title: Gene therapy of skin adhesion disorders (mini review).
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.
authors.affiliation: Center for Regenerative Medicine, University of Modena and Reggio Emilia, Via Gottardi 100, 41125 Modena, Italy.
authors.forename: Alessia
authors.initials: A
authors.lastname: Cavazza
authors.forename: Fulvio
authors.initials: F
authors.lastname: Mavilio
mesh_terms: D002986:Clinical Trials as Topic; D004820:Epidermolysis Bullosa / Q000235:genetics / Q000628:therapy*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D016038:Skin Transplantation / Q000379:methods*; D016896:Treatment Outcome