Figure 7.

NaGlu delivery was associated with a decreased accumulation of GM2 and GM3 gangliosides. Gangliosides were measured in total lipid extracts from the brains of normal mice and untreated MPSIIIB mice and from the injected or noninjected hemisphere of MPSIIIB mice that received a single injection of AAV2-NaGlu vectors in the putamen at 6 weeks of age (AAV2-NaGlu vector-treated MPSIIIB mice). The age of mice at analysis and the number of mice in each group are indicated. Data are expressed as molar percentages of total gangliosides according to NeuAc moiety numbers. Asterisks indicate mean values in treated mice that are significantly lower than those in untreated mice at 5 weeks of age (p ≤ 0.04; Mann-Whitney U test). Mean GM2 and GM3 values at 12 weeks of age in the injected hemisphere of treated mice were equivalent to mean values at the same age in normal mice.