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. 2020 Dec 3;20:312–323. doi: 10.1016/j.omtm.2020.11.018

Figure 1.

Figure 1

Schematic representation of EFS.GBA lentiviral vector integrated proviral DNA and experimental design

(A) The self-inactivating (SIN) lentiviral vector-integrated proviral DNA containing the human codon optimized glucocerebrosidase gene (GBA) cDNA driven by elongation factor 1 alpha short (EFS) promoter. (B) Two studies, the early and late intervention, were conducted using the conditional Gaucher mouse model (GD1 mice). GD1 mice were 11–16 weeks old at the start of the early intervention study while 6- to 9-month-old animals were utilized in the late intervention study. The same transduction and transplantation protocol were employed in both studies, lineage depleted (Lin–) bone marrow (BM) cells were obtained from donor animals, transduced overnight, and transplanted the next day into lethally irradiated recipients. Analyses were conducted 20 weeks posttransplant. (C) Vector copy number (VCN) were estimated 48 h post transduction in a small aliquot of cells from each transduction by qPCR. (D) Transduced cells were used 48 h post transduction in colony forming unit (CFU) assay and single colonies analyzed by PCR after 10–12 days of culture. Transduction efficiency (TE) was estimated by PCR in 70–90 single colonies from each transduction experiment. Error bars represent mean + SD.