Fig. 2.

Characterisation of iPSC-derived NPCs. iPSC-derived NPC morphology was assessed by brightfield microscopy (A). PAX6 and NESTIN both markers characteristic of NPCs were assessed by immunofluorescence (A) and gene expression (B). Sulphamidase activity (C), the monosulphated dissacharide HS storage biomarker (D), ganglioside G_M2 (E) and ganglioside G_M3 (F) were measured in cell extracts and normalised to cell protein. Quantitative data is expressed as the mean ± std. dev of n = 3 (sulphamidase activity) or n = 6 (G_M2 and G_M3) replicates. HN-UA(1S) is expressed as the average of 2 replicates where the individual values are:- Control 0.2 and 0.25 pmol/mg protein; MPS IIIA-intermed 86.5 and 50.5 pmol/mg protein.

* denotes significant difference between MPS IIIA-intermed and control, P < 0.05.

Scale bars are 50 μm as indicated.