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. 2020 May 22;18:50–61. doi: 10.1016/j.omtm.2020.05.015

Figure 2.

Figure 2

Blood and Tissue Human N-Acetylgalactosamine-6-Sulfate Sulfatase (hGALNS) Enzyme Activity 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 hGALNS enzyme activity was measured in (A) knockout (KO) and (B) tolerant (MTOL) mice. 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) hGALNS enzyme activity in tissues including liver, spleen, lung, kidney, heart, and bone (leg) was measured in (C) KO and (D) 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. ∗p < 0.05 versus AAV8-TBG-hGALNS. (A and B) Wild-type (WT), ○; 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. N.D., not detected.