Transgenic Epidermal Cultures for Junctional Epidermolysis Bullosa - 5-Year Outcomes.
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.).
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transdução Genética
/
Queratinócitos
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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