Transgenic Epidermal Cultures for Junctional Epidermolysis Bullosa - 5-Year Outcomes.
N Engl J Med
; 385(24): 2264-2270, 2021 12 09.
Article
en 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.).
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
Transducción Genética
/
Queratinocitos
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Epidermólisis Ampollosa de la Unión
/
Transgenes
/
Epidermis
Tipo de estudio:
Observational_studies
/
Prognostic_studies
Límite:
Child
/
Humans
/
Male
Idioma:
En
Revista:
N Engl J Med
Año:
2021
Tipo del documento:
Article