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Mechanisms of action for therapeutic oligonucleotides. (A) control of splicing by ASOs and alternative splicing. In contrast to gapmers, ASOs that control splicing possess ribose or morpholino modifications throughout the oligomer; (B) block of translation by antisense gapmers (oligonucleotide that contain 2′-modified RNA flanking a central DNA region) targeting mRNA; (C) block of translation by a fully complementary dsRNA and RNA interference.
