GALC activity and distribution. GALC activity was significantly higher in the untreated wt mice compared with the untreated mut and BMT-mut groups (A). There was no significant difference between the untreated mutant and BMT-mut groups (A). A separate analysis showed that the GALC activity in the brains of AAV-mut and AAV+BMT-mut groups was approximately fivefold greater and significantly different from that of the untreated wt group (B). The horizontal bars represent means, and error bars represent SEM. *p < 0.05; **p < 0.01; ***p < 0.001. GALC activity can be seen histochemically in the forebrain (C), cerebellum (G), and spinal cord (K) of the untreated wt group. In contrast, no GALC staining is observed in the forebrain (D), cerebellum (H), or spinal cord (L) of the untreated mut group. Intense GALC staining is observed in the ependyma of lateral and fourth ventricles of the AAV-mut and AAV+BMT-mut groups (asterisks; E, F, I, J). Intense staining was also observed in the meninges of the spinal cord and along the spinal nerve roots (arrows; M, N) of AAV-mut and AAV+BMT-mut mice. C–J were imaged at the same magnification. Scale bars: F, J, ∼600 μm. K–N were imaged at the same magnification. Scale bar: N, ∼600 μm.