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1. WO2020139031 - CRISPR-CAS-BASED COMPOSITION FOR GENE CORRECTION

Publication Number WO/2020/139031
Publication Date 02.07.2020
International Application No. PCT/KR2019/018627
International Filing Date 27.12.2019
IPC
C07K 14/00 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
C12N 9/22 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
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
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 15/63 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
CPC
C07K 14/00
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
C12N 15/10
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 15/63
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
C12N 9/22
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
9Enzymes; Proenzymes; Compositions thereof
14Hydrolases (3)
16acting on ester bonds (3.1)
22Ribonucleases ; RNAses, DNAses
Applicants
  • 한양대학교 산학협력단 INDUSTRY-UNIVERSITY COOPERATION FOUNDATION HANYANG UNIVERSITY [KR]/[KR]
Inventors
  • 최제민 CHOI, Je-Min
  • 구자현 KOO, Ja-Hyun
  • 이홍균 LEE, Hong-Gyun
  • 이재웅 LEE, Jae-Ung
Agents
  • 특허법인 충현 CHUNG HYUN PATENT & LAW FIRM
Priority Data
10-2018-017046327.12.2018KR
10-2019-017490726.12.2019KR
Publication Language Korean (KO)
Filing Language Korean (KO)
Designated States
Title
(EN) CRISPR-CAS-BASED COMPOSITION FOR GENE CORRECTION
(FR) COMPOSITION À BASE DE CRISPR-CAS POUR CORRECTION GÉNIQUE
(KO) CRISPR-CAS를 기반으로 하는 유전자 교정용 조성물
Abstract
(EN)
The present invention relates to a composition for enhancing the cell permeability and gene correction efficiency of a Cas protein and guide RNA, and in the case of currently used techniques for gene correction through CRISPR-Cas, intracellular injection in a complex form is difficult, stability has not been verified even with injection, efficiency is low, and there are off-target problems. However, when the composition for gene correction according to the present invention is used, the efficiency of intracellular delivery is remarkably high, off-target effects can be inhibited, and stability can be ensured, and thus the composition can be effectively used in gene therapy.
(FR)
La présente invention concerne une composition permettant d'améliorer la perméabilité cellulaire et l'efficacité de correction génique d'une protéine Cas et d'ARN guide. Les techniques actuellement utilisées pour la correction génique par CRISPR-Cas, présentent des problèmes se liant à une injection intracellulaire sous une forme complexe difficile, une stabilité non vérifiée même avec l'injection, une faible efficacité, ainsi que l'existence de problèmes hors cible. La présente invention concerne une composition destinée à être utilisée pour la correction génique, ce qui permet d'améliorer de manière remarquable l'efficacité de l'administration intracellulaire, d'inhiber des effets hors cible, et d'assurer la stabilité, ainsi la composition peut être utilisée de manière efficace en thérapie génique.
(KO)
본 발명은 Cas 단백질 및 가이드 RNA의 세포 투과성과 유전자 교정 효율을 향상시키기 위한 조성물에 관한 것으로서 현재 사용되고 있는 CRISPR-Cas를 통한 유전자 교정기술의 경우 복합체 형태로 세포 내에 주입이 어렵고, 주입하더라도 안정성이 입증되지 않았으며 효율이 낮으며, off-target 문제가 있다. 그러나 본 발명의 유전자 교정용 조성물을 이용할 경우 세포 내 전달 효율이 현저히 높을 뿐만 아니라, off-target을 억제할 수 있고, 안정성을 확보할 수 있어, 유전자 치료로서 유용하게 활용될 수 있다.
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Latest bibliographic data on file with the International Bureau