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...

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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
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spelling Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cellsRodríguez Pizà, IgnasiVerma, Inder M.Veiga, AnnaAasen, TrondIzpisúa Belmonte, J. C.Bueren, JuanGarreta Bahima, ElenaTiscornia, GustavoSleep Ronquillo, EduardRaya Chamorro, ÁngelRío, PaulaConsiglio, AntonellaBarrero Núñez, María JoséNavarro, SusannaVassena, RitaGuenechea, GuillermoMedicina regenerativaCél·lules mare embrionàriesThe 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.Nature Publishing Group201120112009info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfapplication/pdfhttp://hdl.handle.net/10230/12426http://dx.doi.org/10.1038/nature08129reponame:Recercat. Dipósit de la Recerca de Catalunyainstname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)InglésNature. 2009;460(7251):53-9© Nature Publishing Groupinfo:eu-repo/semantics/openAccessoai:recercat.cat:10230/124262026-05-29T05:05:01Z
dc.title.none.fl_str_mv Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
title Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
spellingShingle Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
Rodríguez Pizà, Ignasi
Medicina regenerativa
Cél·lules mare embrionàries
title_short Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
title_full Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
title_fullStr Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
title_full_unstemmed Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
title_sort Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
dc.creator.none.fl_str_mv 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
author Rodríguez Pizà, Ignasi
author_facet 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
author_role author
author2 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
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv Medicina regenerativa
Cél·lules mare embrionàries
topic Medicina regenerativa
Cél·lules mare embrionàries
description 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.
publishDate 2009
dc.date.none.fl_str_mv 2009
2011
2011
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/10230/12426
http://dx.doi.org/10.1038/nature08129
url http://hdl.handle.net/10230/12426
http://dx.doi.org/10.1038/nature08129
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Nature. 2009;460(7251):53-9
dc.rights.none.fl_str_mv © Nature Publishing Group
info:eu-repo/semantics/openAccess
rights_invalid_str_mv © Nature Publishing Group
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
application/pdf
dc.publisher.none.fl_str_mv Nature Publishing Group
publisher.none.fl_str_mv Nature Publishing Group
dc.source.none.fl_str_mv reponame:Recercat. Dipósit de la Recerca de Catalunya
instname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
instname_str Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
reponame_str Recercat. Dipósit de la Recerca de Catalunya
collection Recercat. Dipósit de la Recerca de Catalunya
repository.name.fl_str_mv
repository.mail.fl_str_mv
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