Regeneration of the entire human epidermis using transgenic stem cells

November 6th, 2017 Posted by Journal Articles 0 comments on “Regeneration of the entire human epidermis using transgenic stem cells”

Junctional epidermolysis bullosa (JEB) is a severe and often lethal genetic disease caused by mutations in genes encoding the basement membrane component laminin-332.

Surviving patients with JEB develop chronic wounds to the skin and mucosa, which impair their quality of life and lead to skin cancer. Here we show that autologous transgenic keratinocyte cultures regenerated an entire, fully functional epidermis on a seven-year-old child suffering from a devastating, lifethreatening form of JEB.

The proviral integration pattern was maintained in vivo and epidermal renewal did not cause any clonal selection. Clonal tracing showed that the human epidermis is sustained not by equipotent progenitors, but by a limited number of long-lived stem cells, detected as holoclones, that can extensively self-renew in vitro and in vivo and produce progenitors that replenish terminally differentiated keratinocytes.

This study provides a blueprint that can be applied to other stem cell-mediated combined ex vivo cell and gene therapies.


© 2017 Macmillan Publishers Limited, part of Springer Nature. All rights reserved.


Title: Regeneration of the entire human epidermis using transgenic stem cells

Authors: tobias hirsch, tobias Rothoeft, norbert teig, Johann W. Bauer, Graziella Pellegrini, Laura De Rosa, Davide Scaglione, Julia Reichelt, Alfred Klausegger, Daniela Kneisz, oriana Romano, Alessia Secone Seconetti, Roberta contin, Elena Enzo, irena Jurman, Sonia carulli, Frank Jacobsen, thomas Luecke, Marcus Lehnhardt, Meike Fischer, Maximilian Kueckelhaus, Daniela Quaglino, Michele Morgante, Silvio Bicciato, Sergio Bondanza & Michele De Luca.
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