TY - JOUR
T1 - A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells
AU - Raya, Ángel
AU - Rodríguez-Pizá, Ignasi
AU - Navarro, Susana
AU - Richaud-Patin, Yvonne
AU - Guenechea, Guillermo
AU - Sánchez-Danés, Adriana
AU - Consiglio, Antonella
AU - Bueren, Juan
AU - Izpisua Belmonte, Juan Carlos
PY - 2010/4/1
Y1 - 2010/4/1
N2 - The generation of patient-specific induced pluripotent stem cells (iPSCs) offers unprecedented opportunities for modeling and treating human disease. In combination with gene therapy, the iPSC technology can be used to generate disease-free progenitor cells of potential interest for autologous cell therapy. We explain a protocol for the reproducible generation of genetically corrected iPSCs starting from the skin biopsies of Fanconi anemia patients using retroviral transduction with OCT4, SOX2 and KLF4. Before reprogramming, the fibroblasts and/or keratinocytes of the patients are genetically corrected with lentiviruses expressing FANCA. The same approach may be used for other diseases susceptible to gene therapy correction. Genetically corrected, characterized lines of patient-specific iPSCs can be obtained in 4-5 months.
AB - The generation of patient-specific induced pluripotent stem cells (iPSCs) offers unprecedented opportunities for modeling and treating human disease. In combination with gene therapy, the iPSC technology can be used to generate disease-free progenitor cells of potential interest for autologous cell therapy. We explain a protocol for the reproducible generation of genetically corrected iPSCs starting from the skin biopsies of Fanconi anemia patients using retroviral transduction with OCT4, SOX2 and KLF4. Before reprogramming, the fibroblasts and/or keratinocytes of the patients are genetically corrected with lentiviruses expressing FANCA. The same approach may be used for other diseases susceptible to gene therapy correction. Genetically corrected, characterized lines of patient-specific iPSCs can be obtained in 4-5 months.
UR - http://www.scopus.com/inward/record.url?scp=77949427674&partnerID=8YFLogxK
U2 - 10.1038/nprot.2010.9
DO - 10.1038/nprot.2010.9
M3 - Article
C2 - 20224565
AN - SCOPUS:77949427674
VL - 5
SP - 647
EP - 660
JO - Nature Protocols
JF - Nature Protocols
SN - 1754-2189
IS - 4
ER -
