10Screening for compounds of potential therapeutic value involving cells

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  • Genetics & Genomics (AREA)
  • Urology & Nephrology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Food Science & Technology (AREA)
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  • Animal Husbandry (AREA)
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  • Biodiversity & Conservation Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Plant Pathology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2019545322A 2017-02-17 2018-02-15 トランスフェリン受容体トランスジェニックモデル Active JP7669113B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023085382A JP2023116513A (ja) 2017-02-17 2023-05-24 トランスフェリン受容体トランスジェニックモデル
JP2025036281A JP2025087857A (ja) 2017-02-17 2025-03-07 トランスフェリン受容体トランスジェニックモデル

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201762460692P 2017-02-17 2017-02-17
US62/460,692 2017-02-17
US201762543559P 2017-08-10 2017-08-10
US201762543658P 2017-08-10 2017-08-10
US62/543,559 2017-08-10
US62/543,658 2017-08-10
US201762583314P 2017-11-08 2017-11-08
US62/583,314 2017-11-08
PCT/US2018/018302 WO2018152285A1 (en) 2017-02-17 2018-02-15 Transferrin receptor transgenic models

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JP2020510418A JP2020510418A (ja) 2020-04-09
JP2020510418A5 JP2020510418A5 (enExample) 2021-03-11
JP7669113B2 true JP7669113B2 (ja) 2025-04-28

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JP2023085382A Pending JP2023116513A (ja) 2017-02-17 2023-05-24 トランスフェリン受容体トランスジェニックモデル
JP2025036281A Pending JP2025087857A (ja) 2017-02-17 2025-03-07 トランスフェリン受容体トランスジェニックモデル

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EP (2) EP4628586A2 (enExample)
JP (3) JP7669113B2 (enExample)
KR (1) KR102637590B1 (enExample)
CN (1) CN110382526B (enExample)
AU (1) AU2018221704B2 (enExample)
CA (1) CA3053370A1 (enExample)
IL (1) IL268659A (enExample)
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BR112020005271A2 (pt) 2017-10-02 2020-09-15 Denali Therapeutics Inc. proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica
SG11202011743SA (en) 2018-06-18 2021-01-28 Denali Therapeutics Inc Fusion proteins comprising progranulin
WO2021133907A1 (en) 2019-12-23 2021-07-01 Denali Therapeutics Inc. Progranulin variants
CN112243953B (zh) * 2020-07-15 2022-02-11 吉林医药学院 TfR1肺组织特异性敲除小鼠模型及其构建方法和应用
PE20231931A1 (es) 2020-10-14 2023-12-01 Denali Therapeutics Inc Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas
CN112501124A (zh) * 2020-11-27 2021-03-16 领诺(上海)医药科技有限公司 稳定表达人转铁蛋白受体1细胞株的制备方法及其应用
EP4370539A4 (en) * 2021-07-16 2025-05-28 Biocytogen Pharmaceuticals (Beijing) Co., Ltd. Genetically modified non-human animal with human or chimeric tfr1
US20250136963A1 (en) 2021-12-17 2025-05-01 Denali Therapeutics Inc. Fusion proteins comprising alpha-l-iduronidase enzymes and methods
CA3242959A1 (en) 2021-12-17 2025-02-26 Denali Therapeutics Inc POLYPEPTIDE ENGINEERING, LIBRARIES AND ENGINEERED POLYPEPTIDES FOR CD98 HEAVY CHAIN LINK AND TRANSFERRINE RECEIVER
US20250215077A1 (en) 2022-03-28 2025-07-03 Denali Therapeutics Inc. Monovalent anti-trem2 binding molecules and methods of use thereof
KR20250048010A (ko) * 2022-07-29 2025-04-07 리제너론 파마슈티칼스 인코포레이티드 변형된 트랜스페린 수용체 좌위를 포함하는 비-인간 동물
EP4658684A1 (en) 2023-01-30 2025-12-10 ISAR Bioscience GmbH Human anti-trem2 antibody for treating neurodegenerative disorders
CN116784280B (zh) * 2023-08-10 2025-08-19 广东药康生物科技有限公司 一种tfrc人源化小鼠模型的构建方法及其应用
WO2025137213A1 (en) 2023-12-20 2025-06-26 Denali Therapeutics Inc. Fusion proteins comprising acid alpha-glucosidase enzymes and methods thereof

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JP2016526878A (ja) 2013-05-20 2016-09-08 ジェネンテック, インコーポレイテッド 抗トランスフェリン受容体抗体及び使用方法

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KR20190135993A (ko) 2019-12-09
JP2020510418A (ja) 2020-04-09
WO2018152285A1 (en) 2018-08-23
SG11201907419PA (en) 2019-09-27
EP3583118C0 (en) 2025-06-11
EP4628586A2 (en) 2025-10-08
EP3583118B1 (en) 2025-06-11
CA3053370A1 (en) 2018-08-23
IL268659A (en) 2019-10-31
CN110382526B (zh) 2023-12-26
CN110382526A (zh) 2019-10-25
JP2023116513A (ja) 2023-08-22
EP3583118A1 (en) 2019-12-25
AU2018221704A1 (en) 2019-09-19
JP2025087857A (ja) 2025-06-10
AU2018221704B2 (en) 2021-08-19
KR102637590B1 (ko) 2024-02-15

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