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. 2022 Nov 3;27:464–487. doi: 10.1016/j.omtm.2022.10.017

Figure 2.

Figure 2

High levels of GAA in hematopoietic compartment

(A) GAA enzyme activity in bone marrow cells. Individual values, group medians, and interquartile range are presented; Gaa+/+ versus treated (dotted line); GAAco versus GILT-tagged variants; GILTco1-m versus treatment groups (n = 10).n-treated. (B) Western blot analysis of bone marrow lysates showing both precursor (110 kDa) and mature GAA species (75–70 kDa). Loading control: anti-β-actin (n = 3). (C) GAA enzyme activity in plasma at week 16 post transplant, comparing non-treated Gaa+/+with treated Gaa−/−--/-, and Gaa+/+ with treated groups (n = 8-13). (D) Western blot analysis of representative plasma samples (n = 3) at week 16 post transplant using an anti-GAA monoclonal antibody. (E) Correlation analysis between plasma GAA enzyme activity and plasma GAA protein concentration by simple linear regression. GILTco3-m is depicted by red triangles. (p < 0001). (F) VCN measured in bone marrow cells comparing GAAco-treated group with GILT-tagged variants, and GILTco1-m versus GILTco3-m (n = 9–10). (G) Urinary Hex4 at week 16 post transplant; individual values, group medians, and interquartile ranges are presented (n = 4–10). Untreated Gaa−/− mice versus GAAco Kruskal-Wallis test and Dunn’s test for multiple comparison between the treatment arms. (A, C, and F) Exact Wilcoxon rank-sum test comparing arms. ∗p < 0.05, ∗∗p < 0.01, ∗∗∗p < 0.001, and ∗∗∗∗p < 0.0001. ns = not significant. Bu = Busulfex.