Some content of this application is unavailable at the moment.
If this situation persist, please contact us atFeedback&Contact
1. (WO2019044974) SMALL GUIDE ANTISENSE NUCLEIC ACID AND USE THEREOF
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2019/044974 International Application No.: PCT/JP2018/032122
Publication Date: 07.03.2019 International Filing Date: 30.08.2018
IPC:
C12N 15/113 (2010.01) ,A61K 31/7088 (2006.01) ,A61P 35/00 (2006.01) ,A61P 43/00 (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
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
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35
Antineoplastic 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
Applicants:
株式会社Veritas In Silico VERITAS IN SILICO INC. [JP/JP]; 東京都品川区西五反田一丁目11番1号 1-11-1, Nishigotanda, Shinagawa-ku, Tokyo 1410031, JP
Inventors:
上村 孝 KAMIMURA, Takashi; JP
梨本 正之 NASHIMOTO, Masayuki; JP
中村 慎吾 NAKAMURA, Shingo; JP
金 玲 JIN, Ling; JP
Agent:
伊藤 克博 ITO, Katsuhiro; JP
渡邉 伸一 WATANABE, Shinichi; JP
Priority Data:
2017-16664131.08.2017JP
Title (EN) SMALL GUIDE ANTISENSE NUCLEIC ACID AND USE THEREOF
(FR) PETIT ACIDE NUCLÉIQUE ANTISENS GUIDE ET UTILISATION CORRESPONDANTE
(JA) スモールガイドアンチセンス核酸とその使用
Abstract:
(EN) The present invention relates to providing a nucleic acid medicine for managing a disease or disorder related to unwanted gene expression. More specifically, the inventors of the present invention developed a technique for efficiently regulating gene expression in a cell using a relatively short oligonucleotide, by establishing a method for designing a nucleic acid medicine, comprising performing structural analysis based on sequence information of a target RNA sequence as an object for gene expression regulation, calculating the probability of existence of each structure from the obtained results of the structural analysis and the energy of each structure, and calculating the existence probability in unified fashion of an endogenous stem-loop substructure, and thereby specifying a more favorable endogenous stem-loop substructure. This technique can theoretically be applied to regulation of any gene expression, and is therefore useful for treatment or prevention of various diseases and disorders.
(FR) La présente invention concerne la fourniture d'un médicament à base d'acide nucléique pour la gestion d'une maladie ou d'un trouble lié à une expression génique indésirable. Plus spécifiquement, les inventeurs de la présente invention ont développé une technique permettant de réguler efficacement l'expression d'un gène dans une cellule à l'aide d'un oligonucléotide relativement court, en établissant une méthode de conception de médicaments à base d'acide nucléique, comprenant les étapes consistant à réaliser une analyse structurelle sur la base des informations de séquence d'une séquence d'ARN cible constituant un objectif pour la régulation de l'expression génique, calculer la probabilité de l'existence de chaque structure à partir des résultats obtenus à partir de l'analyse structurelle et de l'énergie de chaque structure, et calculer la probabilité d'existence d'une manière unifiée d'une sous-structure de type tige-boucle endogène, et ainsi spécifier une sous-structure de type tige-boucle endogène plus favorable. Cette technique peut théoriquement être appliquée à la régulation de l'expression de n'importe quel gène, et est donc utilisable pour le traitement ou la prévention de maladies et troubles variés.
(JA) 本発明は、望ましくない遺伝子の発現に関連する疾患または障害を処置するための核酸医薬を提供することに関する。より具体的には、本発明者らは、遺伝子発現調節の対象となる標的RNA配列について、その配列情報を元に構造解析を行ない、得られた構造解析結果とそれぞれのエネルギー量から、それぞれの存在確率を算出し、内在性のステムループ部分構造の存在確率を統一的に算出することでより好ましい内在性ステムループ部分構造を特定し、核酸医薬を設計する方法を確立することで、比較的短いオリゴヌクレオチドを用いて、効率よく細胞内における遺伝子発現を調節する手法を開発した。本手法は、原理的にあらゆる遺伝子発現の調節に応用可能であることから、多様な疾患および障害の治療または予防に有用である。
front page image
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)