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

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 grea...

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Autores: Raya, Ángel|||0000-0003-2189-9775, Rodríguez-Pizà, Ignasi, Guenechea, Guillermo, Vassena, Rita|||0000-0002-0846-0365, Navarro Ordóñez, Susana|||0000-0002-0764-5384, Barrero, María José, Consiglio, Antonella, Castellà, Maria, Río, Paula, Sleep, Eduard, González, Federico, Tiscornia, Gustavo, Garreta, Elena|||0000-0002-1071-9794, Aasen, Trond|||0000-0003-0763-2695, Veiga, Anna|||0000-0002-0943-9904, Verma, Inder M., Surralles, Jordi|||0000-0002-4041-7519, Bueren, Juan|||0000-0002-3228-7013, Izpisúa Belmonte, Juan Carlos
Formato: artículo
Fecha de publicación:2009
País:España
Recursos:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:68792
Acesso em linha:https://ddd.uab.cat/record/68792
https://dx.doi.org/urn:doi:10.1038/nature08129
Access Level:acceso abierto
Palavra-chave:Fanconi anemia (FA)
PREI 2010
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spelling Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cellsGeneration of disease-free haematopoietic progenitors from Fanconi anemia-specific induced pluripotent stem cellsRaya, Ángel|||0000-0003-2189-9775Rodríguez-Pizà, IgnasiGuenechea, GuillermoVassena, Rita|||0000-0002-0846-0365Navarro Ordóñez, Susana|||0000-0002-0764-5384Barrero, María JoséConsiglio, AntonellaCastellà, MariaRío, PaulaSleep, EduardGonzález, FedericoTiscornia, GustavoGarreta, Elena|||0000-0002-1071-9794Aasen, Trond|||0000-0003-0763-2695Veiga, Anna|||0000-0002-0943-9904Verma, Inder M.Surralles, Jordi|||0000-0002-4041-7519Bueren, Juan|||0000-0002-3228-7013Izpisúa Belmonte, Juan CarlosFanconi anemia (FA)PREI 2010The 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. 22009-01-0120092009-01-01Articlehttp://purl.org/coar/resource_type/c_6501AMhttp://purl.org/coar/version/c_ab4af688f83e57aainfo:eu-repo/semantics/articleapplication/pdfhttps://ddd.uab.cat/record/68792https://dx.doi.org/urn:doi:10.1038/nature08129reponame:Dipòsit Digital de Documents de la UABinstname:Universitat Autònoma de BarcelonaInglésengopen accesshttp://purl.org/coar/access_right/c_abf2Aquest material està protegit per drets d'autor i/o drets afins. Podeu utilitzar aquest material en funció del que permet la legislació de drets d'autor i drets afins d'aplicació al vostre cas. Per a d'altres usos heu d'obtenir permís del(s) titular(s) de drets.https://rightsstatements.org/vocab/InC/1.0/info:eu-repo/semantics/openAccessoai:ddd.uab.cat:687922026-06-06T12:50:31Z
dc.title.none.fl_str_mv Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
Generation of disease-free haematopoietic progenitors from Fanconi anemia-specific 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
Raya, Ángel|||0000-0003-2189-9775
Fanconi anemia (FA)
PREI 2010
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 Raya, Ángel|||0000-0003-2189-9775
Rodríguez-Pizà, Ignasi
Guenechea, Guillermo
Vassena, Rita|||0000-0002-0846-0365
Navarro Ordóñez, Susana|||0000-0002-0764-5384
Barrero, María José
Consiglio, Antonella
Castellà, Maria
Río, Paula
Sleep, Eduard
González, Federico
Tiscornia, Gustavo
Garreta, Elena|||0000-0002-1071-9794
Aasen, Trond|||0000-0003-0763-2695
Veiga, Anna|||0000-0002-0943-9904
Verma, Inder M.
Surralles, Jordi|||0000-0002-4041-7519
Bueren, Juan|||0000-0002-3228-7013
Izpisúa Belmonte, Juan Carlos
author Raya, Ángel|||0000-0003-2189-9775
author_facet Raya, Ángel|||0000-0003-2189-9775
Rodríguez-Pizà, Ignasi
Guenechea, Guillermo
Vassena, Rita|||0000-0002-0846-0365
Navarro Ordóñez, Susana|||0000-0002-0764-5384
Barrero, María José
Consiglio, Antonella
Castellà, Maria
Río, Paula
Sleep, Eduard
González, Federico
Tiscornia, Gustavo
Garreta, Elena|||0000-0002-1071-9794
Aasen, Trond|||0000-0003-0763-2695
Veiga, Anna|||0000-0002-0943-9904
Verma, Inder M.
Surralles, Jordi|||0000-0002-4041-7519
Bueren, Juan|||0000-0002-3228-7013
Izpisúa Belmonte, Juan Carlos
author_role author
author2 Rodríguez-Pizà, Ignasi
Guenechea, Guillermo
Vassena, Rita|||0000-0002-0846-0365
Navarro Ordóñez, Susana|||0000-0002-0764-5384
Barrero, María José
Consiglio, Antonella
Castellà, Maria
Río, Paula
Sleep, Eduard
González, Federico
Tiscornia, Gustavo
Garreta, Elena|||0000-0002-1071-9794
Aasen, Trond|||0000-0003-0763-2695
Veiga, Anna|||0000-0002-0943-9904
Verma, Inder M.
Surralles, Jordi|||0000-0002-4041-7519
Bueren, Juan|||0000-0002-3228-7013
Izpisúa Belmonte, Juan Carlos
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv Fanconi anemia (FA)
PREI 2010
topic Fanconi anemia (FA)
PREI 2010
description 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.
publishDate 2009
dc.date.none.fl_str_mv 2
2009-01-01
2009
2009-01-01
dc.type.none.fl_str_mv Article
http://purl.org/coar/resource_type/c_6501
AM
http://purl.org/coar/version/c_ab4af688f83e57aa
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://ddd.uab.cat/record/68792
https://dx.doi.org/urn:doi:10.1038/nature08129
url https://ddd.uab.cat/record/68792
https://dx.doi.org/urn:doi:10.1038/nature08129
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://rightsstatements.org/vocab/InC/1.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://rightsstatements.org/vocab/InC/1.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Dipòsit Digital de Documents de la UAB
instname:Universitat Autònoma de Barcelona
instname_str Universitat Autònoma de Barcelona
reponame_str Dipòsit Digital de Documents de la UAB
collection Dipòsit Digital de Documents de la UAB
repository.name.fl_str_mv
repository.mail.fl_str_mv
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