FIG. 2.
Effect of ss-siRNA length on inhibition of HTT expression. (A) List of ss-siRNAs. Subscript “s” indicates PS linkage; 2′-O-Me modified base is shown in uppercase; 2′-F in underlined uppercase; 2′-MOE in enclosed box. Mismatched base is shown in bold and italic. (B) Allele-selective depletion of mutant HTT in patient fibroblasts (GM 04281) treated with ss-siRNAs tested at 25 nM. (C) Effect on HTT expression of increasing concentrations of ISIS 581440 and ISIS 581441. Western analysis data is representative of triplicate experiments that were averaged to yield IC50 and selectivity values. Mut, mutant; nt, nucleotides; Tm, melting temperature with a complementary 22-mer RNA; WT, wild type.