Transferencia génica FANCA para la anemia de Fanconi utilizando un vector lentiviral autoinactivante de alta seguridad y alta eficiencia

Inclusion Criteria:

1. Diagnosis of Fanconi anemia FANCA type based on DNA sequencing and sensitivity test for chromosomal cleavage by mitomycin C or butylene oxide.

2. No cytogenetic abnormalities and the proportion of myelodysplastic abnormalities does not exceed 5% within 3 months prior to stem cell collection.

3. Age: ≥ 4 years.

4. Karnofsky: ≥ 70%.

5. ANC ≥ 5×10^8/L; PLT ≥ 2×10^10/L.

6. Hemoglobin ≥ 8g/dL.

7. Proper renal and hepatic functions (ULN denotes "upper limit of normal range") with

   • serum creatinine ≤ 1.5×ULN;
   • serum bilirubin ≤ 3×ULN;
   • AST/ALT ≤ 5×ULN.

8. Pulmonary function is normal; DLCO > 50%.

9. Written, informed consent obtained prior to any study-specific procedures.

Exclusion Criteria:

1. Diagnosis of active malignant disease or myelodysplastic syndrome.
2. Diagnosis of myeloid leukemia.
3. Pregnant or lactating females.
4. Existence of an available HLA-identical related donor.
5. Subject infected with HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV antibody positive), Treponema pallidum antibody positive or TB culture positive.
6. Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.