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Transgenic Epidermal Cultures for Junctional Epidermolysis Bullosa - 5-Year Outcomes.
Kueckelhaus, Maximilian; Rothoeft, Tobias; De Rosa, Laura; Yeni, Burcu; Ohmann, Tobias; Maier, Christoph; Eitner, Lynn; Metze, Dieter; Losi, Lorena; Secone Seconetti, Alessia; De Luca, Michele; Hirsch, Tobias.
Afiliação
  • Kueckelhaus M; 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 Muen
  • Rothoeft T; 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 Muen
  • De Rosa L; 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 Muen
  • Yeni B; 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 Muen
  • Ohmann T; 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 Muen
  • Maier C; 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 Muen
  • Eitner L; 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 Muen
  • Metze D; 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 Muen
  • Losi L; 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 Muen
  • Secone Seconetti A; 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 Muen
  • De Luca M; 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 Muen
  • Hirsch T; 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 Muen
N Engl J Med ; 385(24): 2264-2270, 2021 12 09.
Article em En | MEDLINE | ID: mdl-34881838
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.).
Assuntos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transdução Genética / Queratinócitos / Epidermólise Bolhosa Juncional / Transgenes / Epiderme Tipo de estudo: Observational_studies / Prognostic_studies Limite: Child / Humans / Male Idioma: En Revista: N Engl J Med Ano de publicação: 2021 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transdução Genética / Queratinócitos / Epidermólise Bolhosa Juncional / Transgenes / Epiderme Tipo de estudo: Observational_studies / Prognostic_studies Limite: Child / Humans / Male Idioma: En Revista: N Engl J Med Ano de publicação: 2021 Tipo de documento: Article