Adapting research techniques

An exciting time for research

“It’s an exciting time for research. We are close to helping people in the near future. If it works, it could open the door for treating other forms of muscle-wasting conditions.”

Professor Matthew Wood is based at the Department of Physiology, Anatomy and Genetics at the University of Oxford. Specialising in neuroscience, he was among the UK scientists who led the development of exon skipping as a treatment for Duchenne muscular dystrophy.

“Exon skipping is a pioneering technique. It helps the body ‘skip’ over the fault on a gene needed to produce a vital muscle protein. It targets the genetic mutation which causes Duchenne muscular dystrophy.”

Muscular Dystrophy UK has invested more than £55m in research into muscle-wasting conditions. We have laid the foundations for the first promising potential treatments for Duchenne muscular dystrophy, several of which are now in clinical trials.

Research grants are vital

“The research grants we get from Muscular Dystrophy UK are vital. They often fund the early stages of our exploration. If the charity weren’t around, today’s clinical trials simply wouldn’t be happening,” said Professor Wood.

Decades ago, when a treatment for any form of muscular dystrophy was hard to conceive of, Muscular Dystrophy UK threw its weight behind developing exon skipping. Today, the technique is being tested in people with Duchenne muscular dystrophy, with promising results. It is hoped this possible treatment painstakingly developed for one condition, may have the potential to help tackle more.

Adapting the technique

Professor Wood is now working to adapt the technique for another muscle-wasting condition, spinal muscular atrophy (SMA), which affects around 2,000 people in the UK. Muscular Dystrophy UK is also funding work by scientists to lay foundations for treating other forms of muscular dystrophy with exon skipping, which could potentially help thousands more.

Muscular Dystrophy UK invests in high quality research to find effective treatments and cures as quickly as possible. We are pressing to ensure early participation for UK patients in clinical trials. We fund clinical trial co-ordinators and the national neuromuscular database to ensure vital patient data is available to clinicians and researchers.

We know we can beat muscle-wasting conditions more quickly by working together. Please join us.

Watch BBC’s Lifeline Appeal on Sunday 22 March at 4:45pm to learn more about Professor Wood and his vital research.

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