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专利概况
专利名称 COMPOSITIONS AND METHODS OF SML2 SPLICING MODULATION IN SUBJECT
申请号 RU2015139553 申请日
公开(公告)号 RU2683772C2 公开(公告)日
申请(专利权)人 BIOGEN MA INC; COLD SPRING HARBOR LABORATORY 发明人 BENNETT C Frank; HUNG Gene; RIGO Frank; KRAINER Adrian R; HUA Yimin; PASSINI Marco A; SHIHABUDDIN Lamya; CHENG Seng H; KLINGER Katherine W
专利来源 国家知识产权局 转化方式
摘要

FIELD : medicine.SUBSTANCE : invention relates to the field of medicine and is intended for the treatment of spinal muscular atrophy (SMA). Method includes the introduction, through a bolus injection into the intrathecal space of a human subject, of an antisense compound containing an antisense oligonucleotide consisting of 18 linked nucleosides, where each internucleoside oligonucleotide bond is a phosphorothioate bond, where each oligonucleotide nucleoside is 2' -MOE nucleoside and where the administration of an antisense compound enhances the incorporation of exon 7 into SMN2 mRNA transcripts in a human subject.EFFECT : invention provides an effective treatment for SMA types I, II and III.12 cl, 13 dwg, 14 tbl, 14 ex

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