Table 1.
Drugs used to target alternative splicing and impacts on autophagy.
| Drugs | Function and target | Diseases | Ref. |
|---|---|---|---|
| Small-molecule splicing modulators | |||
| Spliceostatin A1 | SF3B1 inhibitor | – | 143 |
| Pladienolide B | SF3B1 inhibitor | Myelodysplastic syndrome | 143–145 |
| Herboxidine | SF3B1 inhibitor | – | 143 |
| BET inhibitors | Regulation of alternative splicing and autophagy inducer | – | 146–148 |
| Quercetin | HNRNPA1 inhibitor | Cancer | 149–151 |
| Small-molecule autophagy modulators | |||
| Rapalogs | MTOR inhibitors, autophagy inducers and TRIB3-mediated alternative splicing | Cancer; neurodegenerative diseases | 153 |
| Carbamazepine (CBZ) | Autophagy inducer | Epilepsy; bipolar disorders; metaphyseal chondrodysplasia, Schmid type | 154 |
| Chloroquine (CQ) | Autophagy inhibitor via autophagosome-lysosome fusion; HNRNPK inhibitor | Myotonic dystrophy type 1 | 155–157 |
| Antisense morpholino oligonucleotides | |||
| Specific-target AMOs candidate | pre-mRNAs splicing manipulation. Targets splicing motifs to: reinforce exon selection, excise exons that contain nonsense mutations or those that flank frameshifting rearrangements. | Duchenne muscular dystrophy | 158 |
| Small-molecule targeting RNA/RNA-protein complexes | |||
| Risdiplam | The drug promotes the inclusion of exon 7 in SMN2 and thereby the production of SMN2 protein. | Spinal muscular atrophy | 163–165 |