Duchenne muscular dystrophy (DMD) is the most common genetic muscle disorder, affecting 23,000 boys in the US each year. The disease results from a mutation in the gene that encodes dystrophin — the largest gene discovered to date. Adeno-associated viruses have proven the safest and most effective vehicles for delivering therapeutic genes into the muscle tissue but they are too small to carry a dystrophin gene. Now, the development of dystrophin 'mini-genes' might have resolved that problem.
In a study published in the 5 December issue of
The authors hope this gene therapy strategy could be used in a larger animal model, and eventually to treat patients. The team will also be collaborating with other researchers to develop better ways of delivering the DMD mini-gene systemically, as opposed to performing localised injections.