Peripheral blood- or bone marrow-derived stem cells for treatment of Fanconi's anemia

The aim of the study is to determine the toxicity and efficacy of treatment of Fanconi's anemia by transplantation of gene-modified cells. Peripheral blood stem (progenitor) cells are collected from patients with Fanconi's anemia, genetically modified, by lenti-viruses, to correct the defected gene and then infused back into the patients.