Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells

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• dc.contributor.author Rodríguez Pizà, Ignasica
• dc.contributor.author Verma, Inder M.ca
• dc.contributor.author Veiga, Annaca
• dc.contributor.author Aasen, Trondca
• dc.contributor.author Izpisúa Belmonte, J. C.ca
• dc.contributor.author Bueren, Juanca
• dc.contributor.author Garreta Bahima, Elenaca
• dc.contributor.author Tiscornia, Gustavoca
• dc.contributor.author Sleep Ronquillo, Eduardca
• dc.contributor.author Raya Chamorro, Ángelca
• dc.contributor.author Río, Paulaca
• dc.contributor.author Consiglio, Antonellaca
• dc.contributor.author Barrero Núñez, María Joséca
• dc.contributor.author Navarro, Susannaca
• dc.contributor.author Vassena, Ritaca
• dc.contributor.author Guenechea, Guillermoca
• dc.date.accessioned 2011-07-27T08:27:57Z
• dc.date.available 2011-07-27T08:27:57Z
• dc.date.issued 2009ca
• dc.description.abstract 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.
• dc.format.mimetype application/pdfca
• dc.identifier.citation Raya 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/nature08129
• dc.identifier.doi http://dx.doi.org/10.1038/nature08129
• dc.identifier.issn 0028-0836
• dc.identifier.uri http://hdl.handle.net/10230/12426
• dc.language.iso engca
• dc.publisher Nature Publishing Groupca
• dc.relation.ispartof Nature. 2009;460(7251):53-9
• dc.rights © Nature Publishing Groupca
• dc.rights.accessRights info:eu-repo/semantics/openAccess
• dc.subject.other Medicina regenerativa
• dc.subject.other Cél·lules mare embrionàries
• dc.title Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cellsca
• dc.type info:eu-repo/semantics/articleca
• dc.type.version info:eu-repo/semantics/publishedVersion