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As some wounds in <jats:styled-content style=\"fixed-case\">EB<\/jats:styled-content> individuals are difficult or impossible to access with topical therapy, we examined the potential of systemic therapy with normal haematopoietic stem cells. In both animal models and children with <jats:styled-content style=\"fixed-case\">EB<\/jats:styled-content>, healthy donor cells from the haematopoietic graft migrated to the injured skin; simultaneously, there was an increase in the production of skin\u2010specific structural proteins deficient in <jats:styled-content style=\"fixed-case\">EB<\/jats:styled-content>, increased skin integrity and reduced tendency to blister formation. Even though the majority of evaluable individuals have had a positive response in skin healing, frequently changing their quality of life, the improvement in lifestyle has been varied and the overall clinical response incomplete. To change the current amelioration of disease into a full cure, we propose to (i) increase safety as well as efficacy of haematopoietic cell transplant (<jats:styled-content style=\"fixed-case\">HCT<\/jats:styled-content>) using co\u2010infusion of mesenchymal stromal\/stem cells with haematopoietic stem cells and non\u2010myeloablative conditioning for transplant; (ii) optimize homing of donor cells into the skin erosions in animal models of <jats:styled-content style=\"fixed-case\">EB<\/jats:styled-content>; and (iii) discover and test new drugs for <jats:styled-content style=\"fixed-case\">EB<\/jats:styled-content> therapy using patient\u2010specific induced pluripotent stem cells. We conclude that although <jats:styled-content style=\"fixed-case\">HCT<\/jats:styled-content> has always been a risky treatment restricted to those with serious life\u2010threatening or debilitating diseases, by most benchmarks, the results of <jats:styled-content style=\"fixed-case\">HCT<\/jats:styled-content> in <jats:styled-content style=\"fixed-case\">EB<\/jats:styled-content> have shown that <jats:styled-content style=\"fixed-case\">HCT<\/jats:styled-content> has the potential of being a durable, systemic therapy for people with severe forms of <jats:styled-content style=\"fixed-case\">EB<\/jats:styled-content>.<\/jats:p>","DOI":"10.1111\/exd.12014","type":"journal-article","created":{"date-parts":[[2012,8,21]],"date-time":"2012-08-21T11:21:11Z","timestamp":1345548071000},"page":"896-900","source":"Crossref","is-referenced-by-count":18,"title":["Management of severe epidermolysis bullosa by haematopoietic transplant: principles, perspectives and 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