Abstract
In this issue, Wally et al. (2008) report successful gene expression repair by spliceosome-mediated RNA trans-splicing (SMaRT), a novel achievement in molecular medicine. In their model, SMaRT was able to replace a mutation of the plectin gene in epidermolysis bullosa simplex with muscular dystrophy. This approach is particularly attractive for skin gene therapy of dominant-negative mutations present in a number of blistering genodermatoses.
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