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.)

Transgenic epidermal cultures for junctional epidermolysis bullosa — 5-year outcomes / Kueckelhaus, M.; Rothoeft, T.; de Rosa, L.; Yeni, B.; Ohmann, T.; Maier, C.; Eitner, L.; Metze, D.; Losi, L.; Seconetti, A. S.; de Luca, M.; Hirsch, T.. - In: THE NEW ENGLAND JOURNAL OF MEDICINE. - ISSN 0028-4793. - 385:24(2021), pp. 2264-2270. [10.1056/NEJMoa2108544]

Transgenic epidermal cultures for junctional epidermolysis bullosa — 5-year outcomes

de Rosa L.;Maier C.;Losi L.;de Luca M.;
2021

Abstract

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.)
2021
385
24
2264
2270
Transgenic epidermal cultures for junctional epidermolysis bullosa — 5-year outcomes / Kueckelhaus, M.; Rothoeft, T.; de Rosa, L.; Yeni, B.; Ohmann, T.; Maier, C.; Eitner, L.; Metze, D.; Losi, L.; Seconetti, A. S.; de Luca, M.; Hirsch, T.. - In: THE NEW ENGLAND JOURNAL OF MEDICINE. - ISSN 0028-4793. - 385:24(2021), pp. 2264-2270. [10.1056/NEJMoa2108544]
Kueckelhaus, M.; Rothoeft, T.; de Rosa, L.; Yeni, B.; Ohmann, T.; Maier, C.; Eitner, L.; Metze, D.; Losi, L.; Seconetti, A. S.; de Luca, M.; Hirsch, T.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11380/1264289
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