Figure 3.

Blood and Tissue Glycosaminoglycan (GAG) Levels in MPS IVA Mice Treated with AAV8 Vectors

(A and B) A blood sample was collected from MPS IVA mice every other week until 16 weeks of age, and plasma mono-sulfated KS level was measured in (A) knockout (KO) and (B) tolerant (MTOL) mice. n = 4–8. The tissue sample was collected from MPS IVA mice 12 weeks post-injection of AAV vectors with or without a bone-targeting signal. (C and D) The amount of mono-sulfated KS in tissues, including (C) liver and (D) lung, was measured in KO and MTOL mice. n = 4–8. Statistics were analyzed by one-way ANOVA with a Bonferroni’s post hoc test. Data are presented as mean ± SD. (A and B) ^∗p < 0.05 versus untreated. (C and D) ^∗p < 0.05 versus wild-type (WT); ^#p < 0.05 versus untreated; ^$p < 0.05. (A and B) WT, ○; untreated, ●; AAV8-TBG-hGALNS, ▪; AAV8-TBG-D8-hGALNS, ▲. (C and D) WT, open bar; untreated, black bar; AAV8-TBG-hGALNS, gray bar; AAV8-TBG-D8-hGALNS, striped bar.