BR112019001783A2 - mamífero não humano, e, métodos para produzir o mamífero não humano e de triagem de um composto. - Google Patents

mamífero não humano, e, métodos para produzir o mamífero não humano e de triagem de um composto.

Info

Publication number
BR112019001783A2
BR112019001783A2 BR112019001783-6A BR112019001783A BR112019001783A2 BR 112019001783 A2 BR112019001783 A2 BR 112019001783A2 BR 112019001783 A BR112019001783 A BR 112019001783A BR 112019001783 A2 BR112019001783 A2 BR 112019001783A2
Authority
BR
Brazil
Prior art keywords
npscl
nonhuman mammal
methods
screening
compound
Prior art date
Application number
BR112019001783-6A
Other languages
English (en)
Inventor
Hunt Charleen
Mastaitis Jason
Gong Guochun
Venus Lai Ka-Man
Gromada Jesper
N. Economides Aris
Original Assignee
Regeneron Pharmaceuticals, 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 Regeneron Pharmaceuticals, Inc. filed Critical Regeneron Pharmaceuticals, Inc.
Publication of BR112019001783A2 publication Critical patent/BR112019001783A2/pt

Links

Classifications

    • 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
    • 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
    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • 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
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/054—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
    • A01K2217/056—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function due to mutation of coding region of the transgene (dominant negative)
    • 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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Environmental Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Endocrinology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Pathology (AREA)
  • Urology & Nephrology (AREA)
  • Rheumatology (AREA)
  • Public Health (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

são fornecidos animais não humanos que compreendem uma mutação no gene fbn1 para modelar a síndrome progeroide neonatal com lipodistrofia congênita (npscl). também são fornecidos métodos para fazer tais modelos animais não humanos. os modelos animais não humanos podem ser utilizados para rastrear compostos quanto a atividade na inibição ou redução de npscl ou na melhoria de sintomas semelhantes a npscl ou para rastrear compostos quanto a atividade potencialmente prejudicial na promoção ou exacerbação de npscl, bem como para fornecer percepções sobre o mecanismo de npscl e alvos terapêuticos e de diagnósticos potencialmente novos.
BR112019001783-6A 2016-07-29 2017-07-28 mamífero não humano, e, métodos para produzir o mamífero não humano e de triagem de um composto. BR112019001783A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662368924P 2016-07-29 2016-07-29
US62/368924 2016-07-29
PCT/US2017/044409 WO2018023014A1 (en) 2016-07-29 2017-07-28 Mice comprising mutations resulting in expression of c-truncated fibrillin-1

Publications (1)

Publication Number Publication Date
BR112019001783A2 true BR112019001783A2 (pt) 2019-05-07

Family

ID=59656182

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019001783-6A BR112019001783A2 (pt) 2016-07-29 2017-07-28 mamífero não humano, e, métodos para produzir o mamífero não humano e de triagem de um composto.

Country Status (14)

Country Link
US (2) US10548302B2 (pt)
EP (1) EP3490373B1 (pt)
JP (1) JP2019523009A (pt)
KR (1) KR20190041476A (pt)
CN (1) CN109803530A (pt)
AU (1) AU2017302657A1 (pt)
BR (1) BR112019001783A2 (pt)
CA (1) CA3031206A1 (pt)
ES (1) ES2965134T3 (pt)
IL (1) IL264434A (pt)
MX (1) MX2019001211A (pt)
RU (1) RU2721125C1 (pt)
SG (1) SG11201901305YA (pt)
WO (1) WO2018023014A1 (pt)

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Also Published As

Publication number Publication date
EP3490373C0 (en) 2023-10-25
EP3490373B1 (en) 2023-10-25
US20180027782A1 (en) 2018-02-01
US10548302B2 (en) 2020-02-04
JP2019523009A (ja) 2019-08-22
ES2965134T3 (es) 2024-04-11
KR20190041476A (ko) 2019-04-22
EP3490373A1 (en) 2019-06-05
CA3031206A1 (en) 2018-02-01
CN109803530A (zh) 2019-05-24
MX2019001211A (es) 2019-09-16
WO2018023014A1 (en) 2018-02-01
SG11201901305YA (en) 2019-03-28
US11730150B2 (en) 2023-08-22
RU2721125C1 (ru) 2020-05-18
AU2017302657A1 (en) 2019-02-14
US20200107527A1 (en) 2020-04-09
IL264434A (en) 2019-02-28

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