Transgenic Epidermal Cultures for Junctional Epidermolysis Bullosa - 5-Year Outcomes.
N Engl J Med
; 385(24): 2264-2270, 2021 12 09.
Article
in English
| MEDLINE | ID: covidwho-1560911
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.).
Full text:
Available
Collection:
International databases
Database:
MEDLINE
Main subject:
Transduction, Genetic
/
Keratinocytes
/
Epidermolysis Bullosa, Junctional
/
Transgenes
/
Epidermis
Type of study:
Case report
/
Cohort study
/
Prognostic study
Limits:
Child
/
Humans
/
Male
Language:
English
Journal:
N Engl J Med
Year:
2021
Document Type:
Article
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