Figure 7.

Long-term protection from lipid storage in transplanted MLD mice. (A and B) Toluidine Blue–stained semithin sections of the cerebellum (A) and the hippocampal fimbria (B) of representative 12-month-old MLD mice transplanted with GFP-LV– and ARSA-LV–transduced As2–/– HSCs, or with WT HSCs. Abundant large metachromatic deposits are present in mock-treated and WT HSC–transplanted mice, whereas they are almost absent in mice transplanted with gene-corrected cells. Scale bar: 50 μm. For quantitative data and sample size, see Table 2.