Irish scientists have proven that a treatment for muscular dystrophy involving gene therapy is efficient. This, they say, brings a cure for the disease a step closer.

Muscular dystrophy is a collective term for a range of neuromuscular conditions. It is characterised by the progressive weakening and wasting of muscles and can affect both adults and children.

The most common and life threatening form of the disease is Duchenne muscular dystrophy. It is found in boys and is the most common gender-specific inherited disease in Ireland. First symptoms usually manifest between the ages of three and five. Those affected are usually wheelchair-bound by the age of 12. Around 400 boys in Ireland are currently affected and there is no cure.

A year-long collaboration between the team at Maynooth and scientists at the University of Western Australia focused on a specific method of treating the genes within wasting muscles, known as ‘exon skipping’.

The researchers found that experimental muscles treated with this gene therapy underwent a reversal of the wasting process.

“For the past 20 years the scientific community has been working on finding a cure for this disease. We now know that this approach is the right one and most likely to achieve results for sufferers. It offers a real hope for a reversal of the devastating effects of muscular dystrophy,” explained Prof Kay Ohlendieck, head of the biology department at NUI Maynooth.

He said that these findings ‘set the scene for improved treatment options’ to counteract the symptoms of inherited muscle wasting disorders.

The next step in finding a cure for muscular dystrophy is to bring this treatment to human clinical trial stage. Similar trials have already begun in the UK and US and the results of these are expected next year.

The support group for people with the disease, Muscular Dystrophy Ireland, which helped to fund this research, described the findings as ‘very encouraging news for all our members’.

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