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It is an ideal target for gene therapy as all variants of hereditary <jats:styled-content style=\"fixed-case\">RDEB<\/jats:styled-content> are caused by mutations in a single gene, <jats:italic><jats:styled-content style=\"fixed-case\">COL<\/jats:styled-content>7A1<\/jats:italic>, coding for type <jats:styled-content style=\"fixed-case\">VII<\/jats:styled-content> collagen, a key component of anchoring fibrils that secure attachment of the epidermis to the dermis. <jats:styled-content style=\"fixed-case\">RDEB<\/jats:styled-content> is one of the most severe variants in the epidermolysis bullosa (<jats:styled-content style=\"fixed-case\">EB<\/jats:styled-content>) group of heritable skin diseases. Epidermolysis bullosa is defined by chronic fragility and blistering of the skin and mucous membranes due to mutations in the genes responsible for production of the basement membrane proteins. This condition has a high personal, medical and socio\u2010economic impact. People with <jats:styled-content style=\"fixed-case\">RDEB<\/jats:styled-content> require a broad spectrum of medications and specialised care. Due to this being a systemic condition, most research focus is in the area of gene therapy. Recently, preclinical works have begun to show promise. They focus on the virally mediated <jats:italic>ex vivo<\/jats:italic> correction of autologous epithelium. These corrected cells are then to be expanded and grafted onto the patient following the lead of the first successful gene therapy in dermatology being a grafting of corrected tissue for junctional <jats:styled-content style=\"fixed-case\">EB<\/jats:styled-content> treatment. Current progress, outstanding challenges and future directions in translating these approaches in clinics are reviewed in this article.<\/jats:p>","DOI":"10.1111\/exd.12246","type":"journal-article","created":{"date-parts":[[2013,10,11]],"date-time":"2013-10-11T21:55:26Z","timestamp":1381528526000},"page":"1-6","source":"Crossref","is-referenced-by-count":15,"title":["Gene therapy: pursuing restoration of dermal adhesion in recessive dystrophic epidermolysis bullosa"],"prefix":"10.1111","volume":"23","author":[{"given":"Lara","family":"Cutlar","sequence":"first","affiliation":[{"name":"Network of Excellence for Functional Biomaterials National University of Ireland Galway Ireland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Udo","family":"Greiser","sequence":"additional","affiliation":[{"name":"Network of Excellence for Functional Biomaterials National University of Ireland Galway 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