BR112021022722A2 - Animal não humano, célula de animal não humana, genoma de animal não humano, gene de albumina animal não humana humanizada, vetor de alvejamento, método de avaliação da atividade de um reagente, e, método de otimização da atividade de um reagente - Google Patents
Animal não humano, célula de animal não humana, genoma de animal não humano, gene de albumina animal não humana humanizada, vetor de alvejamento, método de avaliação da atividade de um reagente, e, método de otimização da atividade de um reagenteInfo
- Publication number
- BR112021022722A2 BR112021022722A2 BR112021022722A BR112021022722A BR112021022722A2 BR 112021022722 A2 BR112021022722 A2 BR 112021022722A2 BR 112021022722 A BR112021022722 A BR 112021022722A BR 112021022722 A BR112021022722 A BR 112021022722A BR 112021022722 A2 BR112021022722 A2 BR 112021022722A2
- Authority
- BR
- Brazil
- Prior art keywords
- human animal
- human
- reagent
- activity
- albumin
- Prior art date
Links
- 108010088751 Albumins Proteins 0.000 title abstract 6
- 238000000034 method Methods 0.000 title abstract 6
- 239000003153 chemical reaction reagent Substances 0.000 title abstract 5
- 230000008685 targeting Effects 0.000 title abstract 3
- 210000004102 animal cell Anatomy 0.000 title abstract 2
- 241001465754 Metazoa Species 0.000 title 1
- 102000009027 Albumins Human genes 0.000 abstract 4
- 102000008100 Human Serum Albumin Human genes 0.000 abstract 3
- 108091006905 Human Serum Albumin Proteins 0.000 abstract 3
- 210000004027 cell Anatomy 0.000 abstract 3
- 101000693913 Homo sapiens Albumin Proteins 0.000 abstract 1
- 101710163270 Nuclease Proteins 0.000 abstract 1
- 239000003795 chemical substances by application Substances 0.000 abstract 1
- 239000012634 fragment Substances 0.000 abstract 1
- 102000044814 human ALB Human genes 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
Classifications
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- 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
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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
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- 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
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- 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
- C07K14/76—Albumins
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- 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
- C07K14/76—Albumins
- C07K14/765—Serum albumin, e.g. HSA
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- 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
- C12N15/86—Viral vectors
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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
- A01K2207/00—Modified animals
- A01K2207/15—Humanized animals
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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
- 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
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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
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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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
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
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- 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
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- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Environmental Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Animal Behavior & Ethology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biodiversity & Conservation Biology (AREA)
- Animal Husbandry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Pathology (AREA)
- Public Health (AREA)
- Rheumatology (AREA)
- Epidemiology (AREA)
- Urology & Nephrology (AREA)
- Endocrinology (AREA)
- Diabetes (AREA)
- Virology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
Abstract
animal não humano, célula de animal não humana, genoma de animal não humano, gene de albumina animal não humana humanizada, vetor de alvejamento, método de avaliação da atividade de um reagente, e, método de otimização da atividade de um reagente. são fornecidos genomas de animais não humanos, células de animais não humanos, e animais não humanos que compreendem um locus de albumina (alb) humanizada, e métodos para fabricação e uso de tais genomas de animais não humanos, células de animais não humanos, e animais não humanos. células de animais não humanos, ou animais não humanos, que compreendem um locus de albumina humanizada expressam uma proteína albumina humana ou uma proteína albumina quimérica, fragmentos das quais são de albumina humana. métodos são fornecidos para o uso de tais animais não humanos que compreendem um locus de albumina humanizada para avaliar a eficácia in vivo de reagentes de alvejamento de albumina humana, tais como agentes de nuclease concebidos para alvejar a albumina humana.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962858589P | 2019-06-07 | 2019-06-07 | |
US201962916666P | 2019-10-17 | 2019-10-17 | |
PCT/US2020/036412 WO2020247812A1 (en) | 2019-06-07 | 2020-06-05 | Non-human animals comprising a humanized albumin locus |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112021022722A2 true BR112021022722A2 (pt) | 2022-01-04 |
Family
ID=71899847
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112021022722A BR112021022722A2 (pt) | 2019-06-07 | 2020-06-05 | Animal não humano, célula de animal não humana, genoma de animal não humano, gene de albumina animal não humana humanizada, vetor de alvejamento, método de avaliação da atividade de um reagente, e, método de otimização da atividade de um reagente |
Country Status (13)
Country | Link |
---|---|
US (2) | US11622547B2 (pt) |
EP (1) | EP3796776A1 (pt) |
JP (1) | JP2022534560A (pt) |
KR (1) | KR20220017939A (pt) |
CN (1) | CN113939595A (pt) |
AU (1) | AU2020289581A1 (pt) |
BR (1) | BR112021022722A2 (pt) |
CA (1) | CA3137764A1 (pt) |
IL (1) | IL288606A (pt) |
MX (1) | MX2021015122A (pt) |
SG (1) | SG11202111256XA (pt) |
TW (1) | TW202112229A (pt) |
WO (1) | WO2020247812A1 (pt) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
IL286905B1 (en) | 2019-04-04 | 2024-02-01 | Regeneron Pharma | Non-human animals containing the human coagulation factor 12 locus |
EP3801011A1 (en) | 2019-06-04 | 2021-04-14 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use |
KR20220017939A (ko) | 2019-06-07 | 2022-02-14 | 리제너론 파마슈티칼스 인코포레이티드 | 인간화 알부민 좌위를 포함하는 비-인간 동물 |
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- 2020-06-05 BR BR112021022722A patent/BR112021022722A2/pt unknown
- 2020-06-05 TW TW109119042A patent/TW202112229A/zh unknown
- 2020-06-05 EP EP20750423.4A patent/EP3796776A1/en active Pending
- 2020-06-05 WO PCT/US2020/036412 patent/WO2020247812A1/en unknown
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KR20220017939A (ko) | 2022-02-14 |
IL288606A (en) | 2022-02-01 |
CA3137764A1 (en) | 2020-12-10 |
JP2022534560A (ja) | 2022-08-02 |
MX2021015122A (es) | 2022-04-06 |
US11622547B2 (en) | 2023-04-11 |
CN113939595A (zh) | 2022-01-14 |
AU2020289581A1 (en) | 2021-11-18 |
SG11202111256XA (en) | 2021-11-29 |
US20200383304A1 (en) | 2020-12-10 |
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