FIG. 2.

An ss-siRNA corrects splicing of Duchenne's muscular dystrophy (DMD) within patient-derived fibroblast cells. (A) Polymerase chain reaction (PCR) amplification followed by gel electrophoresis to separate aberrant and correct splice products upon the addition of small RNAs (50 nM). (B) Sequencing PCR products to confirm that visualize products are due to skipping of exon 51. Patient-derived GM03429 fibroblast cells were used. CM, negative control duplex RNA; GFP, green fluorescent protein; Luc, luciferase; PTEN, phosphatase and tensin homolog; RM, negative control ss-siRNA.