Transgenic skin biomechanical properties following first lifesaving epidermal regeneration using combined gene and cell therapy.

BACKGROUND: In 2017, we reported the first life-saving regeneration of virtually an entire epidermis by combined gene and stem cell therapy. Recently, we demonstrated excellent long-term stability of this transgenic epidermis. Skin quality in this experimental approach and its potential application in other conditions were elucidated here regarding long-term outcomes of biomechanical properties. PATIENTS AND METHODS: Analysis of biomechanical properties including skin elasticity, anisotropy and friction was performed on multiple body sites 24, 36 and 60 months following transplantation. Firstly, the sites were matched against and compared to remaining stable non-transgenic areas as well as to a control group of 13 healthy subjects. Parameters for skin elasticity, deformation and friction were assessed non-invasively. RESULTS: Biomechanical properties of the transgenic epidermis showed encouraging results in comparison to both the remaining stable non-transgenic skin as well as healthy controls. Skin elasticity was comparable to the controls. Skin friction showed some decrease in both transgenic and non-transgenic areas as compared to the controls. CONCLUSIONS: The excellent functional outcomes of the transgenic epidermis demonstrate stable long-term results of this novel combined gene and stem cell therapy for epidermal regeneration. Thus, other applications for this technology, such as treatment of specific burns, should be explored.

Transgenic Epidermal Cultures for Junctional Epidermolysis Bullosa - 5-Year Outcomes.

Inherited junctional epidermolysis bullosa is a severe genetic skin disease that leads to epidermal loss caused by structural and mechanical fragility of the integuments. There is no established cure for junctional epidermolysis bullosa. We previously reported that genetically corrected autologous epidermal cultures regenerated almost an entire, fully functional epidermis on a child who had a devastating form of junctional epidermolysis bullosa. We now report long-term clinical outcomes in this patient. (Funded by POR FESR 2014-2020 - Regione Emilia-Romagna and others.).