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1. WO2020197330 - COMPOSITION FOR TREATING HEMOPHILIA BY MEANS OF BLOOD COAGULATION FACTOR VIII GENE INVERSION CORRECTION

Publication Number WO/2020/197330
Publication Date 01.10.2020
International Application No. PCT/KR2020/004232
International Filing Date 27.03.2020
IPC
C12N 15/85 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
C12N 15/10 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
10Processes for the isolation, preparation or purification of DNA or RNA
C12N 15/113 2010.01
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/90 2006.01
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
C12N 9/22 2006.01
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
A61K 31/7105 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
Applicants
  • 주식회사 툴젠 TOOLGEN INCORPORATED [KR]/[KR]
Inventors
  • 송동우 SONG, Dong Woo
  • 이규준 LEE, Kyu Jun
  • 김운기 KIM, Un Gi
  • 최범석 CHOI, Beom Seok
  • 이강인 LEE, Kang In
Agents
  • 이처영 LEE, Cheo Young
  • 장제환 CHANG, Je Hwan
Priority Data
10-2019-003576328.03.2019KR
Publication Language Korean (KO)
Filing Language Korean (KO)
Designated States
Title
(EN) COMPOSITION FOR TREATING HEMOPHILIA BY MEANS OF BLOOD COAGULATION FACTOR VIII GENE INVERSION CORRECTION
(FR) COMPOSITION POUR LE TRAITEMENT DE L'HÉMOPHILIE AU MOYEN D'UNE CORRECTION D'INVERSION DU GÈNE DU FACTEUR VIII DE LA COAGULATION SANGUINE
(KO) 혈액응고인자 VIII 유전자 역위 보정에 의한 혈우병 치료용 조성물
Abstract
(EN)
The present invention relates to: a composition for correcting inversions by targeting a non-inverted gene and a distal-inverted gene and restoring same, and a composition for treating hemophilia; and a gene scissor system (CRISPR/Cas system) having the ability to restore inversion mutations that have occurred in blood coagulation factor VIII (F8), and a technology for treating hemophilia type A by using same.
(FR)
La présente invention concerne une composition pour corriger les inversions par ciblage d'un gène non inversé et d'un gène inversé distal et la restauration de celui-ci ainsi qu'une composition pour traiter l'hémophilie; et un système de ciseaux génétiques (système CRISPR/Cas) ayant la capacité de restaurer des mutations d'inversion qui se sont produites dans le facteur VIII de la coagulation sanguine (F8) et une technologie pour traiter l'hémophilie de type A à l'aide de celle-ci.
(KO)
본 발명은 역위되지 않은 유전자 및 원위의 역위 유전자를 표적으로 하여 이를 복원시키는 역위 보정용 조성물 및 혈우병을 치료하기 위한 조성물로, 혈액응고인자 VIII(F8)에서 발생한 역위 변이에 대한 복원능이 있는 유전자 가위 시스템(CRISPR/Cas system) 및 이를 이용한 A형 혈우병 치료 기술에 관한 것이다.
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