Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
Raya, Ángel (Networking Center of Biomedical Research in Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN))
Rodríguez-Pizà, Ignasi (Center for Regenerative Medicine in Barcelona)
Guenechea, Guillermo (Hematopoiesis and Gene Therapy Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT))
Vassena, Rita (Center for Regenerative Medicine in Barcelona)
Navarro, Susana (Networking Center of Biomedical Research in Rare Diseases (CIBERER))
Barrero, María José (Center for Regenerative Medicine in Barcelona)
Consiglio, Antonella (Center for Regenerative Medicine in Barcelona)
Castellà, Maria (Universitat Autònoma de Barcelona. Department of Genetics and Microbiology)
Río, Paula (Networking Center of Biomedical Research in Rare Diseases (CIBERER))
Sleep, Eduard (Networking Center of Biomedical Research in Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN))
González, Federico (Center for Regenerative Medicine in Barcelona)
Tiscornia, Gustavo (Center for Regenerative Medicine in Barcelona)
Garreta, Elena (Center for Regenerative Medicine in Barcelona)
Aasen, Trond (Center for Regenerative Medicine in Barcelona)
Veiga, Anna (Center for Regenerative Medicine in Barcelona)
Verma, Inder M. (Laboratory of Genetics, Salk Institute for Biological Studies)
Surrallés i Calonge, Jordi (Networking Center of Biomedical Research in Rare Diseases (CIBERER))
Bueren, Juan (Networking Center of Biomedical Research in Rare Diseases (CIBERER))
Izpisúa Belmonte, Juan Carlos (Center for Regenerative Medicine in Barcelona)

Data:	2009
Resum:	The generation of induced pluripotent stem (iPS) cells by ectopic expression of a defined set of factors1-5 has enabled the derivation of patient-specific pluripotent cells and provided valuable experimental platforms to model human disease6-8. Patientspecific iPS cells are also thought to hold great therapeutic potential, although direct evidence for this is still lacking. Here we show that somatic cells from Fanconi anemia (FA) patients, upon correction of the genetic defect, 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 in colony morphology, growth properties, expression of pluripotencyassociated transcription factors and surface markers, and differentiation potential in vitro and in vivo. Most importantly, we show that corrected FA-specific iPS cells can give rise to hematopoietic progenitors of the myeloid and erythroid lineages that are phenotypically normal, i. e. disease-free. These data offer proof-f-concept that iPS cell technology can be used for the generation of disease-corrected, patient-specific cells with potential value for cell therapy applications.
Nota:	Premi a l'excel·lència investigadora. Àmbit de les Ciències de la Salut. 2010
Títol variant:	Títol del post-print: Generation of disease-free haematopoietic progenitors from Fanconi anemia-specific induced pluripotent stem cells
Drets:	Tots els drets reservats
Forma:	article ; acceptedVersion
Matèria:	Fanconi anemia (FA) ; PREI 2010
Publicat a:	Nature, Núm. 460 (05 2009) , p. 53-59, ISSN 1476-4687

DOI: 10.1038/nature08129

Post-print 39 p, 2.8 MB

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