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J Clin Invest. 2003 Jan;111(2):251-5.

Injection of genetically engineered fibroblasts corrects regenerated human epidermolysis bullosa skin tissue.

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1
Veterans Affairs Palo Alto Healthcare System, Palo Alto, California, USA.

Abstract

Current therapeutic strategies for genetic skin disorders rely on the complex process of grafting genetically engineered tissue to recipient wound beds. Because fibroblasts synthesize and secrete extracellular matrix, we explored their utility in recessive dystrophic epidermolysis bullosa (RDEB), a blistering disease due to defective extracellular type VII collagen. Intradermal injection of RDEB fibroblasts overexpressing type VII collagen into intact RDEB skin stably restored correctly localized type VII collagen expression in vivo and normalized hallmark RDEB disease features, including subepidermal blistering and anchoring fibril defects. This article was published online in advance of the print edition. The date of publication is available from the JCI website, http://www.jci.org.

PMID:
12531881
PMCID:
PMC151880
DOI:
10.1172/JCI17193
[Indexed for MEDLINE]
Free PMC Article
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