Gene therapy for muscular dystrophy may at last be showing some success, British researchers have revealed.
A new treatment called "exon skipping" has brought "remarkable" long-term improvement in the symptoms experienced by boys with Duchenne Muscular dystrophy, researchers have reported.
The disease, a severe form of muscular dystrophy, affects one in 3,000 boys and has no known cure.
Muscular dystrophy was one of the first diseases to have its genes identified and the search for genetic therapies has frustrated families and researchers for decades.
Now scientists at Royal Holloway College, London, UK, have reported "encouraging" results from a study of the new treatment.
It works by masking the faulty part of the dystrophin gene, responsible for the disease, allowing a functional dystrophin protein to be produced. The protein is shortened – but, it is hoped, would reduced the severity of the disease.
Writing in Molecular Therapy, Professor George Dickson, of the college, reports on laboratory studies indicating the best dose of the new gene therapy.
Professor Dickson said: "These latest exciting and encouraging results suggest that current ongoing clinical trials of exon skipping in muscular dystrophy patients have great promise as a long-term treatment."
Dr Marita Pohlschmidt, director of research of the Muscular Dystrophy Campaign, said: "Finding the right dose of this new potential drug is key to its success and we are proud that the research we fund continues to shed light on this.
"Professor Dickson’s work has provided us with a good indication of what dose might be effective to improve muscle function in boys with Duchenne muscular dystrophy and we hope that these results are confirmed in clinical trials."
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