Everyone who is following the search for treatments will have heard about ‘gene therapy’. It’s an approach that involves getting artificial genes into muscle cells and making them produce the protein which, when missing or faulty, causes muscular dystrophy. Scientists have already made an artificial gene designed to do the job of the one that is faulty in people with Duchenne MD. Their task now is to deliver copies of these ‘dystrophin’ genes efficiently into muscle and to make sure that they stay put, get to work and stay working.
At the moment the most promising way of delivering the artificial genes into muscle is by using viruses.
In the first section below you’ll be able to read in detail about this work, while other sections examine alternative methods and consider some of the practical and ethical questions involved in potential treatments and cures.