Table 1.
The top 12 hits from the FDA drug set screen with their EC50 and maximum percentage increase in MBNL1 over a negative control
| Compound Name | EC50 (µM) | Maximum Percentage Increase | FDA-approved Therapeutic Area |
|---|---|---|---|
| Mycophenolic acid | 1.3 | 119 | Antiarthritic drugs |
| Chlormethine | 1.9 | 158 | Lymphoma therapy |
| Melphalan | 4.0 | 150 | Cancer therapy |
| Cytarabine | 3.1 | 189 | Lymphoma therapy |
| Thiotepa | 12.2 | 159 | Hematologic agents |
| Monobenzone | 50.0 | 123 | Depigmenting agent |
| Bleomycin | 34.0 | 137 | Cancer therapy |
| Chlorambucil | 17.0 | 262 | Lymphoma therapy |
| Floxuridine | 0.001 | 164 | Cancer therapy |
| Mitomycin | 3.4 | 150 | Cancer therapy |
| Methylthioninium chloride | 3.2 | 120 | Alzheimer's dementia treatment |
| Triamcinolone Acetonide | 0.008 | 71 | Analgesic drugs |
DMSO (0.1%) and ISOX (5 µM) treatments were used as inter-plate negative and positive controls. The percentage increase in ZsGreen-MBNL1 levels were calculated by comparing to 5 µM ISOX treatment (100%) and 0.1% DMSO treatment (0%).