Gene Therapy of Skin Adhesion Disorders (Mini Review)

Alessia      Cavazza; Fulvio      Mavilio

Abstract

Gene therapy is a potential treatment for severe inherited disorders 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 to 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 factors currently limiting its development.

Keywords: Gene therapy, stem cells, epidermolysis bullosa, genetic diseases, retroviral vectors, immunodeficiencies, metabolic disorders, symptomatic treatments, muscular dystrophy, cystic fibrosis, thalassemia, epidermal stem cells, skin diseases, epidermal cells.