KR102545031B1 - C3 사구체병증 모델로서의 보체 인자 h 유전자 녹아웃 래트 - Google Patents

C3 사구체병증 모델로서의 보체 인자 h 유전자 녹아웃 래트 Download PDF

Info

Publication number
KR102545031B1
KR102545031B1 KR1020217010579A KR20217010579A KR102545031B1 KR 102545031 B1 KR102545031 B1 KR 102545031B1 KR 1020217010579 A KR1020217010579 A KR 1020217010579A KR 20217010579 A KR20217010579 A KR 20217010579A KR 102545031 B1 KR102545031 B1 KR 102545031B1
Authority
KR
South Korea
Prior art keywords
genetically modified
rat
rats
weeks
delete delete
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
KR1020217010579A
Other languages
English (en)
Korean (ko)
Other versions
KR20210058889A (ko
Inventor
키쇼어 데발라라자-나라쉬마
제프리 디. 리
로리 모톤
Original Assignee
리제너론 파마슈티칼스 인코포레이티드
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 리제너론 파마슈티칼스 인코포레이티드 filed Critical 리제너론 파마슈티칼스 인코포레이티드
Publication of KR20210058889A publication Critical patent/KR20210058889A/ko
Application granted granted Critical
Publication of KR102545031B1 publication Critical patent/KR102545031B1/ko
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)
KR1020217010579A 2018-09-13 2019-09-12 C3 사구체병증 모델로서의 보체 인자 h 유전자 녹아웃 래트 Active KR102545031B1 (ko)

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
KR20210058889A KR20210058889A (ko) 2021-05-24
KR102545031B1 true KR102545031B1 (ko) 2023-06-20

Family

ID=68052023

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217010579A Active KR102545031B1 (ko) 2018-09-13 2019-09-12 C3 사구체병증 모델로서의 보체 인자 h 유전자 녹아웃 래트

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
Kidney International vol.82, pp.961-968(2012.07.25.)*
Kidney International vol.89, pp.823-832(2016.02.03.)*
Nat. Genet. 31(4):424-428(2002)

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
AU2019339404B2 (en) 2022-11-03
CN112969367A (zh) 2021-06-15
CA3112612C (en) 2024-02-27

Similar Documents

Publication Publication Date Title
AU2021261909B2 (en) Antisense oligomers for treatment of conditions and diseases
KR102768381B1 (ko) Lrrk2 발현 감소용 화합물 및 방법
AU2015286663B2 (en) Antisense oligonucleotides for the treatment of usher syndrome type 2
KR101999410B1 (ko) 염색체 랜딩 패드 및 관련된 용도
CN101142321B (zh) 治疗眼病的方法和组合物
KR20210138587A (ko) 개선된 면역요법을 위한 조합 유전자 표적
AU2019403015B2 (en) Nuclease-mediated repeat expansion
KR20220113743A (ko) 병태 및 질환의 치료를 위한 안티센스 올리고머
AU2022200784B2 (en) Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception
KR102782761B1 (ko) 인간의 앤젤만 증후군에서 부계 ube3a 유전자 발현을 복원하기 위한 조성물 및 방법
KR20220054401A (ko) 감염의 숙주 rna 바이오마커의 신속한 조기-검출 및 인간의 covid-19 코로나바이러스 감염의 조기 식별을 위한 시스템, 방법 및 조성물
CN1423696A (zh) 人类精神分裂症基因
KR101621273B1 (ko) 카텝신 ｃ의 용도
AU2019339404B2 (en) Complement factor h gene knockout rat as a model of C3 glomerulopathy
CN112771162B (zh) 用剪接转换寡核苷酸靶向kit诱导肥大细胞凋亡
KR20220025806A (ko) 핵산의 무작위 구성 표적화 통합
CN101151371B (zh) 治疗中的逆转录转座子抑制
KR20080096495A (ko) 소과 동물에서 배최장근 최대력, 근내 지방, 식용우소매유통 생산량 및 순 사료 섭취량으로부터 선택된 형질을평가하는 방법
CN108135151A (zh) 前列腺癌的啮齿动物模型
US20160184454A1 (en) Rab7l1 interacts with lrrk2 to modify intraneuronal protein sorting and parkinson's disease risk
HK1114882B (en) Retrotransposon inhibition in therapy

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

PA0302 Request for accelerated examination

St.27 status event code: A-1-2-D10-D17-exm-PA0302

St.27 status event code: A-1-2-D10-D16-exm-PA0302

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701

GRNT Written decision to grant
PR0701 Registration of establishment

St.27 status event code: A-2-4-F10-F11-exm-PR0701

PR1002 Payment of registration fee

St.27 status event code: A-2-2-U10-U12-oth-PR1002

Fee payment year number: 1

PG1601 Publication of registration

St.27 status event code: A-4-4-Q10-Q13-nap-PG1601

PR1001 Payment of annual fee

St.27 status event code: A-4-4-U10-U11-oth-PR1001

Fee payment year number: 4

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-OTH-PR1001 (AS PROVIDED BY THE NATIONAL OFFICE)

Year of fee payment: 4