Duchenne muscular dystrophy, Prof K. Davies

Group/Researcher: Prof. K. Davies, University of Oxford


Prof Kay Davies and her colleagues at the University of Oxford are attempting to find a drug that will increase the levels of a protein called utrophin. It is thought this protein may be able to compensate for the lack of dystrophin in Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is a devastating, progressive muscle wasting disease and no effective treatment is currently available. It is caused by a mutation in the gene for dystrophin which results in a lack of functioning dystrophin protein. The aim of this project is to find a drug that will increase the levels of a protein called utrophin in human muscle. Utrophin is very similar to dystrophin and experiments in mice and dogs lacking dystrophin have shown that increased levels of utrophin can prevent muscle damage.

Prof Davies and her colleagues have set up a screening system for small molecules that can increase the levels of utrophin. Initial screens have identified several types of molecule which increase the levels of utrophin in the mdx mouse (the mouse model of DMD). This work has therefore reached an exciting phase and future plans build on these results. This project aims to try to find more small molecules that can increase the production of utrophin as well as carry out more extensive investigations on those molecules that have already been shown to be effective in increasing utrophin in mice. Finally the group wishes to investigate whether increasing utrophin will have a greater effect if combined with other therapies.