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专利概况
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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