A protocol describing the genetic correction of somatic human cells and subsequent generation of IPS cell

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dc.contributor.author	Rodríguez Pizà, Ignasi
dc.contributor.author	Raya Chamorro, Ángel
dc.contributor.author	Navarro, Susana
dc.contributor.author	Izpisúa Belmonte, J. C.
dc.contributor.author	Bueren, Juan
dc.contributor.author	Consiglio, Antonella
dc.contributor.author	Sánchez Danés, Adriana, 1984-
dc.contributor.author	Guenechea, Guillermo
dc.contributor.author	Richaud Patin, Yvonne
dc.date	2010
dc.identifier.citation	Raya Á, Rodríguez I, Navarro S, Richaud-Patin Y, Guenechea G, Sánchez-Danés A et al. A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells. Nat Protoc. 2010; 5: 647-60. DOI 10.1038/nprot.2010.9
dc.identifier.citation	1754-2189
dc.identifier.citation	http://dx.doi.org/10.1038/nprot.2010.9
dc.identifier.uri	http://hdl.handle.net/10230/12431
dc.format	application/pdf
dc.language.iso	eng
dc.publisher	Nature Publishing Group
dc.relation	Nature Protocols. 2010; 5(4): 647-660
dc.rights	© Nature Publishing Group
dc.rights	info:eu-repo/semantics/openAccess
dc.subject	Medicina regenerativa
dc.subject	Cél·lules mare embrionàries
dc.title	A protocol describing the genetic correction of somatic human cells and subsequent generation of IPS cell
dc.type	info:eu-repo/semantics/article
dc.type	info:eu-repo/semantics/publishedVersion
dc.description.abstract	The generation of patient-specific induced pluripotent stem cells (iPSCPSCPSCs) offers unprecedented opportunities for modeling and treating human disease. In combination with gene therapy, the iPSCPSCPSC 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 iPSCPSCPSCs 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 iPSCPSCPSCs can be obtained in 4–5 months.

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