Muscular dystrophy may also be cured by a gene therapy technique which has been promising where hemophilia and skin diseases are concerned, according to the Stanford University School of Medicine researchers . The gene dystrophin was introduced into mice by the researchers as a part of the experiment. According to Thomas Rando, MD, there is nothing new in using gene therapy for treating muscular dystrophy, as different techniques have already been used with varying degrees of success.
The gene therapy technique Rando and postdoctoral fellow Carmen Bertoni, PhD, used was developed by Michele Calos, PhD, associate professor of genetics. One of the main advantages of this method is that it could potentially provide a long-term fix for a variety of genetic diseases, including muscular dystrophy.
In muscular dystrophy, the muscle cells break down and are slowly replaced by fat. Eventually people with the disease are confined to a wheelchair and usually die in their 20s. There is currently no effective treatment for the disease, which explains why gene therapy remains a hope despite the significant hurdles.
Rando said the PNAS paper highlights an additional requirement for any gene therapy to be successful: the introduced gene must produce healthy dystrophin protein in large quantities in order to repair the entire muscle cell. Previous muscular dystrophy gene therapy studies did not look at whether the introduced dystrophin spread along the entire length of the muscle cell, which can be many millimeters long in mice or inches long in humans.