Rodríguez Pizà, IgnasiVerma, Inder M.Veiga, AnnaAasen, TrondIzpisúa Belmonte, J. C.Bueren, JuanGarreta Bahima, ElenaTiscornia, GustavoSleep Ronquillo, EduardRaya Chamorro, ÁngelRío, PaulaConsiglio, AntonellaBarrero Núñez, María JoséNavarro, SusannaVassena, RitaGuenechea, Guillermo2011-07-272011-07-272009Raya Á, Rodríguez-Piza I, Guenechea G, Vassena R, Navarro S, Barrero MJ et al. Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature. 2009;460(7251):53-9. DOI: 10.1038/nature081290028-0836http://hdl.handle.net/10230/12426The generation of induced pluripotent stem (iPS) cells has enabled the derivation of patient-specific pluripotent cells and/nprovided valuable experimental platforms to model human disease. Patient-specific iPS cells are also thought to hold great/ntherapeutic potential, although direct evidence for this is still lacking. Here we show that, on correction of the genetic defect,/nsomatic 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.application/pdfeng© Nature Publishing GroupMedicina regenerativaCél·lules mare embrionàriesinfo:eu-repo/semantics/articlehttp://dx.doi.org/10.1038/nature08129info:eu-repo/semantics/openAccess