fig1

Figure 1. Non-RNase H-dependent ASO steric blocking effects. In yellow is indicated the complementary targeted sequence on the mRNA. ISS: Intronic splice silencer. A: mRNA Translation inhibition; B: Antagomir; C: Inducing skipping of a pathologic exon carrying stop or out-of-frame mutations; D: Splice modulator inhibiting a non-desired naturally occurring exon skipping.