专利名称 | ANTISENSE NUCLEIC ACID | ||
申请号 | JP2019001091 | 申请日 | |
公开(公告)号 | JP2019062913A | 公开(公告)日 | |
申请(专利权)人 | NIPPON SHINYAKU CO LTD; NAT CENTER OF NEUROLOGY PSYCHIATRY | 发明人 | WATANABE NAOKI; SATO YOHEI; TAKEDA SHINICHI; NAGATA TETSUYA |
专利来源 | 国家知识产权局 | 转化方式 | |
摘要 |
To provide agents for skipping of the 53rd exon of a human dystrophin gene with high efficiency.SOLUTION : The present invention provides an oligonucleotide enabling skipping of the 53rd exon of a human dystrophin gene, or an antisense oligomer in which the sugar moiety and/or phosphoric acid binding moiety of at least one nucleotide constituting the oligonucleotide is modified, and a pharmaceutical composition for treating muscular dystrophy containing the antisense oligomer and a pharmaceutically acceptable salt or hydrate thereof as active ingredients.SELECTED DRAWING : None |
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