FIG. 3.

Correction of DMD splicing by ss-siRNA is sequence specific. (A) Correction of DMD splicing by a complementary ss-siRNA and ss-siRNAs containing mismatched bases (50 nM). (B) Correction of dystrophin splicing by ss-siRNA (50 nM) does not provoke an interferon response. Five different interferon-responsive genes were monitored by quantitative PCR after treatment with 50 nM ss-siRNA. Poly I:C is a positive control nucleic acid known to stimulate the interferon response. Patient-derived GM03429 fibroblast cells were used. IFITM1, interferon-induced transmembrane protein 1; ISGF3g, interferon-stimulated transcription factor 3 gamma; MX1, interferon-induced GTP-binding protein Mx1; NT, no treatment with RNA; OAS1/2, 2'-5'-oligoadenylate synthetase 1/2.