[HTML][HTML] Injection of genetically engineered fibroblasts corrects regenerated human epidermolysis bullosa skin tissue

S Ortiz-Urda, Q Lin, CL Green… - The Journal of …, 2003 - Am Soc Clin Investig
S Ortiz-Urda, Q Lin, CL Green, DR Keene, MP Marinkovich, PA Khavari
The Journal of clinical investigation, 2003Am Soc Clin Investig
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 …
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.
The Journal of Clinical Investigation