发明名称 Functionally-modified oligonucleotides and subunits thereof
摘要 Functionally-modified 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 AU2012340390(A1) 申请公布日期 2014.06.26
申请号 AU20120340390 申请日期 2012.11.15
申请人 SAREPTA THERAPEUTICS, INC. 发明人 HANSON, GUNNAR J.;WELLER, DWIGHT D.;CAI, BAO ZHONG;ZHOU, MING
分类号 C04B40/06 主分类号 C04B40/06
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