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Gene therapy

Transgenic stem cells replace skin

Nature volume 551, pages 306–307 (2017)Cite this article

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The treatment of a patient affected by an incurable genetic skin disease demonstrates the efficacy, feasibility and safety of replacing almost the whole skin using genetically corrected stem cells. See Letter p.327

Stem-cell and gene therapies are often considered to be the future of medicine, but there have been many barriers to putting these approaches into practice. Indeed, there are few examples of truly useful human stem-cell therapies1. On page 327, Hirsch et al.2 describe a success in this area — the use of gene therapy to correct the cells of a child who had a devastating genetic disease associated with skin blistering.

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Figure 1: Gene therapy to treat a skin disease.

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References

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Authors and Affiliations

  1. the Laboratory of Stem Cells and Cancer, Université Libre de Bruxelles, Brussels, 1070, Belgium

    Mariaceleste Aragona & Cédric Blanpain

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  1. Mariaceleste Aragona
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  2. Cédric Blanpain
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Correspondence to Cédric Blanpain.

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Gene therapy: Repair and replace

Stem cells: Skin regeneration and repair

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Aragona, M., Blanpain, C. Transgenic stem cells replace skin. Nature 551, 306–307 (2017). https://doi.org/10.1038/nature24753

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