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

doi:10.1038/nprot.2010.9

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

Biological, Environmental Science and Engineering

Research output: Contribution to journal › Article › peer-review

41 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 language	English (US)
Pages (from-to)	647-660
Number of pages	14
Journal	Nature Protocols
Volume	5
Issue number	4
DOIs	https://doi.org/10.1038/nprot.2010.9
State	Published - Apr 1 2010

ASJC Scopus subject areas

Biochemistry, Genetics and Molecular Biology(all)

UN SDGs

This output contributes to the following Sustainable Development Goal(s)

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10.1038/nprot.2010.9

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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.",

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A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells. / Raya, Ángel; Rodríguez-Pizá, Ignasi; Navarro, Susana; Richaud-Patin, Yvonne; Guenechea, Guillermo; Sánchez-Danés, Adriana; Consiglio, Antonella; Bueren, Juan; Izpisua Belmonte, Juan Carlos.

In: Nature Protocols, Vol. 5, No. 4, 01.04.2010, p. 647-660.

Research output: Contribution to journal › Article › peer-review

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AU - Richaud-Patin, Yvonne

AU - Guenechea, Guillermo

AU - Sánchez-Danés, Adriana

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AU - Izpisua Belmonte, Juan Carlos

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

Abstract

ASJC Scopus subject areas

UN SDGs

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