Human urine-derived stem cells rescue cutaneous manifestation and suppress inflammation and fibrosis in vitro and in a mouse model of recessive dystrophic epidermolysis bullosa

Background: Recessive dystrophic epidermolysis bullosa (RDEB) is a life-threatening disease characterized by persistent wound inflammation, tissue fibrosis, and even tumorigenesis in later stages. Despite its significant impact on patient health worldwide, treatment of RDEB has limited options. There is a clear need for now interventions. The goal of this study was to explore the potential efficacy of urine-derived stem cells (USCs) in RDEB. Methods: We isolated human USCs from healthy donors, and assessed their therapeutic effects on RDEB both in vitro with tissue-engineered skin in a three-dimensional co-culture system and in vivo with COL7A1 -/- (RDEB) mice. Results: USCs showed suppressive effects on expression of inflammation- and fibrosis-related genes involved in RDEB in vitro and in vivo. USCs could also extend the median life span (from 3 to 5 days), improve the expression of C7, and migrate to various organs of RDEB mice after intrahepatic administration. Conclusions: In summary, these results suggest the potential effects of USCs on improving the expression of C7 and would repair of RDEB, which supports the future use of USCs for the treatment of RDEB patients.