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 |