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1: Blood. 2008 Oct 27. [Epub ahead of print]
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Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells.

Tolar J, Ishida-Yamamoto A, Riddle M, McElmurry RT, Osborn M, Xia L, Lund T, Slattery C, Uitto J, Christiano AM, Wagner JE, Blazar BR.

Blood and Marrow Transplantation Program, University of Minnesota, Minneapolis, MN, United States.

The recessive dystrophic form of epidermolysis bullosa (RDEB) is a disorder of incurable skin fragility and blistering caused by mutations in the type VII collagen gene (COL7A1). The absence of type VII collagen production leads to the loss of adhesion at the basement membrane zone due to the absence of anchoring fibrils, which are comprised of type VII collagen. We report that wild-type, congenic bone marrow cells homed to damaged skin, produced type VII collagen protein and anchoring fibrils, ameliorated skin fragility and reduced lethality in the murine model of RDEB generated by targeted COL7A1 gene disruption. These data provide the first evidence that a population of marrow cells can correct the basement membrane zone defect found in mice with RDEB and offer a potentially valuable approach for treatment of human RDEB and other extra-cellular matrix disorders.

PMID: 18955559 [PubMed - as supplied by publisher]