Biochemical and functional correction of FA-C cells. (A) FANCC-deficient or unaffected human fibroblasts were transduced with foamyviral vectors encoding either the reporter gene only or MD9-FANCC/EGFP. Forty-eight hours following transduction, the cells were treated with IR (10 gray) and 3 hours later protein extracts were collected. Protein extract (20 μg) was analyzed by Western blot for monoubiquitinated FANCD2. (B-E) Clonogenic progenitor cell growth in the presence of increasing concentrations of MMC (B,D) or TNF-α (C,E). (B,C) Triplicate cultures of transduced c-kit⁺ cells were cultured immediately after transduction. (D,E) Six months following transplantation, transduced bone marrow cells from each experimental group were analyzed in triplicate cultures (WT-MD9-EGFP, n = 3; Fancc^−/−-MD9-EGFP, n = 3; Fancc^−/−-MD9-FANCC/EGFP, n = 5). Data reflect the mean and standard error of the mean (SEM) of all recipients examined in each experimental group. *P < .05 comparing Fancc^−/− EGFP to the other experimental groups.