Abstract
Gene therapy has been suggested as a potential cure for patients with disabling genetic diseases. The possibility of using gene transfer to treat diseases of the skin was suggested nearly 20 years ago by a report demonstrating that human keratinocyte stem cells1 could be genetically manipulated ex vivo using recombinant retroviruses.2 These early studies led to painstaking efforts by many groups, such as those led by De Luca, to develop gene transfer strategies with clinical potential to treat skin disease.3 The successful transplantation of keratinocyte stem cells genetically corrected ex vivo to treat a rare form of junctional epidermolysis bullosa (JEB) by Mavilio et al.4 unambiguously demonstrates the feasibility of gene therapy in the skin. The new work lends credence to the opinion that gene therapy may soon be part of our therapeutic arsenal and that it may eventually allow us to improve the lives of patients suffering from this horrendous skin disease.
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