The joint battle to tackle epidermolysis bullosa through gene therapy

Abstract

Efforts are dedicated to definitively tackle skin lesions plaguing patients with epidermolysis bullosa (EB), a devastating genetic disorder affecting the integumentary system. Both in vivo gene therapy, as recently reported by Gurevich et al., and combined ex vivo cell and gene therapy strategies are under investigation. Here, we address the advantages and disadvantages of these different approaches.

Keywords: cell therapy; epidermal stem cells; epidermolysis bullosa; gene therapy; regenerative medicine.