Transgenic Epidermal Cultures for Junctional Epidermolysis Bullosa — 5-Year Outcomes | Zendy

Maximilian Kueckelhaus | Zendy; Tobias Rothoeft | Zendy; Laura De Rosa | Zendy; Burcu Yeni | Zendy; Tobias Ohmann | Zendy; Christoph Maier | Zendy; Lynn Eitner | Zendy; Dieter Metze | Zendy; Lorena Losi | Zendy; Alessia Secone Seconetti | Zendy; Michele De Luca | Zendy; Tobias Hirsch | Zendy

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

Author(s) -

Maximilian Kueckelhaus,

Tobias Rothoeft,

Laura De Rosa,

Burcu Yeni,

Tobias Ohmann,

Christoph Maier,

Lynn Eitner,

Dieter Metze,

Lorena Losi,

Alessia Secone Seconetti,

Michele De Luca,

Tobias Hirsch

Publication year - 2021

Publication title -

new england journal of medicine

Language(s) - Uncategorized

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa2108544

Subject(s) - junctional epidermolysis bullosa (veterinary medicine) , epidermolysis bullosa , medicine , dermatology , epidermis (zoology) , genetics , mutation , anatomy , biology , gene

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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