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Bone marrow-derived stem cells may offer novel therapeutic option for skin disorder
Date:12/4/2008

s in a mouse model of the disease and offer a potentially valuable approach for treatment of human RDEB and other extracellular matrix disorders. These results provide proof of principle of bone marrow transfer to repair the basement membrane defect in RDEB, and they warrant a clinical trial to assess the safety and efficacy of treatment of human RDEB by means of hematopoietic cell transplantation, said Dr. Tolar.

Research suggests that the systemic infusion of wild-type bone marrow cells could provide benefit to other human disorders of the extracellular matrix. Efforts are underway to identify the requirements of bone marrow-derived stem cells capable of efficiently homing to wounded skin and producing an array of extracellular matrix proteins. As the principal advantage of systemic therapy is its potential to target not only the skin but also the mucosa of the mouth and gastrointestinal tract, the clinical testing of efficacy of human bone marrow for the treatment of human RDEB is underway to determine whether it is of more substantial benefit than local protein, gene, or cellular therapies currently being investigated by other researchers.

An estimated 50 in 1 million live births are diagnosed with EB. The disorder occurs in every racial and ethnic group throughout the world and affects both sexes.


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Contact: Patrick C. Irelan
pirelan@hematology.org
202-292-0253
American Society of Hematology
Source:Eurekalert

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