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1. JP2014065733 - INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS

Office
Japan
Application Number 2013260728
Application Date 18.12.2013
Publication Number 2014065733
Publication Date 17.04.2014
Grant Number 6126983
Grant Date 14.04.2017
Publication Kind B2
IPC
A61K 48/00
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
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 35/76
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
66Microorganisms or materials therefrom
76Viruses; Subviral particles; Bacteriophages
A61P 21/04
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21Drugs for disorders of the muscular or neuromuscular system
04for myasthenia gravis
C12N 15/113
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
CPC
A01K 2267/03
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2267Animals characterised by purpose
03Animal model, e.g. for test or diseases
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 48/005
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
C12N 15/113
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
C12N 2310/315
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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
2310Structure or type of the nucleic acid
30Chemical structure
31of the backbone
315Phosphorothioates
C12N 2310/321
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
2310Structure or type of the nucleic acid
30Chemical structure
32of the sugar
3212'-O-R Modification
Applicants ACADEMISCH ZIEKENHUIS LEIDEN
アカデミシュ ジーケンハウス ライデン ハー.オー.デー.エン. ルムク
Inventors VAN OMMEN GARRIT-JAN BOUDEWIJN
ファン オメン,ハリト-ヤン,バウデヴェイン
VAN DEUTEKOM JUDITH CHRISTINA THEODORA
ファン デーテコム,ユディス,クリスティーナ,テオドラ
DEN DUNNEN JOHANNES THEODORUS
デン ドゥネン,ヨハン,テオドルス
Agents 渡邉 潤三
Priority Data 00203283 21.09.2000 EP
Title
(EN) INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS
(JA) 真核細胞におけるエキソンスキッピングの誘導
Abstract
(EN)

PROBLEM TO BE SOLVED: To provide a method for at least in part decreasing the production of an aberrant protein in a cell, the cell comprising pre-mRNA comprising exons coding for the protein, by inducing so-called exon skipping in the cell.

SOLUTION: Exon-skipping results in mature mRNA that does not contain the skipped exon, and thus, when the exon codes for amino acids, an altered product is obtained. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance, an exon recognition sequence, of an exon. The exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of the exon. The nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance, for the treatment of an inherited disease.

COPYRIGHT: (C)2014,JPO&INPIT

(JA)

【課題】細胞による異常型タンパク質の産生を少なくとも部分的に減少させるための方法であって、異常型タンパク質をコードするエキソンを含むmRNA前駆体を有する細胞にいわゆるエキソンスキッピングを誘導することを特徴とする方法を提供する。
【解決手段】エキソンスキッピングは、スキップされたエキソンを有さない成熟mRNAを生じ、スキップされたエキソンがアミノ酸をコードしている場合、変化した産物が得られる。エキソンスキッピングはあるエキソンのエキソン封入シグナル、例えばエキソン認識配列、を特異的に阻害することが可能な試薬を細胞に与えることによって行われる。該エキソン封入シグナルはエキソンの一部に対して相補的な核酸によって干渉することができる。医薬、例えば遺伝病の治療を目的とした医薬の調製に用いることができる上記の核酸も提供する。
【選択図】なし