Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer
Figure 2
Intrafemoral gene transfer into BM of FA mutant mice. (a) Illustration of the experimental protocol used to determine the corrective potential of intrafemoral LV-mediated gene transfer into BM cells. Wild type (WT), and mice were intrafemorally injected with recombinant FancC-EGFP lentiviral particles. Transgene expression was monitored each month for at least four months. 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 injection of 0.3 mg/kg). (b) FancC-EGFP transgene expression in peripheral blood cells of IF injected WT, , and mice as a function of time. Each dot represents the FancC-EGFP expression of an individual IF-injected mouse. Horizontal line: mean value. . (c) Representative FACS profiles of FancC-EGFP expression in peripheral blood cell lineages at four months after IF injection.
