The generation of patient-specific induced pluripotent stem cells (iPSCs) offers 
unprecedented opportunities for modeling and treating human disease. In 
combination with gene therapy, the iPSC technology can be used to generate 
disease-free progenitor cells of potential interest for autologous cell therapy. 
We explain a protocol for the reproducible generation of genetically corrected 
iPSCs starting from the skin biopsies of Fanconi anemia patients using 
retroviral transduction with OCT4, SOX2 and KLF4. Before reprogramming, the 
fibroblasts and/or keratinocytes of the patients are genetically corrected with 
lentiviruses expressing FANCA. The same approach may be used for other diseases 
susceptible to gene therapy correction. Genetically corrected, characterized 
lines of patient-specific iPSCs can be obtained in 4-5 months.