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.

Gene therapy of skin adhesion disorders (mini review) / Cavazza, Alessia; Mavilio, Fulvio. - In: CURRENT PHARMACEUTICAL BIOTECHNOLOGY. - ISSN 1873-4316. - 13:(2012), pp. 1868-1876. [10.2174/138920112802273119]

Gene therapy of skin adhesion disorders (mini review)

CAVAZZA, ALESSIA;MAVILIO, Fulvio
2012-01-01

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.
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Gene therapy of skin adhesion disorders (mini review) / Cavazza, Alessia; Mavilio, Fulvio. - In: CURRENT PHARMACEUTICAL BIOTECHNOLOGY. - ISSN 1873-4316. - 13:(2012), pp. 1868-1876. [10.2174/138920112802273119]
Cavazza, Alessia; Mavilio, Fulvio
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11380/1074774
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