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 |