Figure 1.
Identification of BH3 mimetic compounds reducing the P525L FUS-eGFP SG phenotype in iPSC-derived neurons. (a) Effect on the cell viability of the compounds in iPSC-derived neurons. Compounds were tested at different concentrations for 24 h. Cell viability was assessed with calcein AM-Red. The mean of three independent experiments (n = 3) is shown; error bars indicate the standard error of the mean (SEM). Related to Figures S1 and S2. (b) Compounds were tested for 24 h at concentrations that were well tolerated. Fluorescent confocal micrographs show that the indicated compounds reduce FUS-eGFP-positive SGs in iPSC-derived neurons treated for 24 h. GA = gambogic acid. Scale bar = 10 μm. (c) Quantification of individual FUS-eGFP-positive SGs from three independent experiments (n = 3); error bars indicate SEM. Significance was tested using the Kruskal-Wallis test with a Dunn post-test. **** denotes the significance of p < 0.0001 between unstressed and arsenite. **** denotes the significance between treatments and arsenite. *, ** and **** indicate p < 0.5, <0.01, and <0.0001, respectively. Related to Figures S1–S5.