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11 条记录 (耗时 0.031 秒)

    MYOCARDIAL DYSFUNCTION THERAPEUTIC AGENT
    3.
    发明申请
    MYOCARDIAL DYSFUNCTION THERAPEUTIC AGENT 有权

    公开(公告)号：US20210207136A1

    公开(公告)日：2021-07-08

    申请号：US17056824

    申请日：2019-06-12

    申请人： DAIICHI SANKYO COMPANY, LIMITED ,  KOBE GAKUIN EDUCATIONAL FOUNDATION

    发明人： Masafumi MATSUO ,  Makoto KOIZUMI ,  Yoshiyuki ONISHI ,  Takao SHOJI

    IPC分类号： C12N15/113

    摘要： The present invention establishes a method for treating cardiac dysfunction. An oligonucleotide of 15-30 bp comprising a nucleotide sequence complementary to a part of the intron 55 region of a dystrophin gene, which comprises the sequence of 5′-TGTCTTCCT-3′ or 5′-CAGCTTGAACCGGGC-3′ (SEQ ID NO: 64) (wherein “T” may be “U” in either sequence), a pharmacologically acceptable salt thereof, or a solvate thereof. A prophylactic and/or a therapeutic for cardiac dysfunction, comprising the above-described oligonucleotide, a pharmacologically acceptable salt thereof, or a solvate thereof. A suppressor of Dp116 expression, comprising the above-described oligonucleotide, a pharmacologically acceptable salt thereof, or a solvate thereof.

    ANTISENSE OLIGONUCLEOTIDE CAPABLE OF ALTERING SPLICING OF DUX4 pre-mRNA
    6.
    发明申请
    ANTISENSE OLIGONUCLEOTIDE CAPABLE OF ALTERING SPLICING OF DUX4 pre-mRNA 有权

    公开(公告)号：US20220364086A1

    公开(公告)日：2022-11-17

    申请号：US17621449

    申请日：2020-07-10

    申请人： DAIICHI SANKYO COMPANY, LIMITED ,  Tokai University Educational System

    发明人： Makoto KOIZUMI ,  Akifumi NAKAMURA ,  Takahiro KATAGIRI ,  Hiroaki MITSUHASHI

    IPC分类号： C12N15/113

    摘要： The present invention aims at establishing a novel therapy for facioscapulohumeral muscular dystrophy.
    An oligonucleotide or a pharmaceutically acceptable salt thereof, wherein the oligonucleotide comprises an oligonucleotide of 15-30 bases consisting of a nucleotide sequence complementary to the region of nucleotide Nos. 502-556 or 578-612 of DUX4-fl mRNA consisting of the nucleotide sequence as shown in SEQ ID NO: 1; the 5′ and/or 3′ end of the oligonucleotide may be chemically modified; and the oligonucleotide is capable of switching the splice form of the DUX4 gene from DUX4-fl to DUX4-s. A pharmaceutical drug comprising the above oligonucleotide or a pharmaceutically acceptable salt thereof (e.g. therapeutic for facioscapulohumeral muscular dystrophy).

    STABLE TARGET-EDITING GUIDE RNA HAVING CHEMICALLY MODIFIED NUCLEIC ACID INTRODUCED THEREINTO
    9.
    发明申请
    STABLE TARGET-EDITING GUIDE RNA HAVING CHEMICALLY MODIFIED NUCLEIC ACID INTRODUCED THEREINTO 有权

    公开(公告)号：US20230061732A1

    公开(公告)日：2023-03-02

    申请号：US17616430

    申请日：2020-06-04

    申请人： FUKUOKA UNIVERSITY ,  DAIICHI SANKYO COMPANY, LIMITED

    发明人： Masatora FUKUDA ,  Makoto KOIZUMI ,  Shinzo IWASHITA

    IPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/7125 ,  A61P43/00

    摘要： An oligonucleotide that induces a site-specific editing for a target RNA, the oligonucleotide including a first oligonucleotide that specifies the target RNA, a second oligonucleotide that is linked to the 3′-side of the first oligonucleotide, a third oligonucleotide that is capable of forming a complementary pair together with the second oligonucleotide, and a first linking portion that links the second oligonucleotide and the third oligonucleotide. The first oligonucleotide is composed of a target-corresponding nucleotide residue that corresponds to an adenosine residue in the target RNA; an oligonucleotide of 10 to 30 residues linked to the 5′-side of the target-corresponding nucleotide residue and having a base sequence complementary to the target RNA; and an oligonucleotide of 3-6 residues linked to the 3′-side of the target-corresponding nucleotide residue and having a base sequence complementary to the target RNA. The second oligonucleotide consists of 5 to 8 residues and the third oligonucleotide consists of 5 to 8 residues. At least one residue selected from a counter region composed of the target-corresponding nucleotide residue and two respective residues on the 3′- and 5′-sides thereof is a nucleotide residue other than a natural ribonucleotide residue.