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Duchenne muscular dystrophy drug discovery – the application of utrophin promoter activation screening

Introduction: Duchenne muscular dystrophy (DMD) is a devastating genetic muscle wasting disease caused by mutations in the DMD gene that in turn lead to an absence of dystrophin. Currently, there is no definitive therapy for DMD. Gene- and cell-based therapies designed to replace dystrophin have met some degree of success, as have strategies that seek to improve the dystrophic pathology independent of dystrophin. Areas covered: In this review the authors focus on utrophin promoter activation-based strategies and their implications on potential therapeutics for DMD. These strategies in common are designed to identify drugs/small molecules that can activate the utrophin promoter and would allow the functional substitution of dystrophin by upregulating utrophin expression in dystrophic muscle. The authors provide an overview of utrophin biology with a focus on regulation of the utrophin promoter and discuss current attempts in identifying utrophin promoter-activating molecules using high-throughput screening (HTS). Expert opinion: The characterisation of utrophin promoter regulatory mechanisms coupled with advances in HTS have allowed researchers to undertake screens and identify a number of promising lead compounds that may prove useful for DMD. In principle, these pharmacological compounds offer significant advantages from a translational viewpoint for developing DMD therapeutics.