Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer
Figure 6
Corrective potential of intrafemoral gene transfer into FA mice with BM aplasia. (a) Illustration of the experimental protocol used to determine the corrective potential of intrafemoral LV-mediated gene transfer into aplastic BM. Wild type (WT), , and mice were preconditioned with MMC five days prior to intrafemoral injection of recombinant FancC-EGFP lentiviral particles. At 7 weeks following IF injections, each group of mice was divided where half of the mice were treated with MMC for 15 weeks (weakly s.c. injection of 0.3 mg/kg). (b) Transgene expression was monitored each month for four months: FancC-EGFP transgene expression in peripheral blood cells of preconditioned IF-injected wild type (WT), , and mice. Each dot represents the FancC-EGFP expression of an individual IF-injected mouse. Horizontal line: mean value. . (c) Representative cytometric profiles of FancC-EGFP expression in peripheral blood cell lineages from preconditioned IF-injected mice at four months following in vivo gene transfer.
