Figure 1.

LV.LCR-EFS Vector Enhances GAA Expression in Erythrocyte-like Cells

(A) Scheme of LV.EFS.GAA (EFS) and LV.LCR-EFS.GAA (LCR) used in the study. (B–D) GAA expression was measured as GAA specific activity in (B) K562 cells and (C) MEL cells, differentiated in erythrocyte-like cells in the presence of 2% DMSO, and (D) hCD34⁺ cells, differentiated in erythrocyte-like cells after transduction with the EFS or LCR vector. (E) GAA activity measured in conditioned media of hCD34⁺ cells differentiated in erythrocyte-like cells. K562 and MEL cells were transduced at an MOI of 5, and data represent the average of at least three independent experiments. hCD34 cells were transduced at an MOI of 50 prior to differentiation in erythrocyte-like cells in vitro, and data represent four independent experiments of two heathy donors. The data are shown as means ± SEM. ∗∗p < 0.01, ∗∗∗p < 0.001, by one-way ANOVA test.