The Centre is a partnership with the University of Oxford and aims to find potential new treatments and get them to patients faster. It has already attracted world-leading researchers from across the globe with Prof Laurent Servais, an internationally renowned neurologist, joining the Centre as Professor of Paediatric Neuromuscular Diseases.
Prof Matthew Wood, Professor of Neuroscience at the University of Oxford and director of the Centre, said,
The MDUK Oxford Neuromuscular Centre will be one of the very first that takes drug development from basic research to clinical trial testing, and will also be increasing the capacity for clinical trials within the UK.
Investigating new ways to make gene therapy more effective
One of the new research projects the MDUK Oxford Neuromuscular Centre Prof Matthew Wood is undertaking is looking at how gene therapy is administered to patients.
Several gene therapies for muscle-wasting conditions are being tested in clinical trials, which use a harmless virus to deliver the desired gene into the body. Viruses can effectively carry information into our cells, making them ideal delivery vehicles for gene therapies.
However, our bodies have naturally evolved to fight viruses. This means some patients will not be able to receive gene therapy (as their bodies will already be immune to the virus carrying the gene) while others may only get one dose before their bodies become immune. Instead of viruses, the research team led by Prof Wood is using exosomes – tiny packages found naturally inside the body – to carry a smaller version of the dystrophin gene (called micro-dystrophin), which is being used as a therapy for Duchenne muscular dystrophy. This could make more people with Duchenne eligible for gene therapy in the future and have similar implications for other muscle-wasting conditions.