This is a Phase I/II clinical trial of gene therapy for treating Fanconi anemia using a self-inactivating lentiviral vector to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
Diagnosis of Fanconi anemia FANCA type based on DNA sequencing and sensitivity test for chromosomal cleavage by mitomycin C or butylene oxide.
No cytogenetic abnormalities and the proportion of myelodysplastic abnormalities does not exceed 5% within 3 months prior to stem cell collection.
Age: ≥ 4 years.
Karnofsky: ≥ 70%.
Diagnosis of active malignant disease or myelodysplastic syndrome.
Diagnosis of myeloid leukemia.
Pregnant or lactating females.
Existence of an available HLA-identical related donor.