The generation of induced pluripotent stem (iPS) cells has enabled the 
derivation of patient-specific pluripotent cells and provided valuable 
experimental platforms to model human disease. Patient-specific iPS cells are 
also thought to hold great therapeutic potential, although direct evidence for 
this is still lacking. Here we show that, on correction of the genetic defect, 
somatic cells from Fanconi anaemia patients can be reprogrammed to pluripotency 
to generate patient-specific iPS cells. These cell lines appear 
indistinguishable from human embryonic stem cells and iPS cells from healthy 
individuals. Most importantly, we show that corrected Fanconi-anaemia-specific 
iPS cells can give rise to haematopoietic progenitors of the myeloid and 
erythroid lineages that are phenotypically normal, that is, disease-free. These 
data offer proof-of-concept that iPS cell technology can be used for the 
generation of disease-corrected, patient-specific cells with potential value for 
cell therapy applications.