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

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...

Descripción completa

Detalles Bibliográficos
Autores: Rodríguez Pizà, Ignasi, Verma, Inder M., Veiga, Anna, Aasen, Trond, Izpisúa Belmonte, J. C., Bueren, Juan, Garreta Bahima, Elena, Tiscornia, Gustavo, Sleep Ronquillo, Eduard, Raya Chamorro, Ángel, Río, Paula, Consiglio, Antonella, Barrero Núñez, María José, Navarro, Susanna, Vassena, Rita, Guenechea, Guillermo
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2009
País:España
Institución:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Repositorio:Recercat. Dipósit de la Recerca de Catalunya
OAI Identifier:oai:recercat.cat:10230/12426
Acceso en línea:http://hdl.handle.net/10230/12426
http://dx.doi.org/10.1038/nature08129
Access Level:acceso abierto
Palabra clave:Medicina regenerativa
Cél·lules mare embrionàries
Descripción
Sumario: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.

Similares en LA Referencia