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1. WO2014119589 - OLIGONUCLEOTIDE STRUCTURE AND METHOD FOR REGULATING GENE EXPRESSION

Publication Number WO/2014/119589
Publication Date 07.08.2014
International Application No. PCT/JP2014/051896
International Filing Date 29.01.2014
Chapter 2 Demand Filed 03.12.2014
IPC
C12N 15/113 2010.1
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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
A61K 31/7088 2006.1
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
A61K 31/7105 2006.1
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61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
A61K 31/711 2006.1
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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
711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
A61K 48/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 43/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43Drugs for specific purposes, not provided for in groups A61P1/-A61P41/108
CPC
A61K 31/7088
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
A61K 31/7105
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
A61K 31/711
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
711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
A61P 43/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
C12N 15/111
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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
111General methods applicable to biologically active non-coding nucleic acids
C12N 2310/3519
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2310Structure or type of the nucleic acid
30Chemical structure
35Nature of the modification
351Conjugate
3519Fusion with another nucleic acid
Applicants
  • 公立大学法人大阪市立大学 OSAKA CITY UNIVERSITY [JP]/[JP]
Inventors
  • 立花 亮 TACHIBANA Akira
  • 田辺 利住 TANABE Toshizumi
Agents
  • 平木 祐輔 HIRAKI Yusuke
Priority Data
2013-01929704.02.2013JP
Publication Language Japanese (ja)
Filing Language Japanese (JA)
Designated States
Title
(EN) OLIGONUCLEOTIDE STRUCTURE AND METHOD FOR REGULATING GENE EXPRESSION
(FR) STRUCTURE D'OLIGONUCLÉOTIDE ET PROCÉDÉ DE RÉGULATION DE L'EXPRESSION GÉNIQUE
(JA) オリゴヌクレオチド構造体および遺伝子発現抑制方法
Abstract
(EN) The purpose of the present invention is to provide an oligonucleotide structure and a method for regulating gene expression, said oligonucleotide structure and said method being capable of functioning exclusively in specific cells. The oligonucleotide structure is configured of three oligonucleotide chains including a trigger chain, an effector chain and a support chain, wherein: a part of each of the three oligonucleotide chains is base paired to a part of the other two chains so as to together form a Y-shaped structure; a part of the trigger chain and a part of the support chain each form a stem-loop structure; the effector chain is complementary to at least a part of a target gene transcription product; a part of the trigger chain is complementary to at least a part of a decay-inducing chain that is an RNA; the trigger chain and the support chain are DNAs; and base pairing of at least a part of the decay-inducing chain to a part of the trigger chain triggers the decay of the Y-shaped structure.
(FR) L'objet de la présente invention est de pourvoir à une structure d'oligonucléotide et à un procédé de régulation de l'expression génique, ladite structure d'oligonucléotide et ledit procédé étant capables de fonctionner exclusivement dans des cellules spécifiques. La structure d'oligonucléotide selon l'invention est constituée de trois chaînes oligonucléotidiques comprenant une chaîne de déclenchement, une chaîne effectrice et une chaîne de support. Dans ladite structure d'oligonucléotide, une partie de chacune des trois chaînes oligonucléotidiques est appariée en bases à une partie des deux autres de façon à ce qu'elles forment ensemble une structure en Y; une partie de la chaîne de déclenchement et une partie de la chaîne de support forment chacune une structure tige-boucle; la chaîne effectrice est complémentaire d'au moins une partie d'un produit de transcription d'un gène cible; une partie de la chaîne de déclenchement est complémentaire d'au moins une partie d'une chaîne induisant la désintégration qui est un ARN; la chaîne de déclenchement et la chaîne de support sont des ADN; et l'appariement des bases d'au moins une partie de la chaîne induisant la désintégration à une partie de la chaîne de déclenchement déclenche la désintégration de la structure en Y.
(JA)  特定の細胞内のみで機能する、オリゴヌクレオチド構造体および遺伝子発現抑制方法を提供することである。 トリガー鎖とエフェクター鎖とサポート鎖の3本のオリゴヌクレオチド鎖で構成されるオリゴヌクレオチド構造体であって、前記3本のオリゴヌクレオチド鎖は、それぞれの鎖の一部が他の2本の鎖の一部と塩基対合して、全体でY字構造を形成し、トリガー鎖の一部およびサポート鎖の一部は、ステムループ構造を形成し、エフェクター鎖は、標的遺伝子転写産物の少なくとも一部と相補性を有し、トリガー鎖の一部は、RNAである崩壊誘導鎖の少なくとも一部と相補性を有し、トリガー鎖および前記サポート鎖は、DNAであり、Y字構造は、崩壊誘導鎖の少なくとも一部がトリガー鎖の一部と塩基対合することが引き金となって崩壊する、オリゴヌクレオチド構造体を提供する。
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