Figure 1
From: An immune deficient mouse model for mucopolysaccharidosis IIIA (Sanfilippo syndrome)
(A) The MPSIIIA-TKO mouse model was generated by backcrossing the immune competent MPSIIIA mouse model to an immune deficient model lacking Rag2, CD47 and Il2rg genes. After 4 generations, a breeding colony of SGSH+/−, Rag2−/−, CD47−/− and Il2rg−/− animals was established. (B) SGSH activity was measured in brain lysate from MPSIIIA-TKO mice at 5–6 months of age. SGSH activity was reduced in mice heterozygous for the SGSH mutation and was undetectable in mice homozygous for the SGSH mutation. One-way ANOVA revealed significant genotype differences F (2, 14) = 49.76, p < 0.0001. Post-hoc analysis revealed significant differences between groups as indicated *p = 0.0125, ***p = 0.0003 and ****p ≤ 0.0001 (n = 7 SGSH+/+, 4 SGSH+/−, 6 SGSH−/−). (C) Rotarod performance was evaluated at 10 weeks of age using the accelerating rotarod. Latency to fall was significantly reduced in the MPSIIIA-TKO SGSH−/− animals when compared to their MPSIIIA-TKO SGSH+/+ littermates (*p = 0.0349) (n = 9 SGSH+/+, 5 SGSH−/−).
