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

    • 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

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) 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)

* Cited by examiner, † Cited by third party
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

Family Cites Families (28)

* 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
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. 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
EP2386316B1 (en) * 2004-06-10 2018-02-28 Omeros Corporation Methods for treating conditions associated with MASP-2 dependent complement activation
AU2005295269B2 (en) 2004-10-19 2010-05-13 Regeneron Pharmaceuticals, Inc. Method for generating an animal homozygous for a genetic modification
EP2357254A1 (en) * 2005-02-14 2011-08-17 University of Iowa Research Foundation Methods and reagents for treatment and diagnosis 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
WO2009126161A1 (en) 2008-04-11 2009-10-15 Utc Fuel Cells, Llc Fuel cell and bipolar plate having manifold sump
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
GB0922659D0 (en) * 2009-12-24 2010-02-10 Univ Edinburgh Factor H
IN2014DN09261A (https=) 2012-04-25 2015-07-10 Regeneron Pharma
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California 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
EP3363902B1 (en) 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
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.
SI2986729T1 (sl) 2013-04-16 2019-02-28 Regeneron Pharmaceuticals, Inc. Ciljana sprememba genoma podgane
US10183988B2 (en) * 2013-06-07 2019-01-22 Duke University Anti-Complement factor H antibodies
US9546384B2 (en) 2013-12-11 2017-01-17 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a mouse genome
ES2731437T3 (es) 2014-11-21 2019-11-15 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida mediante el uso de pares de ARN guías
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
CN118184777A (zh) * 2017-01-30 2024-06-14 亚力兄制药公司 单价抗备解素抗体及抗体片段
US10765762B2 (en) * 2017-02-27 2020-09-08 Regeneron Pharmaceuticals, Inc. Humanized model of kidney and liver disorders

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
AU2019339404A1 (en) 2021-04-22
JP2022500047A (ja) 2022-01-04
ES2974200T3 (es) 2024-06-26
CA3112612C (en) 2024-02-27
WO2020056122A1 (en) 2020-03-19
AU2019339404B2 (en) 2022-11-03
EP3849304B1 (en) 2024-01-10
CA3112612A1 (en) 2020-03-19
KR20210058889A (ko) 2021-05-24
US20220053741A1 (en) 2022-02-24
CN112969367A (zh) 2021-06-15
EP3849304C0 (en) 2024-01-10
JP7222075B2 (ja) 2023-02-14
EP3849304A1 (en) 2021-07-21
CN112969367B (zh) 2023-04-07

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
CN1423696A (zh) 人类精神分裂症基因
KR101621273B1 (ko) 카텝신 c의 용도
KR20220025806A (ko) 핵산의 무작위 구성 표적화 통합
KR102545031B1 (ko) C3 사구체병증 모델로서의 보체 인자 h 유전자 녹아웃 래트
CN112771162B (zh) 用剪接转换寡核苷酸靶向kit诱导肥大细胞凋亡
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

Patent event date: 20210409

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20220817

Comment text: Request for Examination of Application

PA0302 Request for accelerated examination

Patent event date: 20220817

Patent event code: PA03022R01D

Comment text: Request for Accelerated Examination

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

Comment text: Notification of reason for refusal

Patent event date: 20230130

Patent event code: PE09021S01D

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

Patent event code: PE07011S01D

Comment text: Decision to Grant Registration

Patent event date: 20230516

GRNT Written decision to grant
PR0701 Registration of establishment

Comment text: Registration of Establishment

Patent event date: 20230614

Patent event code: PR07011E01D

PR1002 Payment of registration fee

Payment date: 20230615

End annual number: 3

Start annual number: 1

PG1601 Publication of registration