Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells

Jakub Tolar, Ishida Yamamoto Akemi, Megan Riddle, Ron T. McElmurry, Mark Osborn, Lily Xia, Troy Lund, Catherine Slattery, Jouni Uitto, Angela M. Christiano, John E. Wagner, Bruce R. Blazar

Research output: Contribution to journalArticlepeer-review

124 Scopus citations

Abstract

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 (Co17a1). 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 composed 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 Co17a1 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 extracellular matrix disorders.

Original languageEnglish (US)
Pages (from-to)1167-1174
Number of pages8
JournalBlood
Volume113
Issue number5
DOIs
StatePublished - Jan 29 2009

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