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. 2013 Jul 15;41(17):8144–8165. doi: 10.1093/nar/gkt609

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© The Author(s) 2013. Published by Oxford University Press.

This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited.

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Figure 12. — Model of ASO-mediated correction of SMN2 exon 7 splicing. Only portions of exon 7 and intron 7 are shown (not to scale). Presented structure is based on the SHAPE results (see Figure 6). Structural elements of interest are highlighted. Splicing factors are indicated by colored ovals. Annealing sites of ASO 283–297 (green bar) and U1 snRNA (green line) are shown. Sequestration of the 3′ strands of ISTL1 and ISTL2 by ASO 283–297 releases the 5′ strand of ISTL1 and disrupts TSL3. This structural rearrangement leads to enhanced recruitment of U1 snRNP at the 5′ ss of exon 7 and possibly TIA1.