JP7222075B2 - C3糸球体症のモデルとしての補体因子h遺伝子ノックアウトラット - Google Patents

C3糸球体症のモデルとしての補体因子h遺伝子ノックアウトラット Download PDF

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JP7222075B2
JP7222075B2 JP2021514036A JP2021514036A JP7222075B2 JP 7222075 B2 JP7222075 B2 JP 7222075B2 JP 2021514036 A JP2021514036 A JP 2021514036A JP 2021514036 A JP2021514036 A JP 2021514036A JP 7222075 B2 JP7222075 B2 JP 7222075B2
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rat
genetically modified
rats
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age
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JP2022500047A5 (https=
JP2022500047A (ja
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キショー デバララジャ-ナラシマ
ジェフリー ディー. リー,
ローリ モートン,
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals 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
    • A01K67/0276Knock-out 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
    • 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
    • A01K2267/035Animal model for multifactorial diseases
    • 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
    • A01K2267/0393Animal model comprising a reporter system for screening tests
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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

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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)
JP2021514036A 2018-09-13 2019-09-12 C3糸球体症のモデルとしての補体因子h遺伝子ノックアウトラット Active JP7222075B2 (ja)

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 (3)

Publication Number Publication Date
JP2022500047A JP2022500047A (ja) 2022-01-04
JP2022500047A5 JP2022500047A5 (https=) 2022-09-01
JP7222075B2 true JP7222075B2 (ja) 2023-02-14

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JP2021514036A Active JP7222075B2 (ja) 2018-09-13 2019-09-12 C3糸球体症のモデルとしての補体因子h遺伝子ノックアウトラット

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US (1) US20220053741A1 (https=)
EP (1) EP3849304B1 (https=)
JP (1) JP7222075B2 (https=)
KR (1) KR102545031B1 (https=)
CN (1) CN112969367B (https=)
AU (1) AU2019339404B2 (https=)
CA (1) CA3112612C (https=)
ES (1) ES2974200T3 (https=)
WO (1) WO2020056122A1 (https=)

Families Citing this family (2)

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Publication number Priority date Publication date Assignee Title
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008529536A (ja) 2005-02-14 2008-08-07 ユニバーシティー オブ アイオワ リサーチ ファウンデーション 加齢性黄斑変性を処置および診断するための方法および試薬
JP2013515474A (ja) 2009-12-24 2013-05-09 ザ ユニヴァーシティ コート オブ ザ ユニヴァーシティ オブ エディンバラ 組換え体h因子ならびにそのバリアントおよびコンジュゲート

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU8587598A (en) 1997-07-26 1999-02-16 Wisconsin Alumni Research Foundation Trans-species nuclear transfer
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. 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
ES2631127T3 (es) * 2004-06-10 2017-08-28 Omeros Corporation Métodos para tratar afecciones asociadas con la activación del complemento dependiente de MASP-2
ES2463476T3 (es) 2004-10-19 2014-05-28 Regeneron Pharmaceuticals, Inc. Método para generar un ratón homocigótico para una modificación genética
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
JP2011517838A (ja) 2008-04-11 2011-06-16 ユーティーシー パワー コーポレイション マニホルド・サンプを備えたバイポーラプレートおよび燃料電池
WO2010027872A1 (en) * 2008-08-27 2010-03-11 Children's Hospital & Research Center At 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
SG11201406547YA (en) 2012-04-25 2014-11-27 Regeneron Pharma Nuclease-mediated targeting with large targeting vectors
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
WO2014033644A2 (en) 2012-08-28 2014-03-06 Novartis Ag Methods of nuclease-based genetic engineering
PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
GB201301632D0 (en) * 2013-01-30 2013-03-13 Imp Innovations Complement System
MX384291B (es) 2013-02-20 2025-03-14 Regeneron Pharma Modificación genética de ratas.
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
US10183988B2 (en) * 2013-06-07 2019-01-22 Duke University Anti-Complement factor H antibodies
KR102170502B1 (ko) 2013-12-11 2020-10-28 리제너론 파마슈티칼스 인코포레이티드 게놈의 표적화된 변형을 위한 방법 및 조성물
KR102531016B1 (ko) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물
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
MX2019008827A (es) * 2017-01-30 2019-09-26 Alexion Pharma Inc Anticuerpos anti-properdina monovalentes y fragmentos de anticuerpo.
SG10202111663PA (en) * 2017-02-27 2021-12-30 Regeneron Pharma Humanized model of kidney and liver disorders

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008529536A (ja) 2005-02-14 2008-08-07 ユニバーシティー オブ アイオワ リサーチ ファウンデーション 加齢性黄斑変性を処置および診断するための方法および試薬
JP2013515474A (ja) 2009-12-24 2013-05-09 ザ ユニヴァーシティ コート オブ ザ ユニヴァーシティ オブ エディンバラ 組換え体h因子ならびにそのバリアントおよびコンジュゲート

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
J. J. Alexandar, et al.,Kidney International,2012年,Vol.82,p.961-968
L. J. Mullins, et al.,Disease Models & Mechanisms,2016年,Vol.9,p.1419-1433
T. D. Barbour, et al.,Kidney International,2016年,Vol.89,p.823-832

Also Published As

Publication number Publication date
AU2019339404A1 (en) 2021-04-22
CN112969367B (zh) 2023-04-07
AU2019339404B2 (en) 2022-11-03
KR102545031B1 (ko) 2023-06-20
WO2020056122A1 (en) 2020-03-19
EP3849304B1 (en) 2024-01-10
CN112969367A (zh) 2021-06-15
CA3112612C (en) 2024-02-27
EP3849304C0 (en) 2024-01-10
CA3112612A1 (en) 2020-03-19
JP2022500047A (ja) 2022-01-04
ES2974200T3 (es) 2024-06-26
KR20210058889A (ko) 2021-05-24
EP3849304A1 (en) 2021-07-21
US20220053741A1 (en) 2022-02-24

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