Figure 8.

Long-term protection from lipid storage and demyelination in transplanted MLD mice. Toluidine Blue–stained sections of the sciatic nerve of representative 12-month-old MLD mice transplanted with GFP-LV– and ARSA-LV–transduced As2–/– HSCs, or with WT HSCs. Several demyelinated fibers (arrows) and metachromatic granules in Schwann cells are present in mock-treated and WT HSC–transplanted mice, whereas they are almost absent in mice transplanted with gene-corrected cells. Scale bar: 40 μm. For quantitative data and sample size, see Table 2.