提供了包含修饰的亚基间键和/或修饰的3'和/或5'-端基的寡核苷酸类似物。 所公开的化合物可用于治疗其中抑制蛋白质表达或纠正异常mRNA剪接产物产生有益治疗效果的疾病。

The present invention is a oligonucleotide comprising the modified coupling between subunits and / or variations on the 3 'and / or 5'-terminal group provides nucleotide analogs. The described compounds are useful for the treatment of the disease to provide a beneficial therapeutic effect of the correction is suppressed or at least mRNA's fleece product of protein expression.