title: Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells.
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.
authors.affiliation: Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain.
authors.forename: Angel
authors.initials: A
authors.lastname: Raya
authors.forename: Ignasi
authors.initials: I
authors.lastname: Rodríguez-Pizà
authors.forename: Guillermo
authors.initials: G
authors.lastname: Guenechea
authors.forename: Rita
authors.initials: R
authors.lastname: Vassena
authors.forename: Susana
authors.initials: S
authors.lastname: Navarro
authors.forename: María José
authors.initials: MJ
authors.lastname: Barrero
authors.forename: Antonella
authors.initials: A
authors.lastname: Consiglio
authors.forename: Maria
authors.initials: M
authors.lastname: Castellà
authors.forename: Paula
authors.initials: P
authors.lastname: Río
authors.forename: Eduard
authors.initials: E
authors.lastname: Sleep
authors.forename: Federico
authors.initials: F
authors.lastname: González
authors.forename: Gustavo
authors.initials: G
authors.lastname: Tiscornia
authors.forename: Elena
authors.initials: E
authors.lastname: Garreta
authors.forename: Trond
authors.initials: T
authors.lastname: Aasen
authors.forename: Anna
authors.initials: A
authors.lastname: Veiga
authors.forename: Inder M
authors.initials: IM
authors.lastname: Verma
authors.forename: Jordi
authors.initials: J
authors.lastname: Surrallés
authors.forename: Juan
authors.initials: J
authors.lastname: Bueren
authors.forename: Juan Carlos
authors.initials: JC
authors.lastname: Izpisúa Belmonte
mesh_terms: D002460:Cell Line; D065150:Cellular Reprogramming; D005199:Fanconi Anemia / Q000473:pathology* / Q000628:therapy*; D006262:Health; D006412:Hematopoietic Stem Cells / Q000166:cytology* / Q000378:metabolism; D006801:Humans; D039904:Pluripotent Stem Cells / Q000166:cytology* / Q000378:metabolism