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1. (WO2018225807) GENE THERAPY FOR GRANULAR CORNEAL DYSTROPHY
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Pub. No.: WO/2018/225807 International Application No.: PCT/JP2018/021797
Publication Date: 13.12.2018 International Filing Date: 07.06.2018
IPC:
C12N 15/09 (2006.01) ,A61K 31/7105 (2006.01) ,A61K 31/711 (2006.01) ,A61K 48/00 (2006.01) ,A61P 27/02 (2006.01) ,A61P 43/00 (2006.01) ,C12N 9/16 (2006.01) ,C12N 15/113 (2010.01)
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
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
7105
Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
711
Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43
Drugs for specific purposes, not provided for in groups A61P1/-A61P41/108
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)
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
Applicants:
国立大学法人東京大学 THE UNIVERSITY OF TOKYO [JP/JP]; 東京都文京区本郷七丁目3番1号 3-1, Hongo 7-chome, Bunkyo-ku, Tokyo 1138654, JP
国立大学法人千葉大学 CHIBA UNIVERSITY [JP/JP]; 千葉県千葉市稲毛区弥生町1番33号 1-33, Yayoicho, Inage Ward, Chiba-shi, Chiba 2638522, JP
Inventors:
臼井 智彦 USUI, Tomohiko; JP
竹渓 友佳子 TAKETANI, Yukako; JP
大内 靖夫 OUCHI, Yasuo; JP
Agent:
稲葉 良幸 INABA, Yoshiyuki; JP
大貫 敏史 ONUKI, Toshifumi; JP
Priority Data:
2017-11240607.06.2017JP
Title (EN) GENE THERAPY FOR GRANULAR CORNEAL DYSTROPHY
(FR) THÉRAPIE GÉNIQUE POUR DYSTROPHIE CORNÉENNE GRANULAIRE
(JA) 顆粒状角膜変性症に対する遺伝子治療薬
Abstract:
(EN) The present invention pertains to a guide RNA molecule that hybridizes with a target sequence including a mutation site of a transforming growth factor β-inducible (TGFBI) gene associated with granular corneal dystrophy.
(FR) La présente invention concerne une molécule d'ARN guide qui s'hybride avec une séquence cible comprenant un site de mutation d'un gène inductible par le facteur de croissance transformant β (TGFBI) associé à la dystrophie cornéenne granulaire.
(JA) 本発明は、顆粒状角膜変性症に関連する、トランスフォーミング増殖因子β誘発(TGFBI)遺伝子の変異部位を含む標的配列とハイブリダイズする、ガイドRNA分子に関する。
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: Japanese (JA)
Filing Language: Japanese (JA)