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公开(公告)号：US20220364086A1
公开(公告)日：2022-11-17
申请号：US17621449
申请日：2020-07-10
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).-
公开(公告)号：US20200172903A1
公开(公告)日：2020-06-04
申请号：US16616743
申请日：2018-07-03
发明人： Masayuki NAKAMORI , Hideki MOCHIZUKI , Satoshi OBIKA , Makoto KOIZUMI , Akifumi NAKAMURA , Kiyosumi TAKAISHI , Yumiko ASAHI
IPC分类号： C12N15/113 , A61P25/16 , A61P25/28
摘要： The objective of the present invention is to provide nucleic acid therapeutics which exhibits more excellent effect and which shows a substantivity for a prolonged period to suppress an expression of α-synuclein. The oligonucleotide or a pharmacologically acceptable salt thereof according to the present invention is characterized in comprising at least one 2′-O,4′-C-ethylene nucleoside, wherein the oligonucleotide can hybridize with α-synuclein gene, has an activity to suppress an expression of the α-synuclein gene, and is complementary to the α-synuclein gene, 5′ end of the oligonucleotide is a nucleotide complementary to the specific nucleotide, the oligonucleotide is complementary to at least a part of SEQ ID NO: 1, and the oligonucleotide has a length of 13 or more and 15 or less nucleotides.
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