Figure 6.

Characterization of lentiviral vectors for GBA and GRN in a mouse macrophage cell line

(A) Schematic of integrating component of lentiviral vectors from the 5′ long terminal repeat (LTR) to 3′ LTR. RRE, Rev response element; PPT, polypurine tract; MND promoter, myeloproliferative sarcoma virus enhancer, negative control region deleted, dl587rev primer-binding site substituted; WPRE, woodchuck hepatitis virus posttranscriptional regulatory element. (B–I) LVV-GBA increased integrated VCN (B), transgene RNA expression (C), GCase protein expression in lysates (D and F) and media (E), GCase enzymatic activity levels in the lysate (H) and conditioned media (G). Correlation analysis of VCN per diploid genome to transgene RNA expression and transgene RNA expression to lysate GCase activity (I). (J–Q) LVV-GRN increased integrated VCN (J), transgene RNA expression (K), and progranulin protein levels in the lysate (L, N, and P) and conditioned media (M and O). Correlation analysis fo VCN per diploid genome to transgene RNA expression and RNA transgene expression to lysate GRN protein levels (Q). Bars represent means ± SEM. Pearson correlation analysis is shown where indicated, correlating the VCN to RNA expression for cells transduced with LVV.GBA (I) and LVV.GRN (Q).