Discovery of small molecule utrophin modulators for the therapy of Duchenne muscular dystrophy


Duchenne muscular dystrophy (DMD) is a devastating, X-linked muscle-wasting disease caused by lack of the cytoskeletal protein dystrophin. There is currently no cure for DMD, although various promising approaches (e.g. exon skipping, read through of stop codons, gene therapy) are being developed. By transcriptionally reprogramming the temporal and spatial expression of the dystrophin-related protein utrophin, we aim to develop a pharmacological therapy applicable to all DMD patients by targeting the primary defect.


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