title: Bone marrow transplantation in epidermolysis bullosa.
abstract: Epidermolysis bullosa (EB) is a heterogeneous group of inherited blistering skin diseases. Severe forms of EB are associated with increased morbidity and mortality, and there is currently no effective treatment. To combat severe complications of EB, such as chronic erosions, scarring and malignancy, effective therapy needs to be given systemically and at an early age. One recent therapeutic advancement has been a clinical trial of whole bone marrow (BM) transplantation in children with the dystrophic form of EB. This led to correction of the inherent skin basement membrane defect and better skin integrity in some individuals. The challenge now is to precisely identify which BM cells contribute to skin recovery and what mechanisms are involved in tissue regeneration. An improved understanding of the key aspects of BM skin repair is likely to lead to significant health improvements for patients with EB and other skin diseases.
authors.affiliation: St John's Institute of Dermatology, King's College London (Guy's Campus), 9th Floor, Tower Wing, Guy's Hospital, Great Maze Pond, London, SE1 9RT, UK.
authors.forename: Alya
authors.initials: A
authors.lastname: Abdul-Wahab
authors.forename: Gabriela
authors.initials: G
authors.lastname: Petrof
authors.forename: John A
authors.initials: JA
authors.lastname: McGrath
mesh_terms: D000818:Animals; D001485:Basement Membrane / Q000187:drug effects* / Q000473:pathology; D016026:Bone Marrow Transplantation; D002648:Child; D002986:Clinical Trials as Topic; D004820:Epidermolysis Bullosa / Q000628:therapy*; D048091:Guided Tissue Regeneration; D006801:Humans; D012867:Skin / Q000187:drug effects* / Q000473:pathology