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

Ángel Raya, Ignasi Rodríguez-Piz, Guillermo Guenechea, Rita Vassena, Susana Navarro, María José Barrero, Antonella Consiglio, Maria Castell, Paula Río, Eduard Sleep, Federico González, Gustavo Tiscornia, Elena Garreta, Trond Aasen, Anna Veiga, Inder M. Verma, Jordi Surrallés, Juan Bueren, Juan Carlos Izpisa Belmonte

Research output: Contribution to journalReview articlepeer-review

554 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)53-59
Number of pages7
JournalNature
Volume460
Issue number7251
DOIs
StatePublished - Jul 2 2009

ASJC Scopus subject areas

  • General

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