Editor's Summary

2 July 2009

Correcting Fanconi anaemia


The feasibility of deriving patient-specific iPS cells and their value as experimental models for specific diseases were reported almost a year ago. Patient-specific iPS cells are also thought to have great therapeutic potential, though direct evidence was lacking. Raya et al. now show that iPS cells from Fanconi anaemia patients can, after correction of the genetic defect, be reprogrammed to generate patient-specific iPS cells that can give rise to disease-free haematopoietic progenitors of myeloid and erythroid lineages. These cells have potential value for cell therapy.

AuthorsMaking the paper: Juan Carlos Izpisúa Belmonte

Healthy cells derived from diseased ones offer treatment hope.

doi:10.1038/7251009a

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

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

doi:10.1038/nature08129

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