- 专利标题： ANTISENSE OLIGONUCLEOTIDE CAPABLE OF ALTERING SPLICING OF DUX4 pre-mRNA
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申请号： US17621449申请日： 2020-07-10
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公开(公告)号： US20220364086A1公开(公告)日： 2022-11-17
- 发明人: Makoto KOIZUMI , Akifumi NAKAMURA , Takahiro KATAGIRI , Hiroaki MITSUHASHI
- 申请人： DAIICHI SANKYO COMPANY, LIMITED , Tokai University Educational System
- 申请人地址： JP Tokyo; JP Tokyo
- 专利权人： DAIICHI SANKYO COMPANY, LIMITED,Tokai University Educational System
- 当前专利权人： DAIICHI SANKYO COMPANY, LIMITED,Tokai University Educational System
- 当前专利权人地址： JP Tokyo; JP Tokyo
- 优先权： JP2019-129735 20190712
- 国际申请： PCT/JP2020/026950 WO 20200710
- 主分类号： C12N15/113
- 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).
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).
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