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. Author manuscript; available in PMC: 2011 Jan 1.
Published in final edited form as: Front Biosci (Landmark Ed). 2010 Jun 1;15:1191–1204. doi: 10.2741/3670

Figure 3.

Figure 3

Therapeutic strategies targeting splicing correction in SMN2. A. Standard ASOs targeting intron splicing silencers (ISS) and the exon 8 3'ss to promote exon 7 inclusion. B. Bifunctional ASOs linked to binding sites for positively acting splicing factors (SR proteins) to increase exon 7 inclusion. A bifunctional ASO linked to binding sites for inhibitory splicing factors (hnRNP proteins) to block the exon 8 3'ss and increase exon 7 inclusion by inhibiting splicing of exon 6 to 8. C. Trans splicing of an RNA molecule that can be used instead of SMN2 exon 7 to produce full length SMN transcripts. D. Drugs that promote the incorporation of exon 7 (green box) or inhibit the splicing of exon 6 to 8 (red box).