申请人:Sarepta Therapeutics, Inc.
公开号:US11225662B2
公开(公告)日:2022-01-18
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.
提供了与载体肽连接的寡核苷酸类似物。所公开的化合物可用于治疗各种疾病,例如抑制蛋白质表达或校正异常 mRNA 剪接产物可产生有益治疗效果的疾病。