A mouse model of generalized non-Herlitz junctional epidermolysis bullosa.

Document Type

Article

Publication Date

2010

Keywords

Blister, Calcification-Physiologic, Chromosome-Mapping, Disease-Models-Animal, Epidermolysis-Bullosa-Junctional, Female, Genes-Recessive, Laminin, Leukemia-Virus-Murine, Male, Mice, Mice-Inbred-C57BL, Mice-Mutant-Strains, Respiratory-Insufficiency, Skin, Tooth, Virus-Integration

First Page

1819

Last Page

1828

JAX Source

J Invest Dermatol 2010 Jul; 130(7):1819-28.

Abstract

Epidermolysis bullosa (EB) is a class of intractable, rare, genetic disorders characterized by fragile skin and blister formation as a result of dermal-epidermal mechanical instability. EB presents with considerable clinical and molecular heterogeneity. Viable animal models of junctional EB (JEB), that both mimic the human disease and survive beyond the neonatal period, are needed. We identified a spontaneous, autosomal recessive mutation (Lamc2(jeb)) due to a murine leukemia virus long terminal repeat insertion in Lamc2 (laminin gamma2 gene) that results in a hypomorphic allele with reduced levels of LAMC2 protein. These mutant mice develop a progressive blistering disease validated at the gross and microscopic levels to closely resemble generalized non-Herlitz JEB. The Lamc2(jeb) mice display additional extracutaneous features such as loss of bone mineralization and abnormal teeth, as well as a respiratory phenotype that is recognized but not as well characterized in humans. This model faithfully recapitulates human JEB and provides an important preclinical tool to test therapeutic approaches.

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