Allele-selective inhibition of ATN-1 expression
by ss-siRNAs. 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 ss-siRNAs used for panels
B–I. A subscript s indicates a phosphorothioate (PS) linkage.
The 2′-O-methyl (2′-O-Me)-modified base is denoted in lowercase. The 2′-fluoro
(2′-F) group is shown in underlined uppercase. Tm means 2′-methoxyethyl (2′-MOE)-modified RNA T. Mismatched
bases are shown in bold italics. The terminal thymidine has a 5′-phosphate
or vinyl phosphonate. All other linkages are phosphates. (B and C)
Dose–response curves related to ss-siRNAs 553775 and 553822,
respectively. (D and E) ss-siRNAs tested at 3 and 6 nM, respectively.
(F–I) Dose–response curves for ss-siRNAs 557426, 618385,
618204, and 641384, respectively.