Table 2.
Drug developments based on induced MNs
| Reference | Efficiency (%) Isl1 or HB9 |
Total days | Differentiation approach | Rescued phenotype | Number of tested drugs | Number of tested ALS patient-derived cell line | Features of tested cell lines | Drug |
|---|---|---|---|---|---|---|---|---|
| Donnelly et al. (2013) | 30–40 | 40–45 | small molecule | C9orf72 RNA level, nuclear GGGGCC RNA foci, Glutamate exposure susceptibility | 5 (antisense oligo) | 4 | C9orf72 | antisense oligo |
| Wainger et al. (2014) | N.A. (FACS purified) | 52 | small molecule | hyperexcitability | 1 | 2 | SOD1 | ezogabine (retigabine) |
| Naujock et al. (2016) | 60-67 (/DAPI) | >42 | small molecule | spontaneous activity, ion channel imbalance | 1 | 6 | SOD1, FUS | 4-aminopyridine (4AP) |
| Imamura et al. (2017) | 60 (/DAPI) | 14 | TFs polycistronic vector | misfolded SOD1, survival | 1,416 | 1 | SOD1 | bosutinib |
| Guo et al. (2017) | 80 (/DAPI) | 32–38 | small molecule | FUS mis-localization, hyperexcitability, axonal transport | 1 | 3 | FUS | ACY-738/Tubastatin A |
| Fujimori et al. (2018) | 65 (/DAPI) | 60 | small molecule | FUS and TDP-43 aggregation/mis-localization, neurite length, LDH leakage, CC3 | 1,232 | 4 | TDP-43, FUS | ropinirole |
| Thams et al. (2019) | 40 (/DAPI) | 31 | small molecule | survival (under ER stress) | 1,275 | 1 | SOD1 (edited ESC) | TUDCA |