申请人:Sarepta Therapeutics, Inc.
公开号:US10344281B2
公开(公告)日:2019-07-09
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.
本研究提供了功能修饰的寡核苷酸类似物,这些类似物包含修饰的亚基间连接和/或修饰的 3′和/或 5′末端基团。所公开的化合物可用于治疗抑制蛋白质表达或校正异常 mRNA 剪接产物产生有益治疗效果的疾病。