title: Injection of genetically engineered fibroblasts corrects regenerated human epidermolysis bullosa skin tissue.
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.
authors.affiliation: Veterans Affairs Palo Alto Healthcare System, Palo Alto, California, USA.
authors.forename: Susana
authors.initials: S
authors.lastname: Ortiz-Urda
authors.forename: Qun
authors.initials: Q
authors.lastname: Lin
authors.forename: Cheryl L
authors.initials: CL
authors.lastname: Green
authors.forename: Douglas R
authors.initials: DR
authors.lastname: Keene
authors.forename: M Peter
authors.initials: MP
authors.lastname: Marinkovich
authors.forename: Paul A
authors.initials: PA
authors.lastname: Khavari
mesh_terms: D000818:Animals; D024143:Collagen Type VII / Q000235:genetics*; D016108:Epidermolysis Bullosa Dystrophica / Q000628:therapy*; D005347:Fibroblasts / Q000378:metabolism*; D005818:Genetic Engineering; D015316:Genetic Therapy; D006801:Humans; D007267:Injections; D051379:Mice; D016513:Mice, SCID
chemical_list: D024143:Collagen Type VII