Table 2.

Silencing RNAs Tested in the Experiment to Correct DMD Splicing Using Sequence-Specific siRNA

Name	Antisense sequence (5′ to 3′)	ss or dsRNA	Target gene	Mismatch position
CM	5′-GCUAUACCAGCGUCGUCAUdTdT	ds	Negative control
642661	5′-P-sCsAAsGGsAAsGUsUGsGCsAsUsUUsCss	ss	Exon 51, DMD	10
678870	5′-UsCsAsAsGsGsAsAsGsAsUsGsGsCsAsUsUsUsCsU	ss, 2′-OMe	Exon 51, DMD	-
6462662	5′-P-sAsGGsAAsGAsUGsCCsAUsUsUsCsUsAss	ss	Exon 51, DMD	11
678865	5′-P-sCAAGGAAsGUsUGsGCsAsUsUsUsCss	ss, 3 less PS	Exon 51, DMD	10
678866	5′-P-sCAAGGAAGUUGsGCsAsUsUsUsCss	ss, 5 less PS	Exon 51, DMD	10
678867	5′-P-sCsAAsGCsAAsGUsUGsGCsAsUsUsUsCss	ss	Exon 51, DMD	6, 10
678868	5′-P-sCsAUsGCsAAsGUsUGsGCsAsUsUsUsCss	ss	Exon 51, DMD	4, 6, 10
678869	5′-P-sCsAUsGCsAUsGUsUGsGCsAsUsUsUsCss	ss	Exon 51, DMD	4, 6, 8, 10

Modification positions within the sequence are indicated as follows: boxed, 2′-O-MOE (methoxyethyl); underlined, 2′-Fluoro; background shaded, 2′-O-me (methyl); bold and italicized, mismatched bases; subscripted “s,” phosphorothioate (PS) linkage; d,deoxyribose. DMD, Duchenne's muscular dystrophy; dsRNA, double stranded RNA; ssRNA, single-stranded RNA.