申请人:SAREPTA THERAPEUTICS, INC.
公开号:US20160237426A1
公开(公告)日:2016-08-18
Oligonucleotide analogues conjugated to carrier peptides are provided. The disclosed compounds are useful for the treatment of various diseases, for example diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
提供了与载体肽共轭的寡核苷酸类似物。所公开的化合物对于治疗各种疾病是有用的,例如在抑制蛋白质表达或修正异常mRNA剪接产物产生有益的治疗效果的疾病中。