Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells.
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.
Raya
Angel
A
Center for Regenerative Medicine in Barcelona, Dr. Aiguader 88, 08003 Barcelona, Spain.
Rodríguez-Pizà
Ignasi
I
Guenechea
Guillermo
G
Vassena
Rita
R
Navarro
Susana
S
Barrero
María José
MJ
Consiglio
Antonella
A
Castellà
Maria
M
Río
Paula
P
Sleep
Eduard
E
González
Federico
F
Tiscornia
Gustavo
G
Garreta
Elena
E
Aasen
Trond
T
Veiga
Anna
A
Verma
Inder M
IM
Surrallés
Jordi
J
Bueren
Juan
J
Izpisúa Belmonte
Juan Carlos
JC
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