Figure 4.

Correction of Bone Pathology in MPS IVA Mice Treated with AAV8 Vectors

(A and B) Correction of chondrocytes vacuolization was assessed by toluidine blue staining analysis using light microscopy of (A) growth plate and (B) articular disc in the knee joint of MPS IVA mice treated with AAV8 vectors. Bone pathology in knockout (KO) and tolerant (MTOL) mice were compared with wild-type, untreated MPS IVA, and treated MPS IVA with AAV8 vectors with or without bone-targeting signal. Scale bars, 25 μm. (C and D) Chondrocyte cell size in growth plate lesions of (C) femur or (D) tibia was quantified by ImageJ software. Data expressed fold change from wild-type group. n = 4–7. Statistics were analyzed by one-way ANOVA with a Bonferroni’s post hoc test. Data are presented as mean ± SD. ^∗p < 0.05 versus wild-type (WT); ^#p < 0.05 versus untreated. (C and D) WT, open bar; untreated, black bar; AAV8-TBG-hGALNS, gray bar; AAV8-TBG-D8-hGALNS, striped bar.