Chemically modified siRNAs are allele-selective
inhibitors of ATN-1 expression. Gels are Western
analyses of ATN-1 levels.
Graphs are based on triplicate determinations. CM is a control duplex
RNA that is not complementary to ATN-1 mRNA. (A)
Sequences, IC50 values, and selectivities of chemically
modified duplex RNAs used for panels B–D. The bases shown in
bold italics are mismatched relative to the CAG repeat region. Lowercase
denotes 2′-O-methyl base substitutions. (B)
Western analysis showing inhibition of ATN-1 protein expression when
RNAs CMOD1, -2, -12, -13, -14, -22, and -23 and CM are added at a
concentration of 3 nM. (C and D) Dose–response curves related
to IC50 value determination for duplexes for CMOD12 and
CMOD13, respectively.