Fig. 3
SI113 treatment ameliorates hydrocephalus in a genetic rat model (Tmem67P394L). Normal and hydrocephalic (Tmem67.P394L) littermates were treated with either vehicle (DMSO) or SI113 (8 mg/kg BW) daily from P7 to P15 via intraperitoneal injection as described in the treatment schematic in A. Graphs B and C represent matched normal and hydrocephalic animals, respectively. The two graphs show representative animals with the similar starting ventricular volumes at P7, comparing a mean effect of the vehicle or drug treatment on ventricular volume at P15. Coronal slices from MRI scans taken from the same animals depicted in the graphs are shown beside each graph. Graph D summarizes the change in ventricular volume (VV) from P7 to P15 for all animals in the cohort, indicating the drug treatment was effective at halting the progression of ventriculomegaly in the affected animals, and had no effect on the normal animals’ ventricular volumes. E shows the effect of SI113 treatment on cranial dimensions in the hydrocephalic animals. Data represented as mean ± sem in graphs D and E. Veh = vehicle, DMSO; SI = SI113; VV = ventricular volume; ns = not significant; numbers on the bars in D show the p values for the comparisons listed; **** = p < 0.0001