FancA-sW-transduced peripheral blood- or bone marrow- derived stem cells (University of Washington)

The cells were derived by apharesis following filgrastim and plerixafor treatment or bone marrow harvest. The cells are infected by a lentiviral vector, FancA-sW (10 moi), which contains the phosphoglycerate kinase promoter, the FANCA cDNA, and a synthetic, safety-modified woodchuck post transcriptional regulatory element (sW). Transduction of cells was performed by overnight culture on recombinant fibronectin peptide CH-296, in low (5%) oxygen, with the reducing agent, N-acetyl-L-cysteine (NAC), and growth factors (granulocyte colony-stimulating factor (G-CSF), Flt3 ligand, stem cell factor (SCF), and thrombopoietin).