发明名称 OLIGONUCLEOTIDE ANALOGUES HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
摘要 Oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
申请公布号 EP2576574(A2) 申请公布日期 2013.04.10
申请号 EP20110725839 申请日期 2011.05.27
申请人 SAREPTA THERAPEUTICS, INC. 发明人 HANSON, GUNNAR, J.;RUDOLPH, ALEXANDER, CHARLES;CAI, BAO, ZHONG;ZHOU, MING;WELLER, DWIGHT, D.
分类号 C07F9/22;C07F9/24;C07H21/00 主分类号 C07F9/22
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