Innovative therapeutic strategies for recessive dystrophic epidermolysis bullosa.
Actas Dermosifiliogr
; 106(5): 376-82, 2015 Jun.
Article
em En, Es
| MEDLINE
| ID: mdl-25796272
ABSTRACT
Recessive dystrophic epidermolysis bullosa (RDEB) is among the most serious rare skin diseases. It is also the rare skin disease for which most effort has been expended in developing advanced therapeutic interventions. RDEB is caused by collagen VII deficiency resulting from COL7A1 mutations. Therapeutic approaches seek to replenish collagen VII and thus restore dermal-epidermal adhesion. Therapeutic options under development include protein therapy and different cell-based and gene-based therapies. In addition to treating skin defects, some of these therapies may also target internal mucosa. In the coming years, these novel therapeutic approaches should substantially improve the quality of life of patients with RDEB.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Epidermólise Bolhosa Distrófica
/
Terapias em Estudo
Tipo de estudo:
Prognostic_studies
Limite:
Animals
/
Humans
Idioma:
En
/
Es
Ano de publicação:
2015
Tipo de documento:
Article