Carbonic anhydrase inhibitors' effects on dystrophin-deficient mouse muscle

Object: The aim of this study was to show that Caenorhabditis elegans (C. elegans) is a suitable screening model allowing the identification of potential therapeutic substances out of already proved drug compounds in mouse models. Therefore, we analysed the effect of two substances that rescued the muscular degenerative phenotype of a Dys-deficient C. elegans mutant in the mdx mouse model. Introduction: Duchenne muscular dystrophy (DMD) is an X-linked degenerative muscular wasting disease. In C. elegans mutations of the dys-1 gene in association with a mild MyoD mutation result in a muscular degenerative phenoty…