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

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
rat
genetically modified
rats
weeks
age
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2021514036A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022500047A5 (enExample
JP2022500047A (ja
Inventor
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,
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Regeneron Pharmaceuticals Inc
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 JP2022500047A publication Critical patent/JP2022500047A/ja
Publication of JP2022500047A5 publication Critical patent/JP2022500047A5/ja
Application granted granted Critical
Publication of JP7222075B2 publication Critical patent/JP7222075B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • 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

Landscapes

  • 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 (enExample) 2022-09-01
JP7222075B2 true JP7222075B2 (ja) 2023-02-14

Family

ID=68052023

Family Applications (1)

Application Number Title Priority Date Filing Date
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

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

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 (35)

* 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
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
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

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
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
WO2020056122A1 (en) 2020-03-19
JP2022500047A (ja) 2022-01-04
AU2019339404B2 (en) 2022-11-03
KR102545031B1 (ko) 2023-06-20
CN112969367A (zh) 2021-06-15
CA3112612C (en) 2024-02-27

Similar Documents

Publication Publication Date Title
KR102544051B1 (ko) 인간화된 ttr 유전자좌를 포함하는 비인간 동물 및 사용 방법
EP3585158B1 (en) Rodent models of retinoschisis
CN110494168B (zh) 肾病症和肝病症的人源化模型
US11957115B2 (en) Genetically modified mouse expressing human APOE4 and mouse Trem2 p.R47H and methods of use thereof
KR102927708B1 (ko) 베타-슬립 돌연변이를 갖는 인간화 ttr 좌위를 포함하는 비-인간 동물 및 사용 방법
EP3849304B1 (en) Complement factor h gene knockout rat as a model of c3 glomerulopathy
JP7361031B2 (ja) ヒト化trkb遺伝子座を含む非ヒト動物
JP7698587B2 (ja) ヒト化アルブミン遺伝子座を含む非ヒト動物
JP2022528414A (ja) ヒト化凝固因子12遺伝子座を含む非ヒト動物
WO2019246483A1 (en) Genetically modified mouse models of alzheimer's disease
EP4638764A2 (en) Use of rtl8-deficient cells to produce biocompatible, peg10-derived virus-like particles (vlp)
JP5075641B2 (ja) 遺伝子改変動物およびその用途
US20230081547A1 (en) Non-human animals comprising a humanized klkb1 locus and methods of use
HK40048493B (en) Complement factor h gene knockout rat as a model of c3 glomerulopathy
HK40048493A (en) Complement factor h gene knockout rat as a model of c3 glomerulopathy
RU2784927C1 (ru) Отличные от человека животные, включающие в себя гуманизированный ttr локус, и способы применения
JP2025527122A (ja) 改変型トランスフェリン受容体座位を含む非ヒト動物
KR20230078677A (ko) 인간 cr1을 발현하는 설치류 동물

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220824

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220824

A871 Explanation of circumstances concerning accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A871

Effective date: 20220824

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20221012

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221227

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230130

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230202

R150 Certificate of patent or registration of utility model

Ref document number: 7222075

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150