The present invention provides siRNAs with modified bases in the antisense strand, e.g., 5-Iodo-Uridine (U(5I)), 5-Bromo-uridine (U(5Br)), or DAP, and methods for using the modified siRNAs to selectively down-regulate the expression of a mutant allele, even when the mutant mRNA differs from wild-type by only a single nucleotide.
本发明提供了在反义链中具有修饰碱基(如 5-
碘-
尿苷 (U(5I))、5-
溴-
尿苷 (U(5Br))或 DAP)的 siRNA,以及使用修饰的 siRNA 选择性地下调突变等位
基因表达的方法,即使突变 mRNA 与野生型只相差一个核苷酸。