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).