What does this mean for people with Duchenne muscular dystrophy?
• Statement from Professor George Dickson • Further information What are the challenges facing the development of gene therapy for Duchenne muscular dystrophy? Viruses called adeno-associated virus (AAV) are particularly useful vehicles for delivering a healthy copy of a gene to cells affected by a genetic disorder. This is because they are able to infect human cells but do not cause any disease. Studies have also shown that the immune system doesn’t normally have a strong reaction to this type of virus. Mutations in the dystrophin gene cause Duchenne muscular dystrophy. Dystrophin is an important structural component of muscle cells and if absent or faulty the cells are fragile and deteriorate. Unfortunately the dystrophin gene is too large to fit inside adeno-associated viruses so scientists have designed abbreviated versions of the gene. They have removed sections of the gene thought to not be essential for its function, resulting in a gene that is one third the original length. The
