Gene therapeutic strategies for blistering skin diseases

Because of its accessibility, the skin is a good target for gene therapy. However, the barrier function of the skin hampers effective gene insertion into skin cells. Progresses have been made in vector technology and design allowing for successful preclinical studies in skin gene therapy trials using different approaches. Keratinocytes or dermal fibroblasts have been successfully targeted for various applications including the correction of genetic skin disorders, the treatment of systemic diseases or cancer and wound-healing enhancement. We will review the different gene therapy approaches that have been used for the treatment of blistering skin diseases and perspectives in the field provided by new approaches.

Section editor:Mike P. Philpott – Queen Mary School of Medicine and Dentistry, University of London, UK