Figure 1

KPT−8602 improves dystrophic muscle pathology in zebrafish larvae. (A) Zebrafish drug dosing timeline; (B) Representative images of brightfield and birefringence for both unaffected (WT) and affected (sapje) zebrafish larvae. Scale bars represent 500 μm; (C) Graph summarizing the birefringence score (in percentage of affected/unaffected over total number of larvae assessed in the clutch) across untreated, vehicle-treated, KPT−8602-treated and aminophylline-treated (positive control) fish. KPT−8602 and aminophylline treatment significantly reduced the number of affected fish compared to untreated and vehicle-treated fish (data is represented as mean ± SEM, n = 60 per cohort, across 3 independent trials of n = 20 each; * p < 0.05; one-way ANOVA with Tukey’s HSD test).