title: Cell-based therapy for RDEB: how does it work?
abstract: No effective or specific treatment is currently available for recessive dystrophic epidermolysis bullosa (RDEB), a severe heritable blistering disorder caused by mutations in the type VII collagen gene (COL7A1). Recent studies have suggested that delivery of allogeneic fibroblasts to the skin of patients with RDEB may be beneficial in improving skin adhesion and increasing type VII collagen deposition at the dermal-epidermal junction. In this issue, Nagy et al. explore mechanisms of fibroblast therapy in a patient with RDEB displaying reduced type VII collagen protein expression at the dermal-epidermal junction. The results suggest that allogeneic fibroblast injection elicits expression of cytokines, including heparin binding-EGF-like growth factor (HB-EGF), that upregulate the expression of a patient-specific COL7A1 allele. Thus, fibroblast therapy may provide a novel way to improve skin therapy in a select subgroup of patients with this currently intractable disease.
authors.affiliation: Department of Dermatology and Cutaneous Biology, Jefferson Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania 19107, USA. jouni.uitto@jefferson.edu
authors.forename: Jouni
authors.initials: J
authors.lastname: Uitto
mesh_terms: D016026:Bone Marrow Transplantation / Q000379:methods*; D017690:Cell Transplantation / Q000379:methods*; D016108:Epidermolysis Bullosa Dystrophica / Q000503:physiopathology / Q000628:therapy*; D005347:Fibroblasts / Q000637:transplantation*; D006801:Humans; D014184:Transplantation, Homologous