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

Ángel Raya, Ignasi Rodríguez-Pizá, Susana Navarro, Yvonne Richaud-Patin, Guillermo Guenechea, Adriana Sánchez-Danés, Antonella Consiglio, Juan Bueren, Juan Carlos Izpisua Belmonte

Research output: Contribution to journalArticlepeer-review

43 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)647-660
Number of pages14
JournalNature Protocols
Volume5
Issue number4
DOIs
StatePublished - Apr 1 2010

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

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