Fibroblasts as target cells for DEB gene therapy.

Dystrophic epidermolysis bullosa (DEB) is due to mutations in the type VII collagen (C7) gene. Potential therapies for DEB include (i) ex vivo gene therapy and (ii) intradermal injection of gene-corrected DEB fibroblasts, lentiviral vectors expressing C7 or recombinant C7 itself. With regard to molecular engineering, the dermal fibroblast has advantages over epidermal keratinocytes for delivering C7 to DEB patients.