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Novel therapeutic approaches for the treatment of Duchenne muscular dystrophy.

Department of Biomedical Science

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About the Project

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene leading to loss of the cytoskeletal protein dystrophin. A significant consequence of this loss is the disruption of the dystrophin-associated protein complex, a group of transmembrane proteins that serve to stabilise the muscle fibre membrane by making regular-spaced connections between the extracellular matrix and the actin cytoskeleton. An essential part of this complex is the transmembrane adhesion receptor dystroglycan. We have shown that in DMD there is increased phosphorylation of dystroglycan, and that dystroglycan phosphorylation contributes to the disruption of the dystrophin-associated protein complex. We have shown using chemical inhibitors, that preventing dystroglycan phosphorylation improves the muscle pathology in zebrafish and mouse models of DMD, and that the same inhibitors also block dystroglycan phosphorylation in muscle cells from DMD patients. This project will investigate further the actions of various inhibitors in the treatment of DMD using both animal models and in vitro cell culture approaches. In animal models we will use tests of muscle function, assessment of serum biomarkers and histopathology to determine efficacy, whilst in tissue culture we will use quantitative SDS-PAGE and western blotting to identify and measure the levels of important signalling molecules. In vitro studies will also be complemented by immunofluorescence microscopy where relevant.

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Funding Notes

First class or upper second 2(i) in a relevant subject. To formally apply for a PhD, you must complete the University's application form using the following link:

All applicants should ensure that both references are uploaded onto their application as a decision will be unable to be made without this information.

Miller et al. (2012) Hum Mol Genet 21, 4508-4512. Preventing dystroglycan tyrosine phosphorylation ameliorates the dystrophic phenotype in mdx mouse.
Lipscomb, et al. (2016) Hum Mol Genet 25: 266-274. Dasatinib as a treatment for Duchenne muscular dystrophy.
G Marston and SJ Winder (2018) PLoS Curr. MD. Are soy products effective in DMD?

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