I. To determine the safety of lentiviral gene transfer for patients with Fanconi anemia complementation group A (FANCA).
I. To determine the transduction efficiency for human FA patient hematopoietic progenitor cells transduced with a clinical grade lentiviral vector encoding the gene for Fanconi anemia complementation group A.
II. To determine if the clinical grade transduction will result in phenotypic correction of gene modified cells by in vitro assays.
III. To determine if infusion of FANCA gene-modified cells will result in engraftment and improvement in blood counts in FA patients.
BONE MARROW HARVEST FOR CELL COLLECTION: Patients undergo bone marrow harvest for collection of stem/progenitor cells on day -1.
REINFUSION: Patients undergo reinfusion of genetically modified hematopoietic progenitor cells on day 0.
After completion of study treatment, patients are followed up periodically for 15 years.