Title:
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Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
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Author:
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Rodríguez Pizà, Ignasi; Verma, Inder M.; Veiga, Anna; Aasen, Trond; Izpisúa Belmonte, J. C.; Bueren, Juan; Garreta, Elena; Tiscornia, Gustavo; Sleep Ronquillo, Eduard; Raya Chamorro, Ángel; Río, Paula; Consiglio, Antonella; Barrero Núñez, María José; Navarro, Susana; Vassena, Rita; Guenechea, Guillermo
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Abstract:
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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. |
Subject(s):
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-Medicina regenerativa -Cél·lules mare embrionàries |
Rights:
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(c) Nature Publishing Group
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Document type:
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Article Article - Published version |
Published by:
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Nature Publishing Group
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