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. 2015 Jun 15;36(10):1212–1218. doi: 10.1038/aps.2015.43

Table 1. Approaches for the regulation of alternative splicing as a potential therapy.

  Approaches Examples/targeted genes References
Small molecule Targeting protein isoforms Etoricoxib/COX2 35
Targeting expression Aclarubicin/SMA 36
Targeting trans-acting element Toyocamycin/XBP1 37
Antisense oligonucleotide-mediated regulation tDNA-AONs/DMD 38,39
RNAi-based isoform specific modulation SMaRT/MAPT 40