Abstract
Epidermolysis bullosa simplex (EBS) is a severe and potentially life-threatening disorder for which no adequate therapy exists. Most cases are caused by dominant mutations in keratins KRT5 or KRT14, characterized by cytoplasmic keratin aggregates, profound keratinocyte fragility and cytolysis. We hypothesized that pharmacological reduction of keratin aggregates, which compromise keratinocyte integrity, represents a viable strategy for the treatment of EBS. Here we show that the multi-kinase inhibitor PKC412, which is currently in clinical use for acute myeloid leukemia, reduced keratin aggregation by 40% in patient-derived K14.
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