Disease-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 and Juan Carlos Izpisúa Belmonte ()
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Ángel Raya: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Ignasi Rodríguez-Pizà: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Guillermo Guenechea: Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Av. Complutense 22, 28040 Madrid, Spain
Rita Vassena: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Susana Navarro: Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Av. Complutense 22, 28040 Madrid, Spain
María José Barrero: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Antonella Consiglio: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Maria Castellà: Networking Center of Biomedical Research in Rare Diseases (CIBERER),
Paula Río: Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Av. Complutense 22, 28040 Madrid, Spain
Eduard Sleep: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Federico González: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Gustavo Tiscornia: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Elena Garreta: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Trond Aasen: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Anna Veiga: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain
Inder M. Verma: Laboratory of Genetics,
Jordi Surrallés: Networking Center of Biomedical Research in Rare Diseases (CIBERER),
Juan Bueren: Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Av. Complutense 22, 28040 Madrid, Spain
Juan Carlos Izpisúa Belmonte: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain

Nature, 2009, vol. 460, issue 7251, 53-59

Abstract: 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.

Date: 2009
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DOI: 10.1038/nature08129

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