Towards the elucidation of the mechanism of action of small molecule upregulators of utrophin using chemical proteomics

Thesis

Duchenne Muscular Dystrophy (DMD) is an X-linked recessive and progressive muscle-wasting disease caused by a lack of the cytoskeletal protein dystrophin. There is currently no disease modifying treatment for all patients, although various promising approaches (e.g. exon skipping, read through of stop codons and gene therapy) are being developed and are starting to be approved for clinical use. We aim to develop an oral small molecule upregulator that compensates for the missing dystrophin...

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Authors: Vuorinen, A

• Type of award: DPhil
• Level of award: Doctoral
• Awarding institution: University of Oxford
• Language: English