Antiviral drugs prolong survival in murine recessive dystrophic epidermolysis bullosa

ABSTRACT: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare inherited skin disease characterized by defects in type VII collagen leading to a range of fibrotic pathologies resulting from skin fragility, aberrant wound healing and altered dermal fibroblast physiology. Using a novel in vitro model of fibrosis based on endogenously produced extracellular matrix, we screened an FDA-approved compound library and identified antivirals as a class of drug not previously associated with anti-fibrotic action. Pre-clinical validation of our lead hit, daclatasvir, in a mouse model of RDEB demonstrated significant improvement in fibrosis as well as overall quality of life with increased survival, weight gain and activity, and a decrease in pruritus-induced hair loss. Immunohistochemical assessment of d

SUBMITTER: Dr. Grace Tartaglia 

PROVIDER: S-SCDT-10_1038-S44321-024-00048-8 | biostudies-other |

REPOSITORIES: biostudies-other