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

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

N Engl J Med

From the Division of Plastic Surgery, Department of Trauma, Hand and Reconstructive Surgery, University Hospital Muenster, and the Department of Plastic, Reconstructive and Aesthetic Surgery, Hand Surgery, Fachklinik Hornheide (M.K., B.Y., T.H.), and the Department of Dermatology, University of Muenster (D.M.), Muenster, the Department of Neonatology and Pediatric Intensive Care (T.R.) and the Department of Neuropediatrics (L.E.), University Children's Hospital, Ruhr-University Bochum (C.M.), Bochum, and the Research Department, BG Klinikum Duisburg, Duisburg (T.O.) - all in Germany; and Holostem Terapie Avanzate, Center for Regenerative Medicine Stefano Ferrari (L.D.R., A.S.S.), and the Unit of Pathology (L.L.) and the Center for Regenerative Medicine Stefano Ferrari (M.D.L.), Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy.

Published: December 2021

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.).

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Source
http://dx.doi.org/10.1056/NEJMoa2108544DOI Listing

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