IL268659A - Polynucleotides encoding chimeric transferrin receptor polypeptides, host cells and animals comprising same, methods of producing same and uses thereof - Google Patents

Polynucleotides encoding chimeric transferrin receptor polypeptides, host cells and animals comprising same, methods of producing same and uses thereof

Info

Publication number
IL268659A
IL268659A IL26865919A IL26865919A IL268659A IL 268659 A IL268659 A IL 268659A IL 26865919 A IL26865919 A IL 26865919A IL 26865919 A IL26865919 A IL 26865919A IL 268659 A IL268659 A IL 268659A
Authority
IL
Israel
Prior art keywords
production
methods
polynucleotides encoding
transferrin receptor
receptor polypeptides
Prior art date
Application number
IL26865919A
Other languages
English (en)
Hebrew (he)
Original Assignee
Denali Therapeutics Inc
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 Denali Therapeutics Inc filed Critical Denali Therapeutics Inc
Publication of IL268659A publication Critical patent/IL268659A/en

Links

Classifications

    • 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
    • C07K14/70582—CD71
    • 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
    • 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
    • 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
    • C07K14/79—Transferrins, e.g. lactoferrins, ovotransferrins
    • 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
    • G01N33/5082—Supracellular entities, e.g. tissue, organisms
    • G01N33/5088—Supracellular entities, e.g. tissue, organisms of vertebrates
    • 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
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/5308—Immunoassay; Biospecific binding assay; Materials therefor for analytes not provided for elsewhere, e.g. nucleic acids, uric acid, worms, mites
    • 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
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • 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
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2015/8527—Vectors 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
    • 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
    • G01N2500/10—Screening for compounds of potential therapeutic value involving cells

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Cell Biology (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Environmental Sciences (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Veterinary Medicine (AREA)
  • Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Food Science & Technology (AREA)
  • General Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Animal Husbandry (AREA)
  • 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)
IL26865919A 2017-02-17 2019-08-12 Polynucleotides encoding chimeric transferrin receptor polypeptides, host cells and animals comprising same, methods of producing same and uses thereof IL268659A (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762460692P 2017-02-17 2017-02-17
US201762543559P 2017-08-10 2017-08-10
US201762543658P 2017-08-10 2017-08-10
US201762583314P 2017-11-08 2017-11-08
PCT/US2018/018302 WO2018152285A1 (en) 2017-02-17 2018-02-15 Transferrin receptor transgenic models

Publications (1)

Publication Number Publication Date
IL268659A true IL268659A (en) 2019-10-31

Family

ID=61628463

Family Applications (1)

Application Number Title Priority Date Filing Date
IL26865919A IL268659A (en) 2017-02-17 2019-08-12 Polynucleotides encoding chimeric transferrin receptor polypeptides, host cells and animals comprising same, methods of producing same and uses thereof

Country Status (9)

Country Link
EP (2) EP4628586A3 (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)
SG (1) SG11201907419PA (enExample)
WO (1) WO2018152285A1 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020002918A (es) 2017-10-02 2020-07-22 Denali Therapeutics Inc Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas.
EP3807322A1 (en) 2018-06-18 2021-04-21 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
AU2021362209B2 (en) 2020-10-14 2026-02-12 Denali Therapeutics Inc. Fusion proteins comprising sulfoglucosamine sulfohydrolase enzymes and methods thereof
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
WO2023284850A1 (en) * 2021-07-16 2023-01-19 Biocytogen Pharmaceuticals (Beijing) Co., Ltd. Genetically modified non-human animal with human or chimeric tfr1
TW202340235A (zh) 2021-12-17 2023-10-16 美商戴納立製藥公司 多肽工程、文庫及工程ｃｄ９８重鏈以及轉鐵蛋白受體結合多肽
EP4448746A1 (en) 2021-12-17 2024-10-23 Denali Therapeutics Inc. Fusion proteins comprising alpha-l-iduronidase enzymes and methods
WO2023192288A1 (en) 2022-03-28 2023-10-05 Denali Therapeutics Inc. Monovalent anti-trem2 binding molecules and methods of use thereof
IL317261A (en) * 2022-07-29 2025-01-01 Regeneron Pharma Non-human animals containing a modified transferrin receptor locus
WO2024160736A1 (en) 2023-01-30 2024-08-08 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
TW202542190A (zh) 2023-12-20 2025-11-01 美商戴納立製藥公司 包含酸性α—葡萄糖苷酶之融合蛋白及其方法
WO2026006173A1 (en) 2024-06-24 2026-01-02 Denali Therapeutics Inc. Fusion proteins comprising modified glucocerebrosidase polypeptides and methods thereof

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US5898094A (en) 1996-10-21 1999-04-27 University Of South Florida Transgenic mice expressing APPK670N,M671L and a mutant presenilin transgenes
US6175057B1 (en) 1997-10-08 2001-01-16 The Regents Of The University Of California Transgenic mouse model of alzheimer's disease and cerebral amyloid angiopathy
WO2004094647A2 (en) * 2003-04-18 2004-11-04 Cytovia, Inc. Methods of treating diseases responsive to induction of apoptosis and screening assays
JP2011518555A (ja) 2008-04-14 2011-06-30 サンガモ バイオサイエンシーズ， インコーポレイテッド 標的組込みのための線形ドナーコンストラクト
CN102638971B (zh) 2009-07-08 2015-10-07 科马布有限公司 动物模型及治疗分子
JP2013518602A (ja) 2010-02-09 2013-05-23 サンガモ バイオサイエンシーズ， インコーポレイテッド 部分的に一本鎖のドナー分子による標的化ゲノム改変
US8476485B2 (en) 2010-06-24 2013-07-02 Academia Sinica Non-human animal model for amyotrophic lateral sclerosis (ALS) with loss-of-TDP-43 function
US9232775B2 (en) 2012-08-03 2016-01-12 Industry-Academic Cooperation Foundation, Yonsei University Genetically engineered mouse model for autism spectrum disorder having deletion of Shank2 gene and use thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2971007A4 (en) 2013-03-13 2017-03-22 Australian Nuclear Science And Technology Organisation Transgenic non-human organisms with non-functional tspo genes
EA038367B1 (ru) 2013-05-20 2021-08-16 Дженентек, Инк. АНТИТЕЛА ПРОТИВ РЕЦЕПТОРА ТРАНСФЕРРИНА (TfR) И СПОСОБЫ ИХ ИСПОЛЬЗОВАНИЯ
ES2844174T3 (es) 2013-09-18 2021-07-21 Kymab Ltd Métodos, células y organismos
CN103897033A (zh) * 2014-03-31 2014-07-02 中国药科大学 转铁蛋白受体TfR特异性结合肽及其应用
JP6779876B2 (ja) 2014-11-19 2020-11-04 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その使用方法
JP7021955B2 (ja) * 2015-06-24 2022-03-03 エフ・ホフマン－ラ・ロシュ・アクチェンゲゼルシャフト Ｈｅｒ２及び血液脳関門受容体に特異的な三重特異性抗体及び使用方法
CN110506054A (zh) * 2017-02-17 2019-11-26 戴纳立制药公司 经工程改造的多肽

Also Published As

Publication number Publication date
AU2018221704B2 (en) 2021-08-19
EP4628586A3 (en) 2026-01-14
EP3583118C0 (en) 2025-06-11
EP3583118B1 (en) 2025-06-11
CN110382526B (zh) 2023-12-26
CA3053370A1 (en) 2018-08-23
KR102637590B1 (ko) 2024-02-15
EP3583118A1 (en) 2019-12-25
JP2020510418A (ja) 2020-04-09
JP7669113B2 (ja) 2025-04-28
AU2018221704A1 (en) 2019-09-19
JP2025087857A (ja) 2025-06-10
EP4628586A2 (en) 2025-10-08
JP2023116513A (ja) 2023-08-22
SG11201907419PA (en) 2019-09-27
KR20190135993A (ko) 2019-12-09
WO2018152285A1 (en) 2018-08-23
CN110382526A (zh) 2019-10-25

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