I. To determine the safety of lentiviral gene transfer for patients with Fanconi anemia complementation group A (FANCA).
I. To determine the feasibility of collection of the number of hematopoietic progenitor cells from Fanconi anemia complementation group A patients that would be expected to have potential for therapeutic benefit after transduction and infusion. The mobilization will be performed with G-CSF (filgrastim) or with a combination of G-CSF and plerixafor for patients aged 18 and older. Additional bone marrow may be collected if insufficient cells are collected after mobilization and apheresis.
II. 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.
III. To determine if the clinical grade transduction will result in phenotypic correction of gene modified cells by in vitro assays.
IV. To determine if infusion of FANCA gene-modified cells will result in engraftment and improvement in blood counts in FA patients.
STEM CELL MOBILIZATION: Patients receive filgrastim subcutaneously (SC) twice daily on days -5 to -1. Patients 18 years of age or older also receive plerixafor SC on days -2 and -1.
CELL COLLECTION: Patients undergo apheresis for collection of stem/progenitor cells on days -2 and -1. Patients with insufficient cell mobilization undergo bone marrow harvest.
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.
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