Gene therapy of inherited skin adhesion disorders

Gene therapy is a potential treatment for devastating inherited diseases for which there is little hope of finding a conventional cure. These include lethal diseases like immunodeficiencies and metabolic disorders, and non-lethal conditions associated with poor quality of life and life-long symptomatic treatments, like muscular dystrophy, cystic fibrosis or thalassemia. Skin adhesion defects belong to both groups. For the non-lethal forms, gene therapy, or transplantation of cultured skin derived from genetically corrected epidermal stem cells, represents a very attractive therapeutic option, and potentially a definitive treatment. Recent advances in gene transfer and stem cell culture technology are making this option closer than ever. This paper critically reviews the progress and prospects of gene therapy for skin adhesion defects, and the technical and non-technical factors currently limiting its development.

Section editors:Frederick Kaplan – The University of Pennsylvania School of Medicine, Department of Orthopaedic Surgery, Philadelphia, PA 19104, USAAmber Salzman – Cardiokine, Inc., Philadelphia, PA 19102, USA