DMD patients usually die by their early twenties. No effective therapies exist currently, but the results of a study described today in the Proceedings of the National Academy of Sciences may eventually lead to one.
DMD patients lack a protein dubbed dystrophin, which plays a critical role in muscle maintenance.
Xiao Xiao of the University of Pittsburgh and his colleagues thus set out to develop a system by which they could deliver dystrophin to muscles. When the team injected this construct into the calf muscles of dystrophic mice, it delivered the gene to the muscles, where it began producing dystrophin protein. In fact, the protein was expressed in nearly 90 percent of the muscle targeted.