AU2019339404B2 - Complement factor h gene knockout rat as a model of C3 glomerulopathy - Google Patents

Complement factor h gene knockout rat as a model of C3 glomerulopathy Download PDF

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Publication number
AU2019339404B2
AU2019339404B2 AU2019339404A AU2019339404A AU2019339404B2 AU 2019339404 B2 AU2019339404 B2 AU 2019339404B2 AU 2019339404 A AU2019339404 A AU 2019339404A AU 2019339404 A AU2019339404 A AU 2019339404A AU 2019339404 B2 AU2019339404 B2 AU 2019339404B2
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Prior art keywords
rat
genetically modified
weeks
locus
modified rat
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AU2019339404A1 (en
Inventor
Kishor DEVALARAJA-NARASHIMHA
Jeffrey D. Lee
Lori MORTON
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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
    • A01K67/0276—Knock-out 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
    • 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
    • A01K2267/035—Animal model for multifactorial diseases
    • 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
    • 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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  • Life Sciences & Earth Sciences (AREA)
  • Environmental Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2019339404A 2018-09-13 2019-09-12 Complement factor h gene knockout rat as a model of C3 glomerulopathy Active AU2019339404B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862730690P 2018-09-13 2018-09-13
US62/730,690 2018-09-13
PCT/US2019/050792 WO2020056122A1 (en) 2018-09-13 2019-09-12 Complement factor h gene knockout rat as a model of c3 glomerulopathy

Publications (2)

Publication Number Publication Date
AU2019339404A1 AU2019339404A1 (en) 2021-04-22
AU2019339404B2 true AU2019339404B2 (en) 2022-11-03

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019339404A Active AU2019339404B2 (en) 2018-09-13 2019-09-12 Complement factor h gene knockout rat as a model of C3 glomerulopathy

Country Status (9)

Country Link
US (1) US12653168B2 (enExample)
EP (1) EP3849304B1 (enExample)
JP (1) JP7222075B2 (enExample)
KR (1) KR102545031B1 (enExample)
CN (1) CN112969367B (enExample)
AU (1) AU2019339404B2 (enExample)
CA (1) CA3112612C (enExample)
ES (1) ES2974200T3 (enExample)
WO (1) WO2020056122A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12653168B2 (en) 2018-09-13 2026-06-16 Regeneron Pharmaceuticals, Inc. Complement factor H gene knockout rat as a model of C3 glomerulopathy
GB202010009D0 (en) * 2020-06-30 2020-08-12 Syncona Investment Man Ltd Vector
CN114634932B (zh) * 2022-03-11 2023-09-05 安徽医科大学 一种新circRNA、试剂盒及用途

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5523226A (en) 1993-05-14 1996-06-04 Biotechnology Research And Development Corp. Transgenic swine compositions and methods
WO1999005266A2 (en) 1997-07-26 1999-02-04 Wisconsin Alumni Research Foundation Trans-species nuclear transfer
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
US6586251B2 (en) 2000-10-31 2003-07-01 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
AUPR451401A0 (en) 2001-04-20 2001-05-24 Monash University A method of nuclear transfer
US7612250B2 (en) 2002-07-29 2009-11-03 Trustees Of Tufts College Nuclear transfer embryo formation method
EP2465534B1 (en) * 2004-06-10 2017-03-29 Omeros Corporation Methods for treating conditions associated with MASP-2 dependent complement activation
EP1802193B1 (en) 2004-10-19 2014-04-30 Regeneron Pharmaceuticals, Inc. Method for generating a mouse homozygous for a genetic modification
US7745389B2 (en) 2005-02-14 2010-06-29 University Of Iowa Research Foundation Methods for treatment of age-related macular degeneration
CN101117633B (zh) 2006-08-03 2011-07-20 上海交通大学附属儿童医院 一种细胞核移植方法
KR20100120226A (ko) 2008-04-11 2010-11-12 유티씨 파워 코포레이션 매니폴드 섬프를 갖는 바이폴라 플레이트 및 연료 전지
US8476032B2 (en) * 2008-08-27 2013-07-02 Children's Hospital & Research Center Oakland Complement factor H-based assays for serum bactericidal activity against Neisseria meningitidis
US20120159653A1 (en) * 2008-12-04 2012-06-21 Sigma-Aldrich Co. Genomic editing of genes involved in macular degeneration
CN102858985A (zh) * 2009-07-24 2013-01-02 西格马-奥尔德里奇有限责任公司 基因组编辑方法
GB0922659D0 (en) 2009-12-24 2010-02-10 Univ Edinburgh Factor H
ES2683071T3 (es) 2012-04-25 2018-09-24 Regeneron Pharmaceuticals, Inc. Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
WO2014033644A2 (en) 2012-08-28 2014-03-06 Novartis Ag Methods of nuclease-based genetic engineering
DK3363902T3 (da) 2012-12-06 2020-01-02 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
GB201301632D0 (en) * 2013-01-30 2013-03-13 Imp Innovations Complement System
MX2015010841A (es) 2013-02-20 2016-05-09 Regeneron Pharma Modificacion genetica de ratas.
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
EP3003372B1 (en) * 2013-06-07 2019-10-09 Duke University Inhibitors of complement factor h
HRP20231121T1 (hr) 2013-09-23 2023-12-22 Regeneron Pharmaceuticals, Inc. Životinje koje nisu ljudi, koje imaju gen za humanizirani protein koji regulira signale
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
MX2016014504A (es) 2014-05-05 2017-05-23 Regeneron Pharma Animales c5 y c3 humanizados.
US9738897B2 (en) 2014-06-23 2017-08-22 Regeneron Pharmaceuticals, Inc. Nuclease-mediated DNA assembly
DK3161128T3 (en) 2014-06-26 2018-11-05 Regeneron Pharma METHODS AND COMPOSITIONS FOR TARGETED GENTICAL MODIFICATIONS AND PROCEDURES FOR USE THEREOF
US20160081314A1 (en) 2014-09-19 2016-03-24 Regeneron Pharmaceuticals, Inc. Chimeric Antigen Receptors
EP3221457B1 (en) 2014-11-21 2019-03-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
CN108366548B (zh) 2015-11-20 2021-10-08 瑞泽恩制药公司 具有人源化淋巴细胞活化基因3的非人动物
WO2017114401A1 (zh) * 2015-12-31 2017-07-06 江苏匡亚生物医药科技有限公司 具有补体调节活性的重组补体因子h-免疫球蛋白融合蛋白及其制备方法与应用
CN106519030B (zh) * 2016-12-22 2020-01-07 广州泰诺迪生物科技有限公司 补体因子h抑制剂及与之相关的用途
KR20260069620A (ko) * 2017-01-30 2026-05-15 알렉시온 파마슈티칼스, 인코포레이티드 1가 항-프로페르딘 항체 및 항체 단편
WO2018157027A1 (en) * 2017-02-27 2018-08-30 Regeneron Pharmaceuticals, Inc. Humanized model of kidney and liver disorders
US12653168B2 (en) 2018-09-13 2026-06-16 Regeneron Pharmaceuticals, Inc. Complement factor H gene knockout rat as a model of C3 glomerulopathy

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
LINDA J. MULLINS ET AL: "Renal disease pathophysiology and treatment: contributions from the rat", DISEASE MODELS & MECHANISMS, 2016, vol. 9, pages 1419 - 1433 *
ORTIZ ALBERTO ET AL: "Translational value of animal models of kidney failure", EUROPEAN JOURNAL OF PHARMACOLOGY, 2015, vol. 759, pages 205 - 220, *
THOMAS D. BARBOUR ET AL: "Complement receptor 3 mediates renal protection in experimental C3 glomerulopathy", KIDNEY INTERNATIONAL, 2016, vol. 89, pages 823 - 832 *

Also Published As

Publication number Publication date
EP3849304B1 (en) 2024-01-10
EP3849304A1 (en) 2021-07-21
US12653168B2 (en) 2026-06-16
EP3849304C0 (en) 2024-01-10
KR20210058889A (ko) 2021-05-24
AU2019339404A1 (en) 2021-04-22
CA3112612A1 (en) 2020-03-19
US20220053741A1 (en) 2022-02-24
ES2974200T3 (es) 2024-06-26
CN112969367B (zh) 2023-04-07
JP7222075B2 (ja) 2023-02-14
WO2020056122A1 (en) 2020-03-19
JP2022500047A (ja) 2022-01-04
KR102545031B1 (ko) 2023-06-20
CN112969367A (zh) 2021-06-15
CA3112612C (en) 2024-02-27

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