Preclinical safety assessment of modified gamma globin lentiviral vector-mediated autologous hematopoietic stem cell gene therapy for hemoglobinopathies
Fig 1
Vector copy number (VCN) analysis after GbGM lentiviral (LV) vector and SFFV γ-retro viral (RV) vector gene transduction and transplantation in primary wild-type BoyJ mice.
(A) Portion of vector transduced mouse hematopoietic stem and progenitor cells (LSK) from C57BL/6 mice (CD45.2) were expanded in culture for 14–16 days to measure the VCN in the input injected LSK cells. Mock transduced cells (no vector) were used as negative control whereas SFFV RV transduced cells are used as positive control (B-E) Transduced LSK cells were transplanted into sub-lethally irradiated BoyJ recipient mice. The VCN in (B) PB, (C) BM, (D) spleen, and (E) thymus (analyzed via qPCR) at 8 months after transplant in primary BoyJ recipients are shown. Mean ± standard error of the mean (SEM) is shown for each bar. Each symbol in the bar graph represents an individual animal. Statistics: One-way ANOVA; not significant (ns), *P ≤ .05; **P ≤ .01; ***P ≤ .001, ****P ≤ .0001.