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.
Single group
Gene-modified autologous stem cells
GeneticInfusion for 5x10\^6\~1x10\^7 per kilogram of body weight of gene-modified cells; or more infusions depending on the circumstances
Safety in patients using CTCAE version 4.0 standard to evaluate the level of adverse events
Physiological parameter (measuring cytokine response, fever, symptoms)
Treatment responses
Blood routine indexes will be obtained before and after treatment. Objective response, such as complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) will be assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria
Quality of life
Quality of life will be measured using the Functional Assessment of Cancer Therapy-General (FACT-G) before and after treatment.
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