An inducible mouse model for epidermolysis bullosa simplex: implications for gene therapy.
J Cell Biol
; 152(3): 651-6, 2001 Feb 05.
Article
en En
| MEDLINE
| ID: mdl-11157990
ABSTRACT
The Dowling-Meara variant of epidermolysis bullosa simplex (EBS-DM) is a severe blistering disease inherited in an autosomal-dominant fashion. Here we report the generation of a mouse model that allows focal activation of a mutant keratin 14 allele in epidermal stem cells upon topical administration of an inducer, resulting in EBS phenotypes in treated areas. Using laser capture microdissection, we show that induced blisters healed by migration of surrounding nonphenotypic stem cells into the wound bed. This observation provides an explanation for the lack of mosaic forms of EBS-DM. In addition, we show that decreased mutant keratin 14 expression resulted in normal morphology and functions of the skin. Our results have important implications for gene therapy of EBS and other dominantly inherited diseases.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Piel
/
Proteínas Virales
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Regulación de la Expresión Génica
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Epidermólisis Ampollosa Simple
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Modelos Animales de Enfermedad
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Queratinas
Límite:
Animals
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Humans
Idioma:
En
Revista:
J Cell Biol
Año:
2001
Tipo del documento:
Article
País de afiliación:
Estados Unidos