After years of dedicated work involving thousands of experiments, researchers from The University of Western Australia have developed ‘genetic bandaids’ that could alleviate symptoms in many patients with Duchenne muscular dystrophy (DMD), the most common and severe childhood form of the muscle-wasting disease.
DMD is a relentlessly progressive muscle wasting disorder, for which there is no cure. There are limited treatment options for DMD, an inherited condition that affects about one boy in 3500 and is usually inherited from the mother. DMD is caused by mutations in the dystrophin gene that corrupt the genetic message and lead to incomplete synthesis of the dystrophin protein.
Professor Steve Wilton, head of UWA’s Molecular Genetic Therapy Group, research fellow Dr Sue Fletcher and their team have designed a panel of antisense medicines that act as ‘bandaids’ to cover the lesions in the dystrophin message. The compounds developed by the UWA group allows the cell machinery to skip over the disease-causing defect and restore production of a shorter, but still functional gene product.
This personalized genetic therapy for DMD is being tested in a clinical trial in the UK in an ongoing international collaboration between the UWA group, MDEX Consortium UK, and AVI Biopharma Inc, the Oregon-based company that manufactured the first test compound.
“The trial is only appropriate for a particular group of DMD patients but will address limited preliminary safety issues and provide first time human proof-of-concept for this type of compound,” Professor Wilton said.
“The start of these trials has long been anticipated by the DMD community and researchers, and represents a new phase in finding a treatment for this disease. However, a potential treatment is still some years away and clinicians emphasize the need to continue best current clinical treatment for DMD patients in the interim.”
Professor Steve Wilton 61 8 9346 3967
Dr Sue Fletcher 61 8 9346 3583