MX2020005732A - Conejo de inactivacion de genes del factor viii o factor ix, metodo para preparar el mismo y utilizarlo. - Google Patents

Conejo de inactivacion de genes del factor viii o factor ix, metodo para preparar el mismo y utilizarlo.

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Publication number
MX2020005732A
MX2020005732A MX2020005732A MX2020005732A MX2020005732A MX 2020005732 A MX2020005732 A MX 2020005732A MX 2020005732 A MX2020005732 A MX 2020005732A MX 2020005732 A MX2020005732 A MX 2020005732A MX 2020005732 A MX2020005732 A MX 2020005732A
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MX
Mexico
Prior art keywords
factor
factor viii
rabbit
gene knockout
preparing same
Prior art date
Application number
MX2020005732A
Other languages
English (en)
Inventor
Min Jung Kim
Myung Eun Jung
So Ra Kim
Seung Hyun Jo
Sung Ho Hwang
Hee Chun Kwak
Su Min Lee
Hyun Ja Nam
Original Assignee
Green Cross Corp
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 Green Cross Corp filed Critical Green Cross Corp
Publication of MX2020005732A publication Critical patent/MX2020005732A/es

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    • 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
    • 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
    • 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
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1025—Acyltransferases (2.3)
    • C12N9/104—Aminoacyltransferases (2.3.2)
    • C12N9/1044—Protein-glutamine gamma-glutamyltransferase (2.3.2.13), i.e. transglutaminase or factor XIII
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • 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
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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
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    • A01K2217/00—Genetically modified animals
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/203—Animal model comprising inducible/conditional expression system, e.g. hormones, tet
    • 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
    • A01K2227/107—Rabbit
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La presente invención se refiere a un conejo de inactivación del gen del factor VIII o del factor IX, a un método para prepararlo y a un uso del mismo y, más particularmente, a un conejo transgénico cuyo gen del factor VIII o del factor IX ha sido inactivado a través del sistema CRISPR/Cas9, un método para preparar el mismo y un uso del mismo. De acuerdo con la presente invención, en el conejo transgénico, cuyo gen de factor VIII y/o factor IX ha sido desactivado, las funciones del factor VIII y/o factor IX, que son proteínas que realizan funciones críticas para el desarrollo de la hemofilia, son inhibidas, de modo que el conejo transgénico es útil para el desarrollo de tratamientos de hemofilia.
MX2020005732A 2017-09-28 2018-09-20 Conejo de inactivacion de genes del factor viii o factor ix, metodo para preparar el mismo y utilizarlo. MX2020005732A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
KR20170126068 2017-09-28
PCT/KR2018/011118 WO2019066378A1 (ko) 2017-09-28 2018-09-20 Factor viii 또는 factor ix 유전자가 녹아웃된 토끼, 이의 제조방법 및 그 용도

Publications (1)

Publication Number Publication Date
MX2020005732A true MX2020005732A (es) 2021-01-15

Family

ID=65901732

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020005732A MX2020005732A (es) 2017-09-28 2018-09-20 Conejo de inactivacion de genes del factor viii o factor ix, metodo para preparar el mismo y utilizarlo.

Country Status (11)

Country Link
US (1) US20210161111A1 (es)
EP (1) EP3690053A4 (es)
JP (2) JP7091450B2 (es)
KR (1) KR102268072B1 (es)
CN (1) CN111492060A (es)
AU (2) AU2018341437B2 (es)
BR (1) BR112020006311A2 (es)
CA (1) CA3077160A1 (es)
EA (1) EA202090635A1 (es)
MX (1) MX2020005732A (es)
WO (1) WO2019066378A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021083073A1 (zh) * 2019-10-31 2021-05-06 华东师范大学 一种基于肝细胞Alb基因的疾病治疗的产品
WO2022147759A1 (en) * 2021-01-08 2022-07-14 Susheng Biotech (Hainan) Co., Ltd. Grna molecule targeting intron i or intron ii of hbb gene, synthetic method thereof, and method to correct types of hbb gene mutations
CN115943928B (zh) * 2022-12-28 2023-09-26 广州医药研究总院有限公司 可遗传的甲型血友病模型犬的建立方法

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3288594B1 (en) * 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
MX2018005332A (es) * 2015-11-06 2018-11-09 Crispr Therapeutics Ag Materiales y metodos para tratamiento de la enfermedad de almacenamiento de glucogeno tipo 1a.
WO2017112895A1 (en) * 2015-12-23 2017-06-29 Haplomics, Inc. F8 gene repair
CN105567735A (zh) * 2016-01-05 2016-05-11 华东师范大学 一种凝血因子基因突变的定点修复载体系统及方法
CA3013179A1 (en) * 2016-01-30 2017-08-03 Bonac Corporation Artificial single guide rna and use thereof
EP3416689B1 (en) * 2016-02-18 2023-01-18 CRISPR Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome

Also Published As

Publication number Publication date
CA3077160A1 (en) 2019-04-04
KR20200038538A (ko) 2020-04-13
CN111492060A (zh) 2020-08-04
AU2021250834A1 (en) 2021-11-04
AU2018341437A1 (en) 2020-05-07
BR112020006311A2 (pt) 2021-01-12
US20210161111A1 (en) 2021-06-03
JP2022000055A (ja) 2022-01-04
EA202090635A1 (ru) 2020-07-30
EP3690053A1 (en) 2020-08-05
AU2018341437B2 (en) 2022-04-21
JP2020536522A (ja) 2020-12-17
EP3690053A4 (en) 2021-10-06
KR102268072B1 (ko) 2021-06-23
JP7091450B2 (ja) 2022-06-27
WO2019066378A1 (ko) 2019-04-04
JP7199492B2 (ja) 2023-01-05

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