Duchenne Muscular Dystrophy, Prof. V. Straub

Group/Researcher: Prof. Volker Straub, University of Newcastle Upon Tyne


The focus of this grant is the development of non-invasive methods to assess muscle damage and repair. This will assist clinicians in monitoring disease progression, as well as the benefits of potential treatments.

Compared to healthy muscle, it is thought that muscle from people with muscular dystrophies is less capable of coping with mechanical damage. This could either be due to a higher susceptibility of the cells to mechanical stresses during contraction or due to defective repair mechanisms. As a result skeletal muscles gradually degenerate, causing muscle weakness and wasting.

Currently, if a clinician wishes to do a detailed analysis of muscle cell damage in a person with muscular dystrophy, a muscle biopsy must be done. This is a microscopic examination of tissue samples obtained by surgery. Muscle biopsies only allow us to look at a small portion of the muscle of interest and are not always representative of the condition of the entire muscle. Furthermore, since this is a surgical procedure it is not easy for the clinician to take repeat samples. Repeat samples are necessary to monitor and evaluate the course of a disease or can help gauge the success of a therapeutic approach.

In this study, Prof Straub and his colleagues aim to look at the muscle changes occurring during Duchenne muscular dystrophy and in a mouse model of muscular dystrophy using magnetic resonance imaging (MRI). MRI is a non-invasive technique that can be used to visualise the inside of the body. It can be used to produce two-dimensional 'slices' or it can be used to build up a three-dimensional picture of a part of the body. The use of MRI is a particularly powerful tool in medicine because of the possibility to observe how a disease progresses in an individual. In addition to this it can also be used to study the effects of drugs and therapies over time.

MRI can monitor an individual patient, or an animal model, without the need to take a tissue sample, something which can be quite uncomfortable for the patient. This is particularly useful in a clinical trial setting where the muscle must be monitored over time and many tissue samples would have to be taken. When testing a potential therapy in a clinical trial, it is important to have 'base-line' data which gives and indication to clinicians what the muscles normally look like during the course of a disease. The use of MRI will allow the collection of this information. The base-line data can be compared to muscle which has been treated with the potential therapy. This will assist the clinicians when trying to evaluate the benefits of a therapeutic treatment.

This project is being supported by the Big Lottery Fund.