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1. (WO2019066549) GENE MANIPULATION FOR TREATMENT OF RETINAL DYSFUNCTION DISORDER
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Pub. No.: WO/2019/066549 International Application No.: PCT/KR2018/011522
Publication Date: 04.04.2019 International Filing Date: 28.09.2018
IPC:
C12N 15/113 (2010.01) ,C12N 9/22 (2006.01) ,C12N 15/10 (2006.01) ,C12N 15/86 (2006.01) ,C12N 15/90 (2006.01) ,A61K 48/00 (2006.01) ,A61P 27/02 (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
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
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
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
86
Viral vectors
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
87
Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90
Stable introduction of foreign DNA into chromosome
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27
Drugs for disorders of the senses
02
Ophthalmic agents
Applicants:
주식회사 툴젠 TOOLGEN INCORPORATED [KR/KR]; 서울시 금천구 가산디지털1로 219, 1204~1206호 #1204~1206, 219, Gasan digital 1-ro Geumcheon-gu Seoul 08501, KR
서울대학교 산학협력단 SEOUL NATIONAL UNIVERSITY R&DB FOUNDATION [KR/KR]; 서울시 관악구 관악로 1 1, Gwanak-ro Gwanak-gu Seoul 08826, KR
서울대학교 병원 SEOUL NATIONAL UNIVERSITY HOSPITAL [KR/KR]; 서울시 종로구 대학로 101 101, Daehak-ro Jongno-gu Seoul 03080, KR
Inventors:
이정민 LEE, Jung Min; KR
송동우 SONG, Dong Woo; KR
김운기 KIM, Un Gi; KR
김정훈 KIM, Jeong Hun; KR
조동현 JO, Dong Hyun; KR
Agent:
특허법인 아이피에스 IPS PATENT FIRM; 서울시 서초구 반포대로23길 14, 5층 5th Fl., 14, Banpo-daero 23-gil Seocho-gu Seoul 06656, KR
Priority Data:
62/565,83829.09.2017US
Title (EN) GENE MANIPULATION FOR TREATMENT OF RETINAL DYSFUNCTION DISORDER
(FR) MANIPULATION DE GÈNE POUR LE TRAITEMENT D'UN TROUBLE DE DYSFONCTIONNEMENT RÉTINIEN
(KO) 망막 기능장애 질환 치료를 위한 유전자 조작
Abstract:
(EN) The present invention relates to a gene manipulation composition for treatment or alleviation of retinal dysfunction disorder and a method using the same. More specifically, the present invention relates to a gene manipulation composition comprising a guide nucleic acid capable of targeting a retinogenesis gene and a method for treating or alleviating retinal dysfunction-caused diseases by which a retinogenesis gene is artificially manipulated and/or edited using the composition.
(FR) La présente invention concerne une composition de manipulation de gène pour le traitement ou le soulagement d'un trouble de dysfonctionnement rétinien et une méthode l'utilisant. Plus spécifiquement, la présente invention concerne une composition de manipulation de gène comprenant un acide nucléique de guidage capable de cibler un gène de rétinogenèse et une méthode de traitement ou de soulagement de maladies provoquées par un dysfonctionnement rétinien consistant à manipuler et/ou éditer artificiellement un gène de rétinogenèse à l'aide de la composition.
(KO) 본 발명은 망막 기능장애 질환의 치료 또는 개선을 위한 유전자 조작용 조성물 또는 이를 이용한 방법에 관한 것이다. 보다 구체적으로, 망막 기능형성 유전자를 표적화할 수 있는 가이드핵산을 포함하는 유전자 조작용 조성물 및 이를 이용한 망막 기능형성 유전자를 인위적으로 조작 및/또는 교정하여 망막 기능장애로 인한 질환을 치료 또는 개선하는 방법에 관한 것이다.
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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)