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.
Chen
Mei
M
Department of Dermatology, Keck School of Medicine, University of Southern California, Los Angeles, California 90033, USA.
Woodley
David T
DT
D000818:Animals; D024143:Collagen Type VII / Q000235:genetics*; D016108:Epidermolysis Bullosa Dystrophica / Q000628:therapy*; D005347:Fibroblasts / Q000637:transplantation*; D015316:Genetic Therapy; D051379:Mice
D024143:Collagen Type VII