Gene therapy of inherited skin adhesion disorders: a critical overview

M De Luca, G Pellegrini, F Mavilio - British Journal of …, 2009 - academic.oup.com
British Journal of Dermatology, 2009academic.oup.com
Gene therapy has the potential to treat devastating inherited diseases for which there is little
hope of finding a conventional cure. These include lethal diseases, like immunodeficiencies
or several metabolic disorders, or conditions associated with a relatively long life expectancy
but poor quality of life and expensive and life‐long symptomatic treatments, such as
muscular dystrophy, cystic fibrosis and thalassaemia. Skin adhesion defects belong to both
groups. For the nonlethal forms, gene therapy, or transplantation of cultured skin derived …
Summary
Gene therapy has the potential to treat devastating inherited diseases for which there is little hope of finding a conventional cure. These include lethal diseases, like immunodeficiencies or several metabolic disorders, or conditions associated with a relatively long life expectancy but poor quality of life and expensive and life‐long symptomatic treatments, such as muscular dystrophy, cystic fibrosis and thalassaemia. Skin adhesion defects belong to both groups. For the nonlethal 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 epidermolysis bullosa, and the technical and nontechnical factors currently limiting its development.
Oxford University Press