Figure 6.

Thioflavin-S positive inclusions are prevented with AAV-GALC gene therapy but not bone marrow transplantation. The extent of thio-S positive inclusions in the TWI (A) spinal cord, was not significantly improved with bone marrow transplantation (BMT) at either a P40 (D) or an aged (85 days of age) (E) timepoint. Treatment with AAV gene therapy either alone (F,G) or in combination with BMT (H,I) prevented the development of thio-S inclusions at both an early (P40) and late (aged, 350–550 days of age) timepoint. The staining present in the AAV and AAV+BMT treated TWI was comparable to WT mice at the same timepoints (B,C). Observations present in n = 3 animals for all groups except aged TWI-AAV which n = 2. Scale bars represent 0.5 mm.