Fig. 5.

Partial normalization of cholesterol metabolism. DHC/cholesterol in the brain (A) and spinal cord (B) of treated (n = 11) and untreated (n = 5) SLOS mice and their normal (T93M/+) littermates (n = 6). SLOS mice were treated IT with 30 μl of AAV-DHCR7 or saline at 3 days of age and sterol analysis by GC/MS was performed at 8 weeks post-treatment. DHC is the sum of 7-DHC and 8-DHC. Statistical analyses were performed using the non-parametric Mann–Whitney test.