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1. (WO2019066378) FACTOR VIII OR FACTOR IX GENE KNOCKOUT RABBIT, METHOD FOR PREPARING SAME AND USE THEREOF
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Pub. No.: WO/2019/066378 International Application No.: PCT/KR2018/011118
Publication Date: 04.04.2019 International Filing Date: 20.09.2018
IPC:
C12N 15/85 (2006.01) ,C12N 15/10 (2006.01) ,C12N 15/113 (2010.01) ,C12N 9/22 (2006.01) ,C12N 9/10 (2006.01) ,C12N 9/64 (2006.01) ,A01K 67/027 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
10
Processes for the isolation, preparation or purification of DNA or RNA
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14
Hydrolases (3.)
16
acting on ester bonds (3.1)
22
Ribonucleases
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
10
Transferases (2.)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14
Hydrolases (3.)
48
acting on peptide bonds, e.g. thromboplastin, leucine aminopeptidase (3.4)
50
Proteinases
64
derived from animal tissue, e.g. rennin
A HUMAN NECESSITIES
01
AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
K
ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
67
Rearing or breeding animals, not otherwise provided for; New breeds of animals
027
New breeds of vertebrates
Applicants:
주식회사 녹십자 GREEN CROSS CORPORATION [KR/KR]; 경기도 용인시 기흥구 이현로30번길 107 107, Ihyeon-ro 30beon-gil, Giheung-gu Yongin-si Gyeonggi-do 16924, KR
재단법인 목암생명과학연구소 MOGAM INSTITUTE FOR BIOMEDICAL RESEARCH [KR/KR]; 경기도 용인시 기흥구 이현로30번길 93 93, Ihyeon-ro 30beon-gil, Giheung-gu Yongin-si Gyeonggi-do 16924, KR
Inventors:
김소라 KIM, So Ra; KR
정명은 JUNG, Myung Eun; KR
김민정 KIM, Min Jung; KR
조승현 JO, Seung Hyun; KR
황성호 HWANG, Sung Ho; KR
곽희천 KWAK, Hee Chun; KR
이수민 LEE, Su Min; KR
남현자 NAM, Hyun Ja; KR
Agent:
이처영 LEE, Cheo Young; KR
장제환 CHANG, Je Hwan; KR
Priority Data:
10-2017-012606828.09.2017KR
Title (EN) FACTOR VIII OR FACTOR IX GENE KNOCKOUT RABBIT, METHOD FOR PREPARING SAME AND USE THEREOF
(FR) LAPIN À INACTIVATION GÉNIQUE DU FACTEUR VIII OU DU FACTEUR IX, PROCÉDÉ POUR SA PRÉPARATION ET UTILISATION CORRESPONDANTE
(KO) FACTOR VIII 또는 FACTOR IX 유전자가 녹아웃된 토끼, 이의 제조방법 및 그 용도
Abstract:
(EN) The present invention relates to a factor VIII or factor IX gene knockout rabbit, a method for preparing the same and a use thereof and, more particularly, to a transgenic rabbit whose factor VIII or factor IX gene has been knocked out through the CRISPR/Cas9 system, a method for preparing the same and a use thereof. According to the present invention, in the transgenic rabbit, whose factor VIII and/or factor IX gene has been knocked out, the functions of factor VIII and/or factor IX, which are proteins that perform critical functions for the development of hemophilia, are inhibited, such that the transgenic rabbit is useful for the development of hemophilia treatments.
(FR) La présente invention concerne un lapin à inactivation génique du facteur VIII ou du facteur IX, un procédé pour sa préparation et utilisation correspondante et, plus particulièrement, un lapin transgénique dont le gène du facteur VIII ou du facteur IX a été inactivé par l'intermédiaire du système CRISPR/Cas9, un procédé pour sa préparation et une utilisation correspondante. Selon la présente invention, dans le lapin transgénique, dont le gène du facteur VIII et/ou du facteur IX a été inactivé, les fonctions du facteur VIII et/ou du facteur IX, qui sont des protéines qui remplissent des fonctions critiques pour l'apparition de l'hémophilie, sont inhibées, de telle sorte que le lapin transgénique est utile pour l'élaboration de traitements de l'hémophilie.
(KO) 본 발명은 Factor VIII 또는 Factor IX 유전자가 녹아웃된 토끼, 이의 제조방법 및 그 용도에 관한 것으로, 보다 구체적으로는 CRISPR/Cas9 시스템으로 Factor VIII 또는 Factor IX을 녹아웃 시킨 형질전환 토끼, 이의 제조방법 및 그 용도에 관한 것이다. 본 발명에 따른 제 8인자 및/또는 제 9인자가 녹아웃된 형질전환 토끼는 혈우병 발생에 중요한 기능을 수행하는 단백질인 제 8인자 및/또는 제 9인자의 기능이 억제되어 있기 때문에, 혈우병 치료제의 개발에 유용하다.
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: Korean (KO)
Filing Language: Korean (KO)