2026-04-19T16:16:11Zhttps://recercat.cat/oai/request

oai:recercat.cat:10230/124262025-12-19T23:02:33Zcom_2072_6col_2072_452952

Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells Rodríguez Pizà, Ignasi Verma, Inder M. Veiga, Anna Aasen, Trond Izpisúa Belmonte, J. C. Bueren, Juan Garreta Bahima, Elena Tiscornia, Gustavo Sleep Ronquillo, Eduard Raya Chamorro, Ángel Río, Paula Consiglio, Antonella Barrero Núñez, María José Navarro, Susanna Vassena, Rita Guenechea, Guillermo Medicina regenerativa Cél·lules mare embrionàries The 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. 2011-07-27T08:27:57Z 2011-07-27T08:27:57Z 2009 info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion Nature. 2009;460(7251):53-9 © Nature Publishing Group info:eu-repo/semantics/openAccess Nature Publishing Group